Upcoming FDA Dates for Rare Disease and Orphan Drugs

June 17:  PDUFA date for Johnson & Johnson’s DARZALEX (Daratumumab) + pomalidomide and dexamethasone for Relapsed or refractory multiple myeloma

June 24:  Preliminary data due at European Hematology Association (Phase 2a initiated June 2016) for Global Blood Therapeutics’ GBT440 for Sickle cell disease in adolescents

June 26:  PDUFA date for Halozyme’s Subcutaneous rituximab for various blood cancers

July 8-13: Data due on Biomarin’s Phase 1/2 study of BMN 270 for Hemophilia A at International Society of Thrombosis and Haemostasis (ISTH) 2017 Congress. Phase 2b trial to be initiated 3Q 2017.

July 8-13: Updated data due on UniQure’s Phase 1 / 2 study of AMT-060 for Hemophilia B to be presented at International Society of Thrombosis and Hemostasis (ISTH) meeting.

July 12: Advisory Committee Meeting (under priority review announced March 29, 2017) for Novartis’ CTL019 for relapsed/Refractory B-Cell Acute lymphoblastic leukemia. Assume 6-month review so that PDUFA would be September 29, 2017.

By Jul 31: Phase 2 data due on Bellerophon Therapeutic’s INOpulse delivery device for Pulmonary Hypertension associated with Idiopathic Pulmonary Fibrosis (PH-IPF)

Aug 10: PDUFA date for Dynavax’s HEPLISAV-B for Hepatitis B (Advisory Committee Meeting July 28, 2017)