U.S. FDA Orphan Drug Designations Q3 2019

October 2, 2019

The list below reflects U.S. Food and Drug Administration Orphan Drug designations announced during the third quarter of 2019.

FDA Advisory Committee Recommends Approval of Elamipretide for Treatment of Barth Syndrome

Rare Daily Staff The U.S. Food and Drug Administration Cardiovascular and Renal Drugs Advisory Committee voted 10 […]

Every Cure, the non-profit biotech using artificial intelligence to repurpose drugs, has received a five-year $60 million […]

Rare Daily Staff Purespring Therapeutics said it raised $105 million in a series B financing to advance […]

Allison Freedman was an avid hiker, mother of young twins, and had just completed an MBA when […]

Rare Daily Staff Astellas Pharma has secured an exclusive option and license agreement for AVB-101, AviadoBio’s experimental […]

Rare Daily Staff Judo Bio, which is developing oligonucleotide medicines delivered to the kidney, said it raised […]

Rare Daily Staff Genomics England has started to test hundreds of babies for more than 200 rare […]

Despite advancements in genetic testing, people with rare diseases often face a prolonged diagnostic odyssey involving multiple […]

Rare Daily Staff CAMP4 Therapeutics said it entered into a research collaboration with BioMarin Pharmaceutical to advance […]