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.

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

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 ...

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

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).

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 ...

Nuvalent, Inc. shares have fallen approximately 30% after promising updates on its two lead NSCLC candidates rallied the ...

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

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

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