The U.S. Food and Drug Administration has approved Orchard Therapeutics’ Lenmeldy (atidarsagene autotemcel) as the first gene therapy for the treatment of children with metachromatic leukodystrophy (MLD). Lenmeldy is a one-time, individualized single-dose...
Kyowa Kirin’s bet on Orchard Therapeutics has paid off, furnishing the Japanese drugmaker with a newly approved gene therapy in the U.S. that’s already started to prove itself in Europe over the past several years. The FDA on Monday gave a thumbs-up to Orchard’s...
The FDA approved the first nonsteroidal drug to treat all genetic variants of Duchenne muscular dystrophy, clearing drugmaker Italfarmaco’s oral treatment for patients age 6 and older. The drug, givinostat, will be sold as Duvyzat, the FDA said in a press release...
Orchard Therapeutics on Wednesday revealed details regarding the U.S. commercial launch of the newly approved Lenmeldy (atidarsagene autotemcel), giving the one-time gene therapy for metachromatic leukodystrophy a wholesale acquisition cost of $4.25 million. To...
The risks of early death in the trials of Johnson & Johnson and Legend Biotech’s Carvykti and Bristol Myers Squibb and 2seventyBio’s Abcema were not a significant concern for the FDA’s Oncologic Drugs Advisory Committee, which voted 11-0 and 8-3 respectively...
