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

Datavant partners with Indegene to power clinical trial recruitment

Collaboration matches more eligible patients, including those with rare and complex diseases, to appropriate clinical trials

FDA approves Sanofi’s BTK inhibitor for immune thrombocytopenia

ITP is a disease of complex immune dysregulation leading to low platelet counts, bleeding, and reduced quality of life

Sarepta advances siRNA collaboration and sale of arrowhead equity investment

Sarepta has also entered into an agreement with Arrowhead to transfer 2,660,989 shares of Arrowhead common stock in satisfaction of $50 million of the company’s previously announced $100 million milestone payment obligation

Sanofi’s GPRC5D monoclonal antibody earns ODD in US for multiple myeloma

ImCheck’s ICT01 receives FDA ODD for treatment of acute myeloid leukemia

Clinical data showing unprecedented remission rates in newly diagnosed AML patients support advancing ICT01 into pivotal trials