US House passes Bill reauthorizing FDA rare disease incentive program

Chugai’s Tecentriq gets green light in Japan for hard-to-treat Thymic Carcinoma

The drug had previously received orphan drug designation for this rare cancer on March 31, 2025

EU nod to Wayrilz as breakthrough treatment for hard-to-treat ITP

Wayrilz represents a new approach to ITP by targeting the disease at its root through multi-immune modulation

Ambros Therapeutics launches with $125 million to advance breakthrough therapy for rare pain disorder

The funding will support the pivotal Phase 3 clinical trial of neridronate in CRPS-1

Praxis Precision hits milestone in fight against rare paediatric seizures

The EMBOLD study evaluated relutrigine for patients with SCN2A and SCN8A developmental and epileptic encephalopathies

Bayer advances fight against rare kidney disease with new experimental drug

Alport Syndrome is caused by genetic mutations affecting type IV collagen in the kidneys, ears, and eyes

Belite Bio reports landmark phase 3 success for Stargardt Disease therapy

Seeing well-controlled Phase 3 data that shows a marked slowing of lesion growth in Stargardt disease is deeply encouraging

Merck KGaA’s Cladribine capsules gain FDA fast track for rare neuromuscular disorder

FDA grants accelerated approval to sevabertinib for non-squamous non-small cell lung cancer

HER2-mutant NSCLC typically has a poor prognosis and limited treatment options

Cardiol Therapeutics wins US patent protection for heart-disease drug platform

The patent will lock in broad protection for the company’s lead therapy CardiolRx and its novel subcutaneous candidate CRD-38