3 Days, 4 Orphan Drug Approvals, and 1 Conference

James Radke, PhD

RDR was busy

The end of last week was a very busy time for the rare disease community. We at Rare Disease Report attended  NORD's Annual Rare Diseases and Orphan Products Breakthrough Summit where we met and interviewed so many wonderful people who are working to together to make management of orphan diseases better.
We talked with patients, advocates, and leaders in the pharmaceutical industry as well as top officials at the FDA. We even interviewed a pilot coordinator. Be sure to see our growing list of interviews at

The FDA was busier

While we at Rare Disease Report met with leaders in the rare disease space, dined at Morton’s, and stayed at a 4 star hotel, the FDA was very busy approving an astounding 4 orphan drugs in 3 days.
The drugs were:
Stensiq (asfotase alfa) for the treatment for perinatal, infantile and juvenile-onset hypophosphatasia (HPP). It will be marketed by Alexion Pharmaceuticals.
Yondelis (trabectedin) for the treatment of specific soft tissue sarcomas  – unresectable or metastatic liposarcoma and leiomyosarcoma. It will be marketed by Janssen Pharmaceuticals.
Onivyde (irinotecan liposome injection), in combination with fluorouracil and leucovorin for the treatment of patients with advanced (metastatic) pancreatic cancer who have been previously treated with gemcitabine-based chemotherapy. It will be marketed byMerrimack Pharmaceuticals.
Coagadex (factor X coagulant) for the treatment of adults and children (aged 12 years and over) with hereditary factor X deficiency for on-demand treatment and control of bleeding episodes, and perioperative management of bleeding in patients with mild hereditary Factor X deficiency. It will be marketed by Bio Products Laboratory.
And while it seems that all drugs being reviewed by the FDA get approved, the agency let us know that is not always the case. Last week, the FDA issued a Complete Response letter to Spectrum Pharmaceuticals in regard to their NDA submission for Evomela (propylene glycol (PG)-free IV formulation of melphalan)
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