News FDA clears first therapy for rare disease CTX from Mirum Mirum Pharma's Ctexli has become the first FDA-approved therapy for ultra-rare disease cerebrotendinous xanthomatosis (CTX).
News Pfizer drops Beqvez, leaving its gene therapy cupboard bare Pfizer has abandoned its haemophilia B treatment Beqvez in all world markets, saying weak demand made the business non-viable.
News Compounders cast adrift after semaglutide shortage ends The FDA has said there is no longer a shortage in the US market for Novo Nordisk's GLP-1 agonist semaglutide, ending sales of compounded forms.
News Troubled gene therapy player bluebird sold for a song At risk of defaulting on financial commitments, gene therapy firm bluebird bio has agreed to sell itself to private equity firms at a knockdown price.
News NICE backs consolidation use of Blincyto in leukaemia NICE backs NHS use of Amgen's Blincyto for the consolidation phase of treatment for some patients with acute lymphoblastic leukaemia.
News AstraZeneca builds in China as Cosette buys Mayne The reporting week has ended with a flurry of M&A as Cosette Pharma and AstraZeneca agree takeover deals.
News Dexcom is first to join FDA/CMS health tech pilot The FDA has named Dexcom as the first company to take part in its TEMPO pilot, which aims to bring digital health tech to patients more quickly.
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