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

The FDA has approved GRAFAPEX™ and fludarabine as a preparative regimen in acute myeloid leukemia or myelodysplastic syndrome ...

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

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

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

Coral Gables, FL-based Catalyst Pharmaceuticals , Inc. (NASDAQ:CPRX), a $2.69 billion market cap biopharmaceutical company that has delivered an impressive 54% return to shareholders over the past ...

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.