News Uplizna is first drug FDA-cleared for rare disease IgG4-RD Amgen has bagged a second indication for its B cell-depleting therapy Uplizna after the FDA cleared it for rare inflammatory disease IgG4-RD.
News FDA clears first treatment for Prader-Willi syndrome Soleno Therapeutics' Vykat XR has become the first FDA-approved treatment for excessive eating in rare disorder Prader-Willi syndrome.
News Fabhalta is first FDA-approved therapy for rare disease C3G Novartis' oral complement inhibitor Fabhalta has become the first approved treatment for rare kidney disease C3G as it chases a $3bn sales target.
News Third indication beckons for Amgen's Uplizna Amgen is preparing to file a third indication for Uplizna, as a twice-yearly therapy for generalised myasthenia gravis, following new phase 3 data.
News Spain agrees to reimburse Orchard's gene therapy Libmeldy Spain has agreed to reimburse Orchard Therapeutics' ex vivo gene therapy for the rare childhood disease metachromatic leukodystrophy.
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 GSK adds EU to Blenrep checklist as FDA sets new PDUFA date GSK has added the EMA to the list of regulatory bodies approving Blenrep for multiple myeloma, but whether the FDA is next is still an open question.
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