Vertex Pharmaceuticals has won approval in Europe for its next-generation cystic fibrosis therapy Alyftrek (deutivacaftor/tezacaftor/vanzacaftor), shoring up the company’s dominance…
After sweeping cuts to US federal health agencies, the elimination of a committee responsible for recommending newborn screening regulations is…
Sanofi has won US Food and Drug Administration (FDA) approval for haemophilia treatment Qfitlia (fitusiran), as the company aims to…
The US Food and Drug Administration (FDA) has approved SpringWorks Therapeutics’ Gomekli (mirdametinib) to treat neurofibromatosis type 1 (NF1), a…
The US Food and Drug Administration (FDA) has accepted a new drug application (NDA) for Arrowhead Pharmaceutical’s plozasiran in the…
Arrowhead Pharmaceuticals has entered a worldwide licensing and partnership agreement with Sarepta Therapeutics to develop treatments for rare genetic diseases.…
The UK's National Health Service (NHS) is rolling out Kyowa Kirin’s Crysvita (burosumab) for the treatment of adults with a…
The European Commission (EC) has granted marketing authorisation for Pfizer's anti-tissue factor pathway inhibitor (anti-TFPI), HYMPAVZI (marstacimab), to treat prophylaxis…
When Dr Jasmin Barman-Aksӧzen began experiencing painful, unexplained burns on her skin, she turned to Wikipedia for answers. After discovering…
Ascendis Pharma has submitted a supplemental biologics licence application (sBLA) to the US Food and Drug Administration (FDA) for TransCon…
