1. 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

2. 2 Overview Products approved in 2011-2012 Other updates Hopeful Future

3. 3 Therapeutic areas Orphan Designated Products Of 70 biological products designated 28 are CBER products Total 14 grants awarded, 1 was for CBER product

4. 4 Product Approvals

5. 5 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

6. 6 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

7. 7 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

8. 8 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/HowDrugsareDev elopedandApproved/ApprovalApplications/TherapeuticBiologicApplicatio ns/Biosimilars/default.htm

9. 9 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/GuidanceComplianceRe gulatoryInformation/Guidances/default.htm

10. 10 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/GuidanceComplianceRegulatoryInf ormation/ProceduresSOPPs/ucm061218.htm

11. 11 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

12. 12 Thank You!