Tag Archive for: Orchard Therapeutics

Orchard Therapeutics, a unit of Japan’s Kyowa Kirin , priced its newly approved gene therapy for a rare, life-threatening genetic disease on Wednesday at $4.25 million, making it the most expensive drug in the United States.

Orchard Therapeutics secured the FDA’s first approval for an autologous gene therapy to treat the rare metabolic disease metachromatic leukodystrophy in children.

The FDA’s busy March continues with six target action dates remaining on the calendar. Over the next two weeks, the regulator will decide on investigational therapies for Duchenne muscular dystrophy, chronic kidney disease anemia, a rare metabolic disorder and more.

On the potential cusp of FDA approval for its rare disease gene therapy, Orchard Therapeutics is being purchased by Japanese pharma Kyowa Kirin for close to $478 million if all conditions are met, the companies announced on Thursday. 

Nearly three years after European approval, the U.S. regulator has accepted Orchard Therapeutics’ BLA for its gene therapy OTL-200, being proposed for metachromatic leukodystrophy.