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

FDA nod to Koselugo for adults with Rare NF1 tumors, expanding life-changing treatment

Merck KGaA Q3 2025 net sales remains flat amid geopolitical uncertainty

FDA grants Orphan Drug Designation for Tinostamustine in malignant glioma

Tinostamustine is an investigational drug with a potential first-in-class mechanism combining bifunctional alkylating activity and pan histone deacetylase (HDAC) inhibition

Zydus receives FDA’s ODD for Desidustat for the treatment of beta-thalassemia

Orphan drug designation (ODD) by the USFDA for Desidustat, provides eligibility for a potential seven-year marketing exclusivity subject to the USFDA approval