An NHS trust has taken the pioneering step of applying to hold the marketing authorisation for a gene therapy for a rare disease that was abandoned by a biotech developer
Patients in the EU with Fanconi anaemia (FA) could soon have the first gene therapy option for the disorder, as the EMA starts a review of Rocket Pharma’s RP-L102 candidat
AstraZeneca’s Voydeya has become the first oral Factor D inhibitor to be approved by the FDA as a treatment for the rare disease paroxysmal nocturnal haemoglobinuria (PNH)
The March meeting of the EMA’s human medicines committee (CHMP) saw an approval recommendation for Novartis’ Fabhalta, on track to become the first oral monotherapy for ra
Orchard Therapeutics has revealed its US pricing plans for Lenmeldy, its gene therapy for metachromatic leukodystrophy (MLD), placing a $4.25 million price tag on the one-
AstraZeneca has added a late-stage rare disease therapy to its pipeline, buying France’s Amolyt Pharma and its hypoparathyroidism drug eneboparatide for $800 million upfro