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

The goal is to treat familial dysautonomia patients with a single injection.

Zydus has announced that the USFDA has granted Orphan Drug Designation (ODD) to Usnoflast, a novel oral NLRP3 inhibitor, for ...

Zai Lab Limited (ZLAB) announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation or ODD to ZL-1310, a ...

The NPI-001 (N-acetylcysteine amide) tablets are a proprietary investigational therapy for the treatment of patients with ...

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

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 ...

Zydus Lifesciences announced that the USFDA granted Orphan Drug Designation to Usnoflast, a novel oral NLRP3 inhibitor, for ...

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Zai Lab’s delta-like ligand 3 (DLL3) antibody-drug conjugate (ADC), ZL-1310, for small cell lung cancer (SCLC).

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

The HAELO trial will assess the therapy’s safety and efficacy in 60 adult subjects with Type I or Type II HAE.