Tag Archive for: metachromatic leukodystrophy

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.

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.

Despite beating analyst expectations, Takeda sustains a mid-stage defeat in a rare genetic disorder, leaving the fate of an investigational enzyme replacement therapy uncertain.