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

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

Biohaven disappointed as rare disease therapy faces FDA setback 'despite promising results'

The FDA setback highlights ongoing tensions between patient needs, regulatory processes, and the use of real-world evidence in evaluating treatments for rare diseases

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

India invites applications under PRIP scheme to drive pharma-medtech innovation

Early stage projects of up to Rs. 9 crore eligible for up to Rs. 5 crore assistance and later stage projects of up to Rs. 285 crore eligible for up to Rs. 100 crore assistance

Moderna opens manufacturing and R&D facility in UK

The Moderna Innovation and Technology Centre in Harwell, Oxfordshire is now fully operational and Moderna is licensed to supply British-made COVID-19 vaccines to the UK population