This week we have news on pediatric influenza vaccine guidance; Moderna submit data for their COVID-19 booster dose; There’s new FDA warnings for some JAK inhibitors; Trudhesa nasal spray is approved; And the FDA places a clinical hold on a gene therapy for phenylketonuria.
BMN 307 is an investigational AAV5-phenylalanine hydroxylase gene therapy designed to normalize phenylalanine concentration levels.
VS-01 is a novel liposomal-based intraperitoneal fluid that acts as a clearance enhancer of toxic metabolites, in particular ammonia, from the body.
Krabbe disease is a rare inherited disorder caused by mutations in the GALC gene.
Mitapivat is a first-in-class, oral, selective small molecule allosteric activator of pyruvate kinase-R enzymes.
EDG-5506 is a selective, small molecule myosin inhibitor designed to protect injury-susceptible fast skeletal muscle fibers.
VTX-801 is an investigational, novel rAAV-based gene therapy that delivers a shortened version of the ATP7B transgene.
The Food and Drug Administration (FDA) has approved Nexviazyme (avalglucosidase alfa-ngpt), a hydrolytic lysosomal glycogen-specific enzyme, for the treatment of late-onset Pompe disease in patients 1 year of age and older. Pompe disease is a rare, degenerative muscle disorder caused by mutations in the acid alpha-glucosidase (GAA) gene, which leads to the abnormal buildup of…
Tesomet is an investigational fixed-dose combination therapy of tesofensine, a triple monoamine reuptake inhibitor, and metoprolol, a beta-1 selective blocker.
Bylvay is an oral reversible inhibitor of the ileal bile acid transporter.
