Switzerland-based Idorsia has launched a new pivotal Phase 3 trial of lucerastat in Fabry disease, targeting kidney deposits after its first late-stage study failed to reduce nerve pain. The company also signed a development and commercialization agreement with Japan’s Nxera Pharma, which will help fund the program.
Guido Oelkers will leave Swedish Orphan Biovitrum to become BioNTech’s CEO by Feb. 1, 2027, succeeding co-founder Uğur Şahin after overseeing a nine-year transformation that increased Sobi’s revenue more than fivefold.
French pharma giant Servier is spending US$2.5bn to snap up Day One Biopharmaceuticals, gaining a key approved drug for difficult childhood brain tumours along with several promising pipeline candidates.
French biopharma THX Pharma has signed a strategic licensing deal with Biocodex, an independent French pharmaceutical group, to advance two drug candidates in three rare genetic disorders with high unmet need.
Rare disease specialist SynaptixBio Ltd has got its second FDA orphan drug designation for a subtype of TUBB4A leukodystrophy.
Swiss rare disease specialist VectivBio Holding AG is taking over US biotech Comet Therapeutics Inc. for an undisclosed amount. Comet focusses on treatments for metabolic diseases.
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