Federal Circuit Upholds Pfizer's Victory in Gene Therapy Patent Dispute for Hemophilia Treatment
The Federal Circuit has affirmed the Patent Trial and Appeal Board's decision invalidating two patents covering experimental gene therapy for hemophilia treatment, handing Pfizer a significant legal victory.
NICE Extends Access to Brineura for CLN2 Batten Disease While Seeking Long-term Solution
NICE has secured continued access to cerliponase alfa (Brineura) for current patients and those starting treatment before December 2025, despite not recommending it for routine NHS use due to cost concerns.
BioMarin Acquires Inozyme Pharma for $270 Million, Adding Late-Stage Enzyme Therapy for Rare Genetic Disorder
BioMarin Pharmaceutical has entered into a definitive agreement to acquire Inozyme Pharma for $270 million, strengthening its enzyme therapy portfolio with the addition of INZ-701.
Takeda Spotlights Three Late-Stage Readouts as Potential "Inflection Point" Amid Vyvanse Generic Competition
Takeda Pharmaceutical is positioning three upcoming late-stage clinical trial readouts as a strategic "inflection point" to offset revenue losses from Vyvanse's generic competition.
BioMarin's Palynziq Shows Promising Results in Adolescents with PKU, Setting Stage for Label Expansion
BioMarin's Phase 3 PEGASUS trial demonstrated that Palynziq significantly lowered blood phenylalanine levels in adolescents aged 12-17 with phenylketonuria compared to diet management alone.
BioMarin's VOXZOGO Shows Promising Results in Reducing Tibial Bowing in Children with Achondroplasia
New clinical data reveals VOXZOGO (vosoritide) significantly reduces tibial bowing in children with achondroplasia, addressing a common cause of pain and functional impairment that often requires surgical intervention.
Pfizer Discontinues Beqvez Gene Therapy for Hemophilia B Amid Low Market Demand
Pfizer has announced the cessation of development and commercialization of Beqvez, its FDA-approved gene therapy for hemophilia B, citing limited interest from patients and healthcare providers.
FDA Requests Additional Phase 3 Trial for Santhera's DMD Drug Raxone, Delaying Approval
The FDA has requested Santhera Pharmaceuticals to conduct a second Phase 3 trial for Raxone (idebenone) in Duchenne muscular dystrophy treatment, potentially delaying approval until 2020.
Synlogic's Novel Probiotic Therapy Shows Promise in Phase 2 PKU Trial, Advancing to Phase 3
Synlogic's engineered probiotic SYNB1934 demonstrated a significant 34% reduction in phenylalanine levels in phenylketonuria patients during Phase 2 trials, outperforming its predecessor SYNB1618.
European Commission Approves BioMarin's Roctavian for Severe Hemophilia A
The European Commission granted conditional marketing approval for BioMarin's Roctavian (valoctocogene roxaparvovec) for severe hemophilia A in adults without Factor VIII inhibitors.