Savara resubmits FDA application for potential treatment in rare lung disease patients

The resubmission of BLA for MOLBREEVI names Fujifilm as the drug substance manufacturer

Diamyd Medical accelerates Phase 3 Type 1 diabetes trial following FDA guidance

The interim efficacy readout, involving around 170 participants with 15-month data, remains on track for the end of March 2026

CPHI & PMEC India 2025 ignites pharma innovation and partnerships

For 2026 onwards, CPHI and PMEC India are evolving into two distinct, specialized shows

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