Topic: orphan drug status
505(b)(2) drug development by definition utilizes publicly available data sources to support approval.
Supernus makes a return to epilepsy R&D, buying the developer of a traditional Chinese medicine-derived drug for $15 million upfront.
Here's Wednesday's prescription for your biopharma news of note.
Clementia Pharmaceuticals has filed to raise $115 million in an IPO.
The FDA is working on a medical innovation development plan to lower healthcare costs by streamlining the path to market for targeted therapies.
The European Medicines Agency (EMA) has added Alnylam’s acute hepatic porphyria candidate, givosiran, to its priority medicine (PRIME) scheme.
TG Therapeutics got orphan drug designation from the FDA for both its clinical candidates this week.
Gene therapy player AveXis has gotten a Breakthrough Therapy Designation for its only clinical candidate, AVXS-101 to treat spinal muscular atrophy Type 1.
Savara has acquired the rare respiratory disease assets of Serendex Pharmaceuticals for an undisclosed amount of stock upfront—with additional cash milestone payments upon approval of the lead assets.