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

EU approves Alexion’s Koselugo to treat adults with neurofibromatosis type 1 tumours

Approval based on pivotal KOMET Phase III trial demonstrating significant tumour reduction in adults with NF1

Alexion highlights breakthrough C5 inhibition advances in gMG at 2025 AANEM and MGFA scientific sessions

New Phase III data on gefurulimab highlight potential as a self-administered treatment option; real-world evidence reinforces clinical benefits of Ultomiris and Soliris

Sanofi's Q3 profit dips marginally as Dupixent sales top €4 billion

The company's net income attributable to equity holders dropped to €2.80 billion from €2.82 billion last year

Novo Nordisk acquires Omeros’ MASP-3 inhibitor Zaltenibart for up to US$ 340 million

Zaltenibart offers best-in-class potential across multiple rare blood and kidney disorders, enhancing Novo Nordisk’s rare disease portfolio