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

Nxera secures exclusive APAC rights to DMD drug Vamorolone in deal with Santhera

Nxera will take full responsibility for regulatory approvals, clinical development where required, manufacturing and commercialization in the licensed territories

Italfarmaco and JCR Pharmaceuticals partner to bring Duchenne therapy to Japan

The partnership also sets the stage for broader collaboration on rare disease treatments

SN Bioscience’s SNB-101 wins FDA orphan drug designation for gastric cancer

The drug, currently in a phase 1b/2 clinical trial for small cell lung cancer, is a polymer nanoparticle formulation of SN-38

WHO report reveals stark global inequities in genomic research

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

Ipsen’s FALKON trial falls short in rare FOP study, offers key insights

FOP is a rare genetic disorder caused by mutations in the ALK2 kinase

Pharma overhaul: EMA hails “Once-in-a-Generation” reform

The reform is set to modernise how medicines are developed, authorised, and made available across the EU

Sanofi secures two breakthrough nods in China for rare blood disorders

Qfitlia is the first antithrombin (AT)-lowering therapy approved for routine prophylaxis in hemophilia

Debiopharm launches pivotal phase III trial for novel quarterly acromegaly treatment

Acromegaly, a rare chronic endocrine disorder caused by excess growth hormone, is seeing promising advances in treatment development