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 Can Dupixent plug treatment gap in bullous pemphigoid? The FDA is scheduled to deliver a verdict on Sanofi and Regeneron's Dupixent for rare autoimmune skin disorder bullous pemphigoid in June.
News EU approves CSL's once-monthly HAE drug Andembry The EU has approved CSL's once-monthly Andembry for hereditary angioedema, challenging therapies from Takeda and Biocryst.
News MIT team develops 'years-long' drug delivery tech Researchers in the US have devised a new depot system that could provide months or even years of delivery from a single subcutaneous injection.
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