News FDA clears first drug for MCT8 deficiency Sweden's Egetis has won approval in the US for the first-ever treatment for people living with the ultra-rare genetic disease MCT8 deficiency.
News AbbVie's Cerevel takeover delivers Parkinson's approval The first drug from AbbVie's $8.7 billion takeover of Cerevel two years ago, Parkinson's disease therapy Juvmo, has won FDA approval.
News FDA approves Mirum's oral drug for rare disease FOP Mirum's new oral therapy for rare bone disease fibrodysplasia ossificans progressiva (FOP) looks set to challenge Regeneron's intravenous Pasatru.
News EU clears first drug therapy for rare disease cALD Neuraxpharm and Minoryx' Nezglyal has become the first approved drug therapy in the EU for rare neurodegenerative disease cALD.
News Lilly's rival to Novo's new Awiqli insulin gets FDA approval The FDA has approved Eli Lilly's once-weekly basal insulin product Onswik, setting up a market tussle with Novo's recently launched Awiqli.
News Ultragenyx gets FDA nod for first Sanfilippo A gene therapy Patients in the US with Sanfilippo syndrome type A have their first FDA-approved therapy, Ultragenyx Pharma's $3.9m one-shot gene therapy Fayuvi.
News Shionogi's diversification continues with $2bn IntraBio buy Shionogi has reached a $2bn agreement to buy Anglo-US biotech IntraBio and its recently approved Aqneursa therapy for two rare genetic disorders.
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