HBM New Drug Approval Report 2013

July 2013

1

HBM New Drug Approval Report 2013 Authors: Dr. Ulrich Geilinger, Rossella Belleli, Cédric Barra

Trends in New Drug Approvals by the US FDA 2003-2012

New US Drug Approvals During the First Half of 2013

Peak Sales Potential of New Drugs Approved Since 2010

This report including key data of new drugs (NMEs) approved by the FDA since 2003 is available under www.hbmpartners.com/report.

About the HBM New Drug Approval Report

HBM Partners is supporting two master theses carried out at the University Basel and the Swiss Federal Institute of Technology Zurich (ETHZ). Rosella Belleli is researching the clinical development characteristics of new therapeutic drugs approved from 2003 to 2012 (Master thesis at University of Basel, Department of Public Health, Prof. Dr. med. Thomas D. Szucs, European Center of Pharmaceutical Medicine). Cédric Barra is analyzing the collaborations during the drug development process within the pharmaceutical industry (Master thesis at ETHZ, Department of Management, Technology and Economics, Prof. Dr. Georg von Krogh, Chair of Strategic Management and Innovation).

For their master thesis, Belleli and Barra have with the support of HBM Partners compiled certain data on new drug approvals available from the FDA website (www.fda.gov) as well as from other public sources (mainly press releases by drug sponsors). The HBM New Drug Approval Report provides an initial analysis of this data. Further data used for the above-mentioned master theses will be added by the two researchers.

We hope that this data will be of use to industry participants and researchers as a basis for further analysis and research on the topic of new drug approvals.

The authors of the report welcome any feedback or corrections:

Dr. Ulrich Geilinger, [email protected] Rossella Belleli, [email protected] Cédric Barra, [email protected]

http://www.hbmpartners.com/report

http://www.fda.gov/

mailto:[email protected]



HBM New Drug Approval Report 2013

2

Trends in New Drug Approvals by the US FDA 2003-2012

Introduction

For 2011 and 2012, the US FDA reported significantly higher numbers of novel drug approvals (also called New

Molecular Entities or NMEsa) as compared to previous years

1. This is certainly good news for the

pharmaceutical and biotechnology sectors, as the industry is faced with increasing drug development spending

that (until 2011) did not result in an increase of the number of novel drugs approved. The “productivity crisis”

in pharmaceutical drug development has been the topic of numerous reports and studies. The recent jump in

new drug approvals by FDA in 2011 and 2012 was commented upon extensively in the press. FDA itself in its

"FY 2011 [and FY 2012] Innovative Drug Approvals" publications highlights that these new drugs include

"groundbreaking treatments" for several diseases. The reports also emphasize that in 2011 and 2012, 64% and

75%, respectively, of all novel drugs were first approved in the US. New NDA/BLA applications per year were

pretty much in-line with approvals2 so the recent increase of approvals is not due to a “backlog effect”.

Chart 1: NMEs Approved by FDA 2003-2012

1HBM Partners

New NMEs Approved by FDA 2003-2012 (NCEs & Biologics)

Source: FDA, HBM analysis / NME = new molecular entity, NCE = new chemcial entity, i.e. small molecule

4 2 4 2 36 6 6 6

21

32

1818

16

2120

15

24

3321

36

2022

18

2426

21

30

39

21

28

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2003 2004 2005 2006 2007 2008 2009 2010 2011 2012

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New NCEs approved (FDA statistics)

New biologics approved (FDA statistics)

New therapeutics approved (HBM analysis)

Source: FDA (www.fda.gov), HBM analysis / Notes: NCEs = New chemical entities, i.e. new small molecules

Note: 2003 FDA numbers do not include biologics

This report analyses the trends in novel drug approvals by the FDA for the ten-year period from 2003 to 2012

and provides some perspective on the recent increase of such approval numbers. We mainly compare the five

years from 2008 to 2012 with the five previous years, as well as the years 2011 and 2012 (the years with the

highest approval numbers) with the eight preceding years (2003 to 2010). It should be noted that the number

of novel drug approvals as published by the FDA (green and grey bars in the chart above) also include certain

diagnostics agents, adjuvants, antidotes and other non-therapeutic agents. For the purpose of our analysis, we

have therefore reclassified the data, limiting it to novel therapeutic drugs approved for human use by the FDA

(highlighted orange in the chart above). We have excluded preventive vaccines from our analysis as these

include new versions of seasonal vaccines and only some vaccines are included in the FDA statistics. We added

three new biologics (1 in 2003, 1 in 2010 and 1 in 2011) that are not included in the FDA numbers. For more

details of inclusion/exclusion of NMEs see page 3 and Annexe 1. In this report, we focus on therapeutic NMEs

and the terms “new drugs” or “NMEs” will be used with this meaning. During the ten year period from 2003 to

2012, 237 such therapeutic NMEs were approved by the FDA.

a Quote from “FY 2011 Innovative Drug Approvals”, FDA, November 2011: “… NMEs are drugs, including biological products, with novel chemical structures that have never been approved before to treat any disease, and often represent the most innovative drugs entering the market. These drugs include products approved by FDA’s Center for Drug Evaluation and Research [CDER] and FDA’s Center for Biologics Evaluation and Research [CBER].” The term “New Active Substance” (or NAS) is also used sometimes in this context.

Total 237

Total 257

http://www.fda.gov/


3

FDA New Drug Approvals 2003-2012: Summary Results

Generally speaking, our analysis shows that the increase in number of novel drug approvals (therapeutic NMEs)

during the second half of the ten years analyzed and specifically during 2011 and 2012 is broad-based, i.e.

there was an increase in most therapeutic areas and in most drug categories (NCEs, biologics, orphan and non-

orphan drugs, priority and standard review etc). Therapeutic areas with a

particularly high increase in approvals were: oncology, genetic diseases,

immunology, hematology, ophthalmology, GI

and respiratory diseases. Above-average growth

in approvals was seen in biologics (until 2011).

Both in 2011 and in 2012, a record number of

over 10 orphan drugs were approved. With six

new orphan drugs approved until June, 2013 is likely to become another strong year

for orphan drug approvals.

Both large pharma and smaller biopharma companies have contributed to the increase in approvals. Approval

numbers both for NMEs originated in-house as well as for partnered/acquired compounds (45% of all drugs

approved 2003-2012) show a positive trend. Drugs developed in-house at large pharma accounted for about

20% of all approved novel drugs in these ten years with a constant

share in 2011 and 2012. Thus, the notion that large pharma’s

internal research and development has become less productive is

not supported by the recent drug approval numbers. Note: Large

pharma’s share of novel drugs approved (including licensed or

acquired compounds) has been consistently around one third.

Over the ten-year period, 51% of the novel drugs approved by FDA originated from US companies or

institutions and 58% were owned by US companiesa. 2012 approval numbers indicate a growing dominance of

US companies as sponsors or owners of new drugs (66% share). 33% of the NMEs approved by FDA originated

in Europe, 31% were owned by European companies at time of approval.

Japanese pharma companies only received 15 drug approvals in the US

(6% share), but 25 of the approved NMEs originated in Japan. Companies

in the “rest of the world”b still play a relatively minor role as sponsors of

new US drugs with a share of around 5% to 10% (depending on the year).

A higher number of novel drugs approved (as in 2011 or 2012) does not necessarily mean that large numbers of

patients will have access to high-efficacy drugs. A third of the novel drugs approved in 2011-2012 were Orphan

Drugs, targeting only small patient populations. And at least some of the 21 approved cancer drugs in the two

years provide only a modest average survival benefit for patients. On the other hand, other approvals (not

included in our analysis) such as new formulations, new drug

combinations or new indications for existing drugs may also

provide significant clinical benefits and substantial sales

potential. Nevertheless, NMEs usually present the most

innovative medicines coming to

market and thus the number of NMEs approved is a key measure of success for

the biopharmaceutical industry. The peak sales potential of NMEs approved in

2012 was estimated to be $30.4 billion, up from $22.6 billion for the 2011

approvals. Furthermore, ten drugs approved in each 2011 and 2012 have “blockbuster” potential, i.e. they

could reach $1 billion of sales or more.

a At time of approval; including drugs where US rights were owned by US companies.

b NMEs approved by companies located in Korea, Australia, Canada and Israel. No approvals for firms located elsewhere.

„The notion that large pharma’s

internal research and development

has become less productive is not

supported by the recent drug

approval numbers.”

„Peak sales potential of new drugs

approved in 2012 was estimated to be

$30.4 billion, up from $22.6 billion

for drugs approved in 2011.”

„Therapeutic areas with high

increases in approvals were:

oncology, genetic diseases,

immunology, hematology,

ophthalmology, GI and

respiratory diseases.”

„2012 approval numbers

indicate a growing dominance

of US companies as sponsors

or owners of new drugs.”

“Both in 2011 and

in 2012, a record

number of over 10

orphan drugs were

approved.”

„Ten drugs approved in

each 2011 and 2012 have

blockbuster potential.”


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The “Real” Number of New Drugs

According to the FDA, New Molecular Entities (NMEs) are "drugs with novel chemical structures as well as

biological products that have never been approved in the U.S. to treat any disease; these generally represent

the most innovative drugs entering the market."3. FDA further notes that "many of these products contain

active moieties that have not been approved by FDA previously, either as a single ingredient drug or as part of

a combination product.”4

As mentioned, the approvals of NMEs published by FDA in its "Drug and Biologic Approval Reports"5 (used as a

reference by most other secondary sources) include a number of new molecules that are not therapeutic

medicines. These are diagnostic imaging agents, diagnostic pre-treatment agents, adjuvants, poison antidotes

or cosmetics, all of which have been excluded from our analysis. We have further excluded the preventive and

seasonal vaccines approved, most of which are not listed in the FDA’s "Drug and Biologic Approval

Reports"a.Raxibacumab, an new antibody approved in 2012 to treat potential Anthrax infections is also not

included in our numbers. For our analysis, we added the following three therapeutics approved by the Center

for Biologics Evaluation & Research (CBER): Advate (a recombinant antihemophilic factor approved in 2003),

Provenge (a cell-based cancer therapy approved in 2010) and Corifact (the first coagulation factor XIII approved

in 2011). Provenge, the first cancer vaccine on the market, is included even it is not strictly speaking a

“molecular entity”). For details of the NMEs excluded/included in our analysis, please refer to Annexe 1.

In this report, we do not cover approvals of new formulations, new combinations of existing compounds or

approvals of new indications for existing drugs.

The graph below provides an overview of „our“ approval numbers as compared to the FDA numbers. The

number of therapeutic NMEs approved (HBM numbers) show a 26% increase for the five years 2008-2012 as

compared to the previous five years (FDA numbers show a more modest increase of 20%). For 2011-2012, the

average number of new therapeutics approved per year jumped by 48% as compared to the eight previous

years (similar increase for FDA approval numbers).

Chart 2: New Drug Approvals 2003-2012 – FDA Numbers vs. HBM Analysis

2HBM Partners

New NMEs/Drugs Approved by FDA 2003-2012

Source: FDA, HBM analysis / NME = new molecular entity

2128

18 2117 20

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New antidotes, adjuvants etc.

New diagnostic agents

New drugs / therapeutic NMEs (HBM analysis)

New NMEs (FDA statistics)

Including

AdvateIncluding

Provenge

Including

Corifact

Source: FDA, HBM analysis

a Provenge, the first therapeutic cancer vaccine approved in 2010, is included in our numbers.

Total 237

Total 257


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The table below provides an overview of the new drug approvals by certain drug categories and by therapeutic

areas. Above-average increases of approvals during 2008-2012 (compared to 2003-2007) and 2011-2012

(compared to 2003-2010) are underlined.

Table 1: New Drug Approvals by FDA 2003-2012 – Overview

10 Years 5 Years 5 Years % Increase 2 Years % Increase

2003-12 2003-07 2008-12 2011-12 (note 1)

New NMEs Approved (FDA Numbers)

All approvals 257 117 140 20% 69 47%

New Therapeutic NMEs Approved (HBM Numbers)

All approvals 237 105 132 26% 64 48%

Approvals p.a. 23.7 21.0 26.4 32

Biologics (BLA) 41 13 28 115% 12 66%

% share 17% 12% 21% 19%

Orphan Drugs 74 29 45 55% 23 80%

% share 31% 28% 34% 36%

Fast Track 62 28 34 21% 29 252%

% share 26% 27% 26% 45%

Priority Reviews 109 53 56 6% 28 38%

% share 46% 50% 42% 44%

Accelerated Approval 28 16 12 -25% 8 60%

% share 12% 15% 9% 13%

US Sponsors 138 65 73 12% 39 58%

% share 58% 62% 55% 61%

European Sponsors 74 33 41 24% 17 19%

% share 31% 31% 31% 27%

Japanese Sponsors 15 4 11 175% 3 0%

% share 6% 4% 8% 5%

ROW Sponsors 10 3 7 133% 5 300%

% share 4% 3% 5% 8%

External (note 2) 105 43 62 44% 25 25%

% share 44% 41% 47% 39%

Large Pharma (note 3) 80 36 44 22% 25 82%

% share 34% 34% 33% 39%

Approvals by 10 Years 5 Years 5 Years % Increase 2 Years % Increase

Therapeutic Area 2003-12 2003-07 2008-12 2011-12 (note 1)

Cancer/Cancer Related 54 21 33 57% 21 155%

Infections 35 23 12 -48% 7 0%

CNS/Pain/Behaviour 31 13 18 38% 5 -23%

Genetic 17 5 12 140% 5 67%

Cardiovascular/Vascular 15 7 8 14% 3 0%

Immunology 14 2 12 500% 4 60%

Hematology 11 6 5 -17% 3 50%

Ophthalmology 11 4 7 75% 3 50%

Metabolic/ Endocrinology 10 5 5 0% 2 0%

Urology/Nephrology 10 7 3 -57% 2 0%

Gastroenterology 9 4 5 25% 3 100%

Diabetes 8 5 3 -40% 1 -43%

Respiratory 6 2 4 100% 4 700%

Women's Health 4 1 3 200% - -100%

Dermatology 2 - 2 n.a. 1 300%

Note 1: Increase of average number of drugs approved per year 2011-12 as compared to 2003-10

Note 2: Drugs originating externally (usually in-licensed or acquired by drug sponsor)

Note 3: Large pharma companies with a market capitalization of $50 billion or more at time of drug approval.


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Highlight: New Drug Approvals during the First Half of 2013

So far, 2013 appears to be another good year for new drug approvals. Up to the end of June 2013, FDA

approved 13 therapeutic NMEs. The estimated peak sales potential of these new drugs $12.4 bilion. Five new

drugs have an estimated peak sales potential of $1 billion or more (Tecfidera, Breo Ellipta, Kadcyla, Invokana

and Xofigo). Pomalyst and Nesina could also reach the billion-dollar mark. The drug with the largest market

potential clearly is Biogen Idec’s new oral multiple sclerosis drug Tecfidera with expected peak sales of

$2.0-3.5 billion.

Chart 3: New Drug Approvals H1 2013

4HBM Partners

Peak Sales Estimates of New Drugs Approved by FDA 2010 – H1 2013

Peak Sales

Estimate H1

($ billion)

Source: FD, HBM Analysis

New therapeutics NMEs only

913

811

8 811 10

1713 13

12

15

10

10

912

1512

12 22

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2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013

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First Half


Approvals by therapeutic area: Approvals in oncology continued to be strong with 5 new cancer drugs so far

(43% of approvals). Three of these could reach sales of over $1 billion: Xofigo (Bayer) for late-stage metastatic

castration-resistant prostate cancer, Kadcyla (Genentech/Roche) for metastatic breast cancer and Pomalyst

(Celgene) for third-line treatment of multiple myeloma.

Approvals were granted for two new diabetes drugs: Nesina, a further DPP4 inhibitor launched by Takeda, and

Invokana, the first approved SGLT2 inhibitor. Invokana was licensed by Janssen/Johnson & Johnson from

Mitsubishi Tanabe and has the potential to reach blockbuster status.

Other NMEs approved during the first half of 2013 were: Kynamro (Genzyme, homozygous familial

hypercholesterolemia), Kcentra (CSL, acute bleeding), Breo Ellipta (Glaxo Smith Kline, COPD) and Osphena

(Shionogi, dyspareunia). Of these, Breo Ellipta, an inhaled combination drug with a new ingredient, has the

highest market potential (peak sales estimates range from $550 million to $4 billion).

Approvals by size of company: Drug approvals during 2013 were so far dominated by large pharma companies

with 6 approvals (in prior years, they had a significantly lower share of approvals). No approval was granted to

a small company (with a market cap of less than $1 billion). More than half of the newly approved drugs (7 of

13) were in-licensed or acquired by the drug sponsors (in previous years, the majority of approved drugs

originated in-house).

Biologics: Of the 13 NMEs approved, only three were biologics (approved as a BLA): Kcentra, a prothrombin-

complex derived from blood plasma to stop acute bleeding (CSL); Kadcyla, an antibody-drug conjugate

consisting of the monoclonal antibody trastuzumab (Herceptin) linked to a cytotoxic agent (Genentech/Roche);

and Rixubis, a factor IX protein to prevent or stop bleeding episodes in hemophilia B patients (Baxter).

Orphan drugs: Almost half of the new drugs (6 out of 13) were approved with Orphan Drug Designation. The

trend of an increasing share of orphan drug approvals therefore continued.


7

Highlight: Peak Sales Potential of New US Drugs Approved Since 2010

We have collected peak sales estimates for most therapeutic NMEs approved by FDA in 2010 and later. Such

estimates are usually provided by banking analysts and pharma consultants. Wherever available, we used

consensus estimates or – in the case of different estimates – we calculated the averagesa. In most cases, the

numbers relate to the worldwide market potential. Thus, approvals in other jurisdictions will be needed to

achieve such peak sales. For our analysis, we used the peak sales as estimated at the time of approval (these

estimates are usually adjusted thereafter based on the actual sales results). It should be noted that peak sales

estimates (at the time of approval) are generally optimistic and actual sales more often than not fall short of

expectations.

Chart 4: Peak Sales Potential of New Drugs Approved by FDA 2010 – H1 2013

5HBM Partners

Peak Sales Potential of New Drugs Approved by FDA 2010 – H1 2013

Source: FDA, HBM Analysis / NME = new molecular entity

17.522.6

30.4

12.4

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2010 2011 2012 H1 2013

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+ 35%

Number of NMEs

approved



The higher number of novel drugs approved by FDA in 2012 is also reflected in a higher total peak sales

potential for these drugs (+35% as compared to 2011). While our analysis of peak sales estimates of earlier

years is still “work in progress”, the “class of 2012” seems to have the highest commercial potential of new

drugs approved in any year since 2003. The table on the next page provides a summary of peak sales estimates

of new drugs since 2010, including a list of potential blockbuster drugs.

Average peak sales per drug: Average expected peak sales for new drugs have risen from around $800 million

per drug in 2010 and 2011 to $950 million in 2013. This trend of increasing peak sales per new drug clearly

contradicts other studies such a recent the publication by Accenture „The New Reality: Maximizing Product

Launches in the Post-Blockbuster World”6 which forecasts a decrease in revenue potential per new drug.

Therapeutic areas with very high average peak sales per drug of over $1 billion were immunology (8 drugs

approved since 2010), hematology (6 drugs) and diabetes (4 drugs). Low average peak sales (below $0.5 billion)

of new drugs were observed in CNS/Pain (7 drugs approved since 2010), women’s health (4 drugs), GI (3 drugs),

urology/nephrology (2 drugs) and dermatology (2 drugs).

a Peak sales estimates were not available for some drugs, mainly for niche drugs and drugs launched by private companies. We assume that the revenue potential of these drugs (without peak sale estimates) is modest, so our aggregated peak sales should encompass substantially all the commercial potential of the newly approved drugs.


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Table 2: Peak Sales Potential of New Drugs Approved by FDA 2010 – H1 2013

2010 2011 2012 H1 2013 Total

# of NMEs (note 1) 22 29 35 13 99

Peak Sales Estimates ($ bn) 17.5 22.6 30.4 12.4 82.8

Peak Sales per NME ($ mn) 795 780 868 951 837

Peak Sales Estimates ($ bn) by Therapeutic Area

Cancer 2.2 6.2 10.6 4.5 23.5

Immunology 5.5 1.7 3.2 2.8 13.2

Hematology 2.0 1.7 4.3 0.2 8.2

Infections (note 2) 0.2 5.3 2.0 0.0 7.6

Diabetes 2.5 1.3 0.0 2.3 6.1

Genetic diseases 2.0 0.7 2.5 0.4 5.6

Potential Blockbusters Gilenya (MS)

NovartisIncivek (HCV)

Vertex

Eliquis (Anti-coagulant)

BMS

Tecfidera (MS)

Biogen Idec

Victoza (Diabetes)

Novo Nordisk

Eylea (AMD)

Regeneron

Xeljanz (RA)

Pfizer

Breo Ellipta (COPD)

GSK

Actemra (RA)

Roche

Yervoy (Melanoma)

BMS

Xtandi (Prost. cancer)

Medivation

Kadcyla (Breast cancer)

Roche

Pradaxa (Anti-coagulant)

Boehringer Ingelheim

Victrelis (HCV)

Merck & Co.

Perjeta (Breast cancer)

Roche

Invokana (Diabetes)

Johnson & Johnson

Lumizyme (Pompe)

Genzyme/Sanofi

Xarelto (Anti-coagulant)

Johnson & Johnson

Belviq (Obesity)

Arena Pharmaceuticals

Xofigo (Prost. cancer)

Bayer

Brilinta (ACS)

Astra Zeneca

Kalydeco (Cyst. Fibr.)

Vertex Pharmaceuticals

Zytiga (Prostate cancer)

Johnson & Johnson

Stribild (HIV)

Gilead Sciences

Tradjenta (Diabetes,

Boehringer Ingelheim)

Kyprolis (Mult. myel.)

Onyx Pharmaceuticals

Benlysta (Lupus)

Human Genome

Sciences

Erivedge (Skin cancer)

Roche

Xalkori (Lung cancer)

Pfizer

Fycompa (Epilepsy)

Eisai Note 1: Therapeutic NMEs approved / Note 2: Mostly drugs to treat HCV and HIV

Peak sales by therapeutic area: New cancer drugs approved since 2010 have by far the largest commercial

potential ($23.5 billion) with an increasing share of total peak sales. New drugs in immunology are expected to

generate peak sales of $13.2 billion. Two blockbuster products were approved in multiple sclerosis (Tecfidera

and Gilenya) and two in rheumatoid arthritis (Xeljanz and Actemra). Approvals for three new anti-coagulants

(Eliquis, Pradaxa and Xarelto) lifted the revenue potential in the hematology area to $8.3 billion. Most of the

$7.6 billion of revenue potential of new anti-infectives comes from two new Hepatitis C drugs (Incivek and

Victrelis) and two new HIV drugs (Virtrelis and Edurant). In diabetes, 4 new drugs – all with substantial market

potential – were approved. If one adds Belviq (obesity) with estimated peak sales of around $2 billion, then the

diabetes/obesity area looks quite strong. Finally, new (orphan) drugs to treat genetic diseases are expected to

generate more total sales than the new drugs in broader disease areas such as CNS/pain, cardiovascular or

respiratory diseases.

Biologics (see chart on next page): Total expected peak sales of new biologics (approved as BLA) dropped from

over $6 billion for those approved in 2010 and in 2011 to below $4 billion in 2012, with fewer biologics

approved in 2012. The commercial potential of new biologics as a percentage of total expected sales of new

drugs seems to be trending downward from a high of 40% to below 20% in 2012 (and in H1 2013). Also,

average peak sales estimates of biologics do not differ significantly from the estimates for NCEs. Recently

approved biologics even seem to have a lower revenue potential than NCEs. However it should be noted that

the potential peak sales on average for the 7 antibodies approved since 2010 is still high ($1.5 billion.)


9

Chart 5: Peak Sales Potential of New Biologics (BLAs) Approved by FDA 2010 – H1 2013

6HBM Partners

Peak Sales Estimates of New Biologics (BLA) Approved by FDA


Source: FDA, HBM Analysis

6.6 6.2

3.62.1

7 7

5

3

1'099

879

723 700

908

748

922

1'026

0

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800

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2010 2011 2012 H1 2013

Ave

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($ m

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Pea

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)

Avg. peak salesbiologics

Number ofnew biologcs

approved

Avg. peak salesNCEs

Source: FDA, HBM analysis / Note: In 2010, peak sales estimates available for only 6 biologics

Orphan drugs: As discussed later in this report, orphan drug approvals have risen strongly since 2011,

accounting now for about 30-40% of all new drug approvals. This trend continued during the first half of 2013.

As peak sales estimates for orphan drugs are on average only about half of those for other drugs, their

commercial impact is lower. Nevertheless, new orphan drugs account for up to a quarter of the total

commercial potential of approved NMEs.

Chart 6: Peak Sales Potential of New Orphan Drugs Approved by FDA 2010 – H1 2013

7HBM Partners

Peak Sales Estimates of New Orphan Drugs Approved by FDA

3.3

6.17.1

2.2

6

1211

6

19%

27%23%

18%

0%

10%

20%

30%

40%

50%

60%

0

2

4

6

8

10

12

2010 2011 2012 H1 2013

Pea

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Est

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es($

bill

ion

)

Number of new

orphan drugs

approved



% of peak sales

estimates of

all aproved drugs



10

Trends in New Drug Approvals by US FDA 2003-2012 (continued): Biologics vs. NCEs

Of all new drugs approved during the ten-year period, 41 (or 17%%) were biologics (approved as a Biologic

License Application or BLA, highlighted in green in the chart below)a. The number of biologics approvals has

risen, at least until 2011. Rising biologics approvals thus have been clearly one of the factors driving up drug

approval numbers in recent years. The high number NCEs approved in 2012 indicate, however, that small

molecules are making a “come back”.

Chart 7: New Drugs Approvals 2003-2012 – Biologics vs. NCEs

8HBM Partners

New NCEs and Biologics Approved by FDA 2003-2012

14 2 4 2 3

6 7 7 521 4

32

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2003 2004 2005 2006 2007 2008 2009 2010 2011 2012

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New NCEs (NDA)

New other large molecules (NDA)

New biologics (BLAs)


Source: FDA / NCE = new chemcial entity, i.e. small molecule


A number of large molecules (mainly peptides) have been approved by CDER under an NDA. These –

highlighted in orange in the graph above – are included in the further analysis of biologics approvals. The graph

below shows the new biologics (and large molecules) by type. There was a marked increase in new antibodies

approved in 2009, 2010 and 2011. In 2012, only one new therapeutic antibody (Perjeta, treatment of

metastatic breast cancer, sponsored by Genentech) was approved. A further antibody, Raxibacumab against

Anthrax infections, was also approved in 2012 (not included in our numbers).

Chart 8: New Biologics / Large Molecule Approvals by Type of Drug

9HBM Partners

New Biologics and Large Molecules Approved by FDA 2003-2012

32

1 1

4

23

11

2

41

2

2

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21

2

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1

2

2

1

1 3

1

2

2

1

1

0

5

10

2003 2004 2005 2006 2007 2008 2009 2010 2011 2012

# o

fN

ew B

iolo

gic

s/L

arg

e M

ole

cule

sA

pp

rove

db

yF

DA

Other

Peptides

Other Proteins

Enzymes

Antibodies




a The number of biologics in Chart 7 differ slightly from the “official” FDA numbers for biologics approvals in Chart 1. In Chart 7 we included Advate (2003) as 2003 FDA numbers do not yet cover biologics. In 2010 Provenge and in 2011 Corifact were added. Raxibacumab, approved in 2012, is excluded.


11

Of the ten therapeutic antibodies approved since 2009, seven were fully human, four were developed for

cancer treatment and four for immunology indications.

Enzymes and other protein drugs also saw more approvals recently. Enzyme approvals were mostly related to

orphan drugs treating genetic diseases. The drugs marked as "Other " were Provenge (2010), the first

therapeutic cancer vaccine to reach the market, and Corifact (2011), the first coagulation factor XIII approved.

It should be mentioned that Provenge is not really a “molecular entity”.

Of the 53 biologics and large molecules approved, 13 (or 24%) were owned at approval by large pharma

companies, including seven antibodies. Note: According to our information, only three antibodies approved by

big pharma were discovered in-house. Not unexpectedly, large pharma’s share of new biologics and large

molecules approved is lower than its share of new small molecule drugs (36% share).

Interestingly, small molecules (NCEs) were more frequently in-licensed or acquired by M&A (49% of NCEs) as

compared to biologics or large molecules. 41% of the approved antibodies were in-licensed or acquired.

Enzymes and other proteins, as well as peptides were mostly developed in-house.

The following companies had two or more biologics and/or large molecules approved during the ten-year

period from 2003 to 2012:

Roche: Fuzeon* in 2003, Mircera in 2007, Actemra in 2010 and Perjeta in 2012. Genentech (now owned by

Roche) also had two blockbuster biologics approved (Avastin in 2004 and Lucentis in 2006)

Amgen: Kepivance in 2004, Vectibix in 2006, Nplate in 2008 and Prolia in 2010

Bristol-Myers Squibb: Orencia in 2005, Nulojix and Yervoy in 2011

Sanofi: Zemaira in 2003 (developed by Aventis), Apidra* in 2004 and Zaltrap in 2012. Genzyme (now owned

by Sanofi) also had two approvals (Myozyme in 2006, Lumizyme in 2010). It should be noted that Myozyme

and Lumizyme are substantially the same product with a different manufacturing process.

Amylin: Byetta* in 2005 and Symlin* in 2005

Johnson & Johnson: Simponi and Stelara in 2009

Novo Nordisk: Levemir* in 2005 and Victoza* in 2010

Regeneron: Arcalyst in 2008 and Eyela in 2011

Shire: Elaprase 2006 and Vpriv* 2010

* Approved under an NDA. All such approved products are peptides, except for Vpriv which is an enzyme.


12

Orphan Drugs

Orphan Drug Designation is granted to products developed to treat a rare disease or condition (affecting fewer

than 200,000 people in the US). The sponsor of an orphan drug receives certain benefits such as extended

exclusivity periods. During the ten-year period studied, 74 of the 237 newly approved drugs (31%) had received

an Orphan Drug Designation. Orphan drug approvals were most common in cancer (28 orphan drug approvals

during 2003-2012, 52% of all cancer approvals), genetic diseases (all 17 approvals had orphan drug

designation), endocrinology (7) and CNS/pain (6).

Chart 9: New Orphan Drug Approvals 2003-2012

10HBM Partners

New Orphan Drugs Approved by FDA 2003-2012

1 12

12 2

3 32

1 32

3

22

3

7

5

1

54

33

4

3

2

4

14%

29%

39%

24%

35% 35% 35%

27%

41%

31%

0%

15%

30%

45%

0

5

10

15

2003 2004 2005 2006 2007 2008 2009 2010 2011 2012

# of New OrphanDrugs Approved

by FDA

Other

Cancer

Genetic

% Share of All

Drugs Approved




Over the ten years studied, the number of new orphan drugs approved rose significantly. More than ten

orphan drugs were approved both in 2011 and in 2012. These record numbers were driven by the high number

of new cancer drugs with Orphan Drug Designation and the continued attraction of rare genetic diseases where

drug reimbursement is still very attractive. As we show in the next chapter, orphan drugs are more likely to

receive Fast Track, Priority and/or Accelerated Approval (see pages 18ff) providing a potentially faster path to

market.

Large pharma companies sponsored about one third of all orphan drugs approved in the ten-year period, in line

with large pharma’s overall share of new drugs approvals. Their share of orphan drug approvals has been

increasing over the years, indicating that large companies have become more interested in the field. Another

factor could be that, increasingly, new drugs are initially targeted to a narrow indication and the field of use is

later broadened by subsequent trials and approvals. As expected, smaller companies (with a market cap at

approval of $1 billion or less) more often chose orphan diseases for drug development, as timelines are usually

shorter and costs lower.


13

New Drug Approvals by Therapeutic Area

We have categorized the new drugs approved by therapeutic areas as listed in the table below. Most obvious is

the increase in cancer drugs (including cancer-related treatments). In 2011 and 2012, cancer drugs accounted

for almost one third of approvals. Drugs (all orphan) approved to treat genetic diseases also grew strongly.

Substantially more immunology drugs were approved in the second half of the decade, mainly targeting

autoimmune diseases such a rheumatoid arthritis, multiple sclerosis, lupus, Crohn’s disease etc. Approvals of

three new anti-coagulants (one each in 2010, 2011 and 2012 each) drove up approval numbers in the

hematology category. Approval numbers in ophthalmology continued to be quite strong, following in the

footsteps of Lucentis, which was approved in 2003. Last, but not least, three new COPD drugs since 2010

pushed approval numbers higher in the respiratory field.

Table 3: New Drug Approvals by Therapeutic Area

Therapeutic Area 2003-2012 2003-2007 2008-2012 % Change 2011-2012 % Change *

New Therapeutic NMEs 237 105 132 26% 64 48%

Cancer/Cancer Related 54 21 33 57% 21 155%

Infections 35 23 12 -48% 7 0%

Anti-Virals 15 10 5 -50% 4 45%

Anti-Bacterials 12 8 4 -50% 2 -20%

Anti-Fungal/Parasites 8 5 3 -40% 1 -43%

CNS/Pain/Behaviour 31 13 18 38% 5 -23%

Genetic 17 5 12 140% 5 67%

Cardiovascular/Vascular 15 7 8 14% 3 0%

Immunology 14 2 12 500% 4 60%

Hematology 11 6 5 -17% 3 50%

Ophthalmology 11 4 7 75% 3 50%

Metabolic/ Endocrinology 10 5 5 0% 2 0%

Urology/Nephrology 10 7 3 -57% 2 0%

Gastroenterology 9 4 5 25% 3 100%

Diabetes 8 5 3 -40% 1 -43%

Respiratory 6 2 4 100% 4 700%

Women's Health 4 1 3 200% - -100%

Dermatology 2 - 2 n.a. 1 300%

# of Approvals

Source: FDA, HBM Analysis / * = Increase of average number of drugs approved per year 2011-12 as compared to 2003-10


14

New Drug Approvals by Location of Drug Sponsor

Over the ten-year period, 51% of the novel drugs approved by FDA originated from US companies or

institutions and 58% were owned or sponsored by US companiesa. 2012 approval numbers indicate a growing

dominance of US companies with a 68% share. Overall, 33% of the new NMEs approved originated in Europe

and 31% were owned by European companies at time of approval. 25 drugs approved originated in Japan, but

only 15 approved drugs during the ten years were sponsored/owned by Japanese pharma companies. Thus,

rights for the US market were out-licensed quite frequently prior to approval.

Chart 10: New Drug Approvals by Location of Drug Sponsor/Owner

15HBM Partners

New Drugs Approved by FDA 2003-2012 – By Location Of Drug Sponsor

12

20

9

15

913 13

8

16

23

8

7

6

4

84

9

11

9

81

32

4

2

1

2

1 2 11

3

2

0

10

20

30

40

2003 2004 2005 2006 2007 2008 2009 2010 2011 2012

# o

fN

ew D

rug

s A

pp

rove

db

yF

DA

Rest of the World

Japan

Europe

US




Companies in the “rest of the world” still play a relatively minor role as sponsors of new US drugs but their

approval numbers have risen recently with 6 approvals since 2010. The sponsors of these 6 drugs were

Theratechnologies/Canada (Egrifta, 2010), Valeant/Canada (Potiga, 2011), Apotex/Canada (Ferriprox, 2011),

CSL/Australia (Corifact, 2011), and Teva/Israel (Synribo and Neutroval, 2012).

a At time of approval; including drugs where only the US rights were owned by US companies.


15

New Drug Approvals by Size of Drug Sponsor

Companies of all sizes have contributed to the increase of drug approvals over time. The distribution of

approvals per company size categorya has been quite stable. It is worth noting that large pharma’s share of

approvals even rose slightly in 2011 and 2012. There were only three large biotech companies with a market

cap of at least $50 billion at the time of approval: Amgen, Genentech and Gilead. Therefore, the approval

number in this category is modest.

Chart 11: New Drug Approvals by Size of Drug Sponsor/Owner

16HBM Partners

New Drugs Approved by FDA 2003-2012 – By Size of Drug Sponsor

8 6 49 9

4

105

12 1311

2

1 1

18 8

8

45

5

27

872 5

22 3

67 3

57

3

8

34 4

7

6

4

7

0

10

20

30

40

2003 2004 2005 2006 2007 2008 2009 2010 2011 2012

# o

fN

ew D

rug

Ap

pro

vals

<$1bn

$1bn - $10bn

$10bn - $50bn

Large biotech (>$50bn)

Large pharma (>$50bn)

Market Capitalization at Time of Approval




Mid-sized and smaller biopharma companies all had more approvals in recent years. In the size category with a

market cap of $1 billion or less, US companies clearly dominate with 35 out of 46 approvals for this group of

firms.

Companies with Most New Drug Approvals Since 2003

Table 4: Novel Drug Approvals by Drug Owner/Sponsor

Company Rank # of Drugs

Approved

# of Drugs

Approved

Peak Sales Estimates

of Drugs Approved

2003-2012 2010-H1 13 2010-H1 13 ($ bn)

Pfizer 1 15 5 4.7

Johnson & Johnson 2 12 6 5.2

Bristol Myers Squibb 3 11 3 5.9

Novartis 3 11 3 3.8

Sanofi 3 11 5 3.9

Glaxo Smith Kline 6 10 5 4.1

Merck & Co. 7 9 2 2.0

Roche 7 9 5 8.2

Forest Laboratories 9 7 5 1.9

Astellas 10 6 1 0.6

Amgen 11 5 1 0.8

Eisai 11 5 2 1.7

Eli Lilly 11 5 - -

Shire 11 5 2 0.4

Takeda 11 5 2 1.3 Source: FDA, HBM Analysis / Note: Includes drugs approved by companies that were acquired, e.g. Roche including Genentech etc.

a Market capitalization range at the time of the approval.


16

The table on the previous page shows the number of new drug approvals for drug sponsors/owners that had 5

or more approvals during the ten-year period. The numbers include drugs approved by companies that were

acquired by the drug sponsors after the approval, e.g. Roche’s numbers include approvals for Genentech.

The number one position goes to Pfizer (15 approvals), followed by Johnson & Johnson with 12 approvals.

Bristol-Myers Squibb, Novartis, Sanofi and GSK had 10 or more approvals each. The companies with most drugs

approvals in the ten-year period also ranked high in more recent approvals (2010 to H1 13).

New Drugs Originating from External Collaborations

Quite often, new drugs originate from small innovative biotech companies or research institutions, but are

brought to approval and marketed by larger companies. As the smaller companies developing new drugs have

limited financial means, they either out-license the compounds before approval or they get acquired (for more

information on M&A activity in the biopharma industry please refer to the “HBM Pharma/Biotech M&A

Reports”, www.hbmpartners.com/report). This aspect of “collaborative innovation” in drug development will

be further studied by Cédric Barra in his master thesis.

Of the 237 new drugs approved in the ten-year period, 80 (or 34%) were in-licensed or acquired by the drug

sponsor. A further 25 of new NMEs (or 11%) stem from company acquisitions some time before the drug was

approved. Thus, only 55% of all new drugs approved during these ten years were discovered and developed in-

house. Large pharma companies had the highest percentage of in-house developed compounds.

Chart 12: % of Approved Drugs Developed In-House, In-Licensed or Gained through M&A

17HBM Partners

55% 60% 56%46%

34% 30%31% 50%

11% 10% 13%4%

0%

20%

40%

60%

80%

100%

All Companies

Large Pharma

$1bn - $50bn >$1bn

By company acquisition

Licensed / product acquired

In-house

Market Capitalization



_____________ ________________________________

Market Cap at Time of Approval

Source: FDA, HBM Analysis / Large pharma = market cap of >$50 billion (excluding large biotech companies)

As the chart above shows, smaller companies (with a market cap below $1 billion at time of approval) in-

licensed 50% of their approved compounds. The reason for this unexpected finding may be that a number of

specialty pharma companies were built around one in-licensed or acquired product candidate with the goal to

get this compound approved. At the same time, 44 (or 42%) of the 105 NMEs that were in-licensed or acquired

originated from small companies. Small biopharma companies were thus active both on the “sell” and “buy”

side.

Our data does not support the often quoted opinion, that new drugs increasingly stem from in-licensing or

acquisitions.



17

Priority Review, Accelerated Approval, and Fast Track

FDA has implemented three paths (Priority Review, Accelerated Approval and Fast Track) in order to facilitate

faster drug approvals with the goal of “Accelerating Availability of New Drugs for Patients with Serious Diseases

… [which is] in everyone's interest, especially when the drugs are the first available treatment or have

advantages over existing treatments.”2 The three mechanisms are distinct, but can be overlapping. A new

Breakthrough Therapy path was introduced in 2012. No drug has been approved under this designation yet.

As the chart below shows, the share of new drugs approved under any of these designations has been

relatively stable over the years. In 2011 and 2012, the Fast Track approvals jumped to record numbers. Drugs

approved under Priority Review also saw an increase; Accelerated Approvals were in the normal range.

Chart 13: Drugs Approved under Priority Review, Accelerated Approval and Fast Track

11HBM Partners

New Drugs Approved vy FDA 2003-2012 (Priority, Accelerated Approvals & Fast Track)

8

1413

10

8 89

11

15

13

4

1

5

2

4

21 1

4 45

9

67

10

1

4

151448%

54%

72%

52% 53%

40%35%

59% 59%54%

0%

10%

20%

30%

40%

50%

60%

70%

0

5

10

15

20

25

2003 2004 2005 2006 2007 2008 2009 2010 2011 2012

# of

New

Dru

gs A

ppro

ved

byFD

A

Priority Review Accelerated Approval Fast Track

% Share of Drugs approved under

Priority Review, Fast Track and/or

Accelerated Approvals




Priority Review can be granted by the FDA for drugs that offer major advances in treatment, or provide a

treatment where no adequate therapy exists with the goal to a reduced FDA review timeline of 6 months as

compared to the standard 12 months.

During the ten years, 109 NMEs approved (or 46%) were granted Priority Review. Orphan drugs accounted for

over 50% of the Priority Reviews and three quarters of orphan drugs received Priority Review. Biologics also

were treated more often under Priority Review than NCEs. During the ten-year period, Priority Review was

granted most often for cancer drugs (42 drugs approved), anti-infectives (17, thereof 10 anti-virals, mostly HIV),

drugs for treating genetic diseases (14), drugs in immunology (7), ophthalmology and CNS/pain (6 each).

Chart 14: Priority Review Drug Approvals

12HBM Partners

New Drugs Approved by FDA 2003-2012 – Priority Review

3

6

24 3 2 3 3

8 8

5

8

11 6

5 66

8

7 5

38%

50%

72%

48% 47%

40%35%

50%52%

37%

0%

20%

40%

60%

80%

0

5

10

15

20

2003 2004 2005 2006 2007 2008 2009 2010 2011 2012

# of Drugs Approved

by FDA underPriority Review

Other Drugs

Cancer Drugs



% Share of All

Drugs Approved



18

As the chart on the previous page shows, the higher number of drugs approved in 2011-12 under Priority

Review is driven by the increase in cancer drugs. Over the years, the share of drugs approved under Priority

Review has ranged from 35% (in 2009) to 72% (in 2005).

Accelerated Approval on surrogate endpoints (such as biomarkers) is granted to certain drugs to treat serious

diseases and that fill an unmet medical need. Clinical benefits have to be demonstrated in post-approval

studies. According to the FDA2, more than 80 new products have been approved by way of Accelerated

Approvals. While the FDA marks the Priority Review and Fast Track approvals in its database, Accelerated

Approvals are not highlighted, so the respective numbers in our dataset (from FDA and other public sources)

might not be complete.

During the ten years, 28 new drugs (or 12%) were approved under the Accelerated Approval program. Orphan

drugs (predominantly in oncology) accounted for over two thirds of the Accelerated Approvals. According to

our information, only 4 biologics received Accelerated Approval (Adectris/antibody, Prolia/antibody,

Corifact/fractionated blood plasma product and Iplex/fusion protein).

Chart 15: Accelerated Drug Approvals

13HBM Partners

New Drugs Approved by FDA 2003-2012 – Accelerated Approvals

3

1 1 1 1 1

2

3

1

0

1 1

2

1

00

3

0

1

1

0

1

21

19%

4%

28%

10%

24%

10%

4% 5%

14%

11%

0%

5%

10%

15%

20%

25%

30%

0

1

2

3

4

5

6

2003 2004 2005 2006 2007 2008 2009 2010 2011 2012

# of New Drugs Approved

by FDA AcceleratedApproval

Other drugs

Anti-virals (HIV)

Cancer Drugs

% Share of All

Drugs Approved




Accelerated Approvals were most common in oncology (13 approvals) and in anti-HIV drugs (6), most of which

were approved during 2003-07. Other therapeutic areas with two Accelerated Approvals were hematology and

genetic diseases.

Fast Track is granted in certain cases by FDA in order to facilitate and speed up the development and review of

drugs that treat serious diseases (such as AIDS, cancer, diabetes, heart failure, depression, epilepsy etc.) and fill

an unmet medical need. An "unmet medical need" is hereby defined as a therapy in an area where none exists

yet or a therapy which is potentially superior in efficacy or avoids serious side effects compared to existing

therapies. Drugs under Fast Track enjoy certain benefits such as more frequent meetings with the FDA during

the development etc. and are also eligible for Accelerated Approval and/or Priority Review.

During the ten years, 62 of the newly approved drugs (or 26%) were developed under the Fast Track path.

Similar to Priority Review, Fast Track was granted most often to cancer drugs (34), drugs targeting genetic

diseases (9) as well as anti-infectives (8, thereof 5 HIV/HCV drugs and 3 anti-bacterials). Not surprisingly,

orphan drugs accounted for half of the Fast Track Approvals.

The strong increase in Fast Track approvals is thus related to the increasing number of drug applications in

cancer and (other) orphan drugs. Furthermore, it might be the result of FDA’s efforts to reduce the time to

market for drugs addressing particularly serious diseases.


19

Chart 16: Fast-Track Drugs Approvals

14HBM Partners

New Drugs Approved by FDA 2003-2012 – Fast Track

2

7

24

1 12

78

1 1

2

32

1

1

21

1

11

1

2

31

22

24%32% 33% 33%

6%

0%

4%

18%

52%

40%

0%

10%

20%

30%

40%

50%

60%

70%

80%

0

2

4

6

8

10

12

14

16

2003 2004 2005 2006 2007 2008 2009 2010 2011 2012

# of New Drugs ApprovedFast Track

Other drugs

Anti-bacterials

Anti-virals (HIV and Hep C)

Genetic diseases

Cancer drugs

% Share of All

Drugs Approved




Final Remarks

We hope that the analysis provided in this report is of interest to industry participants. We further hope that

the data we collected can be used by others as a basis for further research and analysis. The list of NMEs

approved by the FDA from 2003 to 2012 is available for download at www.hbmpartners.com/report. The use of

charts and data from this report is permitted with reference to “HBM New Drug Approval Report 2013”.

Please send any comments, enquiries or corrections to Ulrich Geilinger ([email protected]).

About HBM Partners

HBM Partners is a globally active, healthcare-focused investment management group

headquartered in Switzerland. HBM Healthcare Investments AG and funds advised by HBM

Partners invest in private and public companies across North America, Europe, India and

China. Since 2001, HBM has generated more than 40 trade sales and IPOs of pharma/biotech,

medtech and diagnostics companies.

More information can be found at www.hbmpartners.com.



http://www.hbmpartners.com/


20

Annexe 1: List of Excluded/Included NMEs

NMEs Excluded from HBM Approval Statistics

FDA Approval Date Year NME Name Comment

02.10.03 2003 Radiogardase Antidotes (radiation injury)

09.04.04 2004 ChiRhoStim Diagnostic Pre-Treatment

05.05.04 2004 Vitrase Adjuvant

02.07.04 2004 NeutroSpec Diagnostic/imaging

11.08.04 2004 Pentetate Calcium Trisodium Antidote (radiation injury)

11.08.04 2004 Pentetate ZincTrisodium Antidote (radiation injury)

26.10.04 2004 Amphadase Adjuvant

23.11.04 2004 Multihance Diagnostic/imaging

16.12.04 2004 Vision Blue Diagnostic Pre-Treatment

25.10.05 2005 Hydase Adjuvant

01.12.05 2005 Hylenex Adjuvant

21.06.06 2006 Anthelios SX Cosmetic product

30.08.07 2007 Ammonia 13 Diagnostic/imaging

10.04.08 2008 Lexiscan Diagnostic/imaging

03.07.08 2008 Eovist Diagnostic/imaging

19.09.08 2008 Adre View Diagnostic/imaging

22.12.08 2008 Vasovist Diagnostic/imaging

14.01.11 2011 DaTscan Diagnostic/imaging

14.03.11 2011 Gadavist Diagnostic/imaging

06.04.12 2012 Amyvid Diagnostic/imaging

16.07.12 2012 Prepopik Diagnostic Pre-Treatment

12.09.12 2012 Choline C11 Diagnostic/imaging

14.12.12 2012 Raxibacumab Anthrax treatment

Note: (Preventive) vaccines are not included in the HBM statistics!

NMEs Included in the HBM Approval Statistics

(not included in FDA's "CDER Drug and Biologic Approvals" List)

FDA Approval Date

Year NME Name Active Ingredient

25.07.03 2003 Advate Antihemophilic Factor (Recombinant), Plasma/Albumin-Free Method

30.10.10 2010 Provenge sipuleucel_T (cancer vaccine)

17.02.11 2011 Corifact coagulation factor XIII (human)


21

References:

1 “ „FY 2012 Innovative Drug Approvals”, FDA, December 2012 and “FY 2011 Innovative Drug Approvals”; FDA,

December 2011

2 “Impact Innovation Predictability Report” FDA, January 2013

3 "FY 2012 Innovative Drug Approvals", FDA, December 2012

4 “New Molecular Entity Approvals for 2012, FDA website: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm336115.htm

5 “NME Drug and New Biologic Approvals”, FDA website: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/DrugandBiologicApprovalReports/ucm121136.htm

6 „The New Reality: Maximizing Product Launches in the Post-Blockbuster World”, Accenture, 2012

http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm336115.htm

http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/DrugandBiologicApprovalReports/ucm121136.htm

http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/DrugandBiologicApprovalReports/ucm121136.htm

HBM New Drug Approval Report 2013

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