rare diseases
Regeneron’s Veopoz scores FDA approval for rare immune disease
Regeneron Pharmaceuticals has received US Food and Drug Administration (FDA) approvals for two of its drugs, namely Veopoz (pozelimab-bbfg) and…
Amicus gears up to launch Pompe disease combination therapy in the UK
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved Amicus Therapeutics’ combination treatment of Pombiliti (cipaglucosidase alfa) and…
Galecto terminates development for its lead candidate
Galecto has axed the development of its lead pipeline candidate, GB0139, after announcing that the Phase IIb trial for the…
PTC Therapeutics to commercialise Akcea’s two rare disease drugs
Ionis Pharmaceuticals affiliate Akcea Therapeutics has signed an agreement with biopharmaceutical company PTC Therapeutics for the commercialisation of its rare…
BioNTech and Genevant Sciences to develop rare disease drugs
German biopharmaceutical firm BioNTech has partnered with Genevant Sciences, a genetic disorder therapeutics maker, to develop mRNA therapy programmes, which…
