1 CBER Rare Diseases Program - Update 2011-2012 Nisha Jain, MD Chief, Clinical Review Branch DH/OBRR/CBER/FDA May 15, 2012 NORD Corporate Council.

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CBER Rare Diseases Program - Update 2011-2012

Nisha Jain, MDChief, Clinical Review BranchDH/OBRR/CBER/FDA

May 15, 2012NORD Corporate Council

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Overview

• Products approved in 2011-2012• Other updates• Hopeful Future

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Therapeutic areas Orphan Designated Products

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Metabolic cancer Hematological Neurological Other

Of 70 biological products designated 28 are CBER products

Total 14 grants awarded, 1 was for CBER product

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Product Approvals

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6

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OBRR OCTGT Total

Total # of Products

Products for Rare diseases

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Approvals

• Sipuleucel-T: manufactured by Dendreon – Indication: For the treatment of asymptomatic or

minimally symptomatic metastatic castrate resistant (hormone refractory) prostate cancer

• Apligraf: manufactured by Organogenesis– Indication: treatment of surgically created gingival and

alveolar mucosal surface defects

• Azficel-T: Manufactured by Fibrocell Technologies– Indication: for improvement of the appearance of

moderate to severe nasolabial fold wrinkles in adults

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Approvals (contd.)

• HEMACORD, HPC-C: manufactured by New York Blood Center– Indication: For use in unrelated donor

hematopoietic progenitor cell transplantation procedures in conjunction with an appropriate preparative regimen for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment

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Approvals (contd.)

• Corifact: Factor XIII concentrate (Human)– Indication: prophylactic treatment of

congenital Factor XIII (FXIII) deficiency

• Anascorp: Centruroides (Scorpion) Immune F(ab’)2 (Equine) – Indication: for treatment of clinical signs of

scorpion envenomation

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Guidances• Biosimilars: 3 draft guidances published in February

2012– Scientific Considerations in Demonstrating

Biosimilarity to a Reference Product – Quality Considerations in Demonstrating

Biosimilarity to a Reference Protein Product– Biosimilars: Questions and Answers

Regarding Implementation of the Biologics Price Competition and Innovation Act of 2009

http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/TherapeuticBiologicApplications/Biosimilars/default.htm

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Guidances (contd.)

• October 2011: Guidance for Industry Clinical Considerations for Therapeutic Cancer Vaccines

• December 2011: Guidance for Industry Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm

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Collaboration with Foreign Regulators

• Parallel Scientific Advice with European Medicines Agency (EMA)– Increase the dialogue between the two agencies and

sponsors from the beginning of the lifecycle of a new product

– a deeper understanding of the bases of scientific advice

– the opportunity to optimize product development and avoid unnecessary testing replication or unnecessary diverse testing methodologies

http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/ProceduresSOPPs/ucm061218.htm

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Future Directions

• PDUFA V : CBER Rare Disease liaison within the Office of Center Director

• Continue participation in CDER’s rare disease regulatory events: education, training, workshops etc.

• To work with CDER to expand the searchable database to include CBER products

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Thank You!