Asahi Kasei Pharma & Alchemedicine launch first human trials for hard-to-treat diseases drugs

Roche’s Gazyva hits major milestone in rare kidney disease

Primary membranous nephropathy is a chronic autoimmune disease that attacks the kidneys’ filtering units

Amgen scores EU nod for UPLIZNA in rare autoimmune disease

The approval marks a major advance in targeted treatment for this rare, chronic autoimmune disease, offering twice-yearly maintenance dosing after two initial loading doses

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