ImCheck’s ICT01 Receives FDA Orphan Drug Designation for Treatment of Acute Myeloid Leukemia Clinical data showing ...

SH-110, a liquid cancer treatment for glioma, earned orphan drug status from the FDA, helping patients who struggle to ...

The GOP tax bill repeals the Inflation Reduction Act’s price controls for drugs for rare diseases.

STOCKHOLM, SE / ACCESS Newswire / July 15, 2025 / AlzeCure Pharma (STO:ALZCUR) (FRA:AC6) AlzeCure Pharma AB (publ) (FN ...

The FDA designates SH-110 as an orphan drug, offering a safer oral treatment option for glioma patients with swallowing ...

Amphix Bio LLC has been granted U.S. FDA orphan drug designation for its lead candidate AMFX-200 for the treatment of acute spinal cord injury (SCI). AMFX-200 is an FGFR (fibroblast growth factor ...

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral Sclerosis (ALS) developed at the Universitat Autònoma de Barcelona and ...

KLTO‑202 is an experimental gene therapy that would deliver the instructions for a protein that may reduce inflammation and ...

President Donald Trump’s One Big Beautiful Bill, signed into law last week, reintroduces broader exemptions for orphan drugs ...

FDA designates MB-101 CAR T-cell therapy for glioblastoma, highlighting its potential in treating aggressive brain tumors ...