Zydus receives USFDA ODD for Desidustat for the treatment of sickle cell disease

This Orphan Drug Designation from the USFDA underlines the urgent medical need to develop a therapy for sickle cell disease

Alembic Pharmaceuticals gets USFDA nod for generic Parkinson's disease treatment tablets

The approved ANDA is therapeutically equivalent to the reference listed drug product (RLD), Stalevo Tablets

GSK’s Nucala receives European Commission approval to treat chronic obstructive pulmonary disease

Nucala is the first and only monthly biologic in the EU evaluated in a wide COPD population with an eosinophilic phenotype

Sanofi’s Venglustat shows breakthrough results in rare neurological Gaucher disease

Venglustat, a glucosylceramide synthase inhibitor (GCSi), works by reducing the abnormal buildup of sugar-and-fat molecules in cells and organs

Moderna teams up with Recordati to commercialize rare disease therapy

Will Forte joins Teva Pharmaceuticals to shine spotlight on Huntington’s disease

HD causes cognitive, behavioral, and physical challenges, including involuntary movements known as HD chorea, which can disrupt daily life

Bausch Health’s RED-C phase 3 trials fall short, company committed to liver disease research

The trials, which enrolled over 1,000 patients across 398 sites in 17 countries, showed that the drug was safe and well-tolerated—but ultimately failed to deliver the hoped-for clinical results

EU green light to IntraBio’s AQNEURSA for rare neurological disease Niemann-Pick Type C

The approval is grounded in data from a Phase III randomized, double-blind, placebo-controlled, crossover study in NPC patients

FDA nod to Boehringer Ingelheim's heart disease treatment for dogs 'Vetmedin'

VETMEDIN (pimobendan) chewable tablets and VETMEDIN solution (pimobendan oral solution) can delay the onset of congestive heart failure (CHF) in dogs

FDA nod to first-ever treatment for fatal pediatric Menkes disease