Apellis Pharma and partner Sobi have won EU approval for their paroxysmal nocturnal haemoglobinuria (PNH) drug pegcetacoplan in Europe, with a more restricted label than in the US.
Acadia Pharma's trofinetide has hit its efficacy targets in a phase 3 trial involving girls with the devastating neurological disorder Rett syndrome, although safety has dampened enthusiasm
Novartis has chalked up another successful trial for its targeted factor B inhibitor iptacopan, an orally-active drug trying to challenge antibody-based therapies for diseases associated wi
Sanofi and Regeneron's Dupixent has become the first biologic drug to show activity in prurigo nodularis (PN), raising the hope of a new treatment option for the rare and highly debilitatin
UK cost-effectiveness watchdog NICE is set to recommend NHS use of Alnylam's gene-silencing therapy Givlaari in England and Wales for the rare disease acute hepatic porphyria (AHP), after i
18 months after saying "no" to regular NHS funding for Sanofi and Regeneron's Dupixent (dupilumab) for severe asthma in initial guidance, NICE has now backed the drug.