Soligenix, Inc., a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases and areas of unmet medical need, has a portfolio of products aimed at ...

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, ...

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 ...

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

From Adrenoleukodystrophy to Zollinger Ellison Syndrome, there are currently over 7,000 diseases identified as rare. In the U.S., the Orphan Drug Act of 1983 defines a disease as “rare” if it affects ...

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

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 ...

The Bruton tyrosine kinase inhibitor was approved in combination with bendamustine and rituximab in previously untreated ...

The Centers for Medicare and Medicaid Services (CMS) has selected 15 more drugs for Medicare price negotiation, announcing ...

Archimed, KKR and Novo Holdings are among the PE firms investing in pharma research, development and sales for genetic medicine.

The FDA has narrowly beaten the European Medicines Agency (EMA) to the most approved drugs with a new active substance in ...