Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and ...

Zydus Lifesciences announced that Usnoflast, a treatment for amyotrophic lateral sclerosis (ALS), has received Orphan Drug Designation from the US FDA. This designation supports the development of ...

CAMBRIDGE, Mass. & SALISBURY, England - KalVista Pharmaceuticals, Inc. (NASDAQ: NASDAQ:KALV) has received Orphan Drug ...

Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and ...

Medexus will target a commercial launch in 1H CY2025; potential for annual product-level revenue to exceed US$100 million within five years after commercial launchToronto, Ontario and Chicago, ...

KalVista Pharmaceuticals, Inc. (KALV) announced Tuesday that Japan's Ministry of Health, Labour and Welfare (MHLW) has granted ...

KalVista (KALV) announced that Japan’s Ministry of Health, Labour and Welfare, or MHLW, has granted sebetralstat orphan drug designation. The ...

R289 has gained orphan drug status from the FDA for the treatment of patients with myelodysplastic syndromes. The FDA has ...

Mo Qatanani, the Chief Scientific Officer of Scholar Rock Holding Corp (NASDAQ:SRRK), executed a sale of 7,347 shares of common stock, according to a recent SEC filing. The shares were sold at an ...

The Orphan Drug Designation provides Numiera with a number of incentives, including seven years of post-approval market exclusivity, tax credits, and fee waivers. This designation significantly ...