Orphan Drug Production
FDA grants orphan drug designation for potential scleroderma treatment
The FDA has granted orphan drug designation to BLR-200, an investigational therapy for the treatment of scleroderma, according to a press release from manufacturer.
Orphan drug designation sought for stem cell therapy to treat frontotemporal dementia
Therapeutic Solutions International has filed with the FDA to receive orphan drug designation for its JadiCell adult stem cell therapy for treatment of frontotemporal dementia.
FDA grants orphan drug designation for Xorlo to treat autosomal dominant PKD
The FDA granted orphan drug designation for Xorlo, a drug from Xortx Therapeutics Inc. intended to treat autosomal dominant polycystic kidney disease, according to the press release.
FDA news: Fast track for glioblastoma drug, breakthrough designation for leukemia therapy
The FDA announced several regulatory actions the past few weeks.
FDA grants efzofitimod orphan drug designation for systemic sclerosis
The FDA has granted an orphan drug designation for biotherapeutics firm aTyr’s potential first-in-class immunomodulator efzofitimod in the treatment of systemic sclerosis, according to a company press release.
Ketamine treatment for status epilepticus receives orphan drug designation
FDA gives orphan drug designation to subarachnoid hemorrhage treatment
FDA fast tracks limb-girdle muscular dystrophy type 2i treatment
Paratek receives orphan drug status for Nuzyra to treat NTM lung disease
The FDA granted Paratek Pharmaceuticals orphan drug designation for Nuzyra to treat infections caused by nontuberculous mycobacteria, the company announced this week.
Court ruling in orphan drug lawsuit reframes outlook on market exclusivity
On Sept. 29, a federal judge dismissed a lawsuit from Catalyst Pharmaceuticals against the FDA and Jacobus Pharmaceuticals over the approval of a rival drug for a rare autoimmune disease — a ruling that disputes the “exclusivity” of the Orphan Drug Act.