Orchard Therapeutics outlines U.S. launch plans for Lenmeldy

Orchard Therapeutics (ORTX), recently acquired by Kyowa Kirin (KYKOF) with the goal of accelerating the delivery of new gene therapies to patients around the globe, announced the details of its U.S. commercial launch of Lenmeldy, formerly known as OTL-200, the first FDA-approved therapy for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile-collectively referred to as early-onset-metachromatic leukodystrophy. Lenmeldy aims to correct the underlying genetic cause of MLD by inserting one or more functional copies of the human ARSA gene ex vivo into the genome of a patient’s own hematopoietic stem cells using a lentiviral vector. “Lenmeldy is truly a paradigm-shifting medicine and has the potential to stop or slow the progression of this devastating childhood disease with a single treatment, particularly when administered prior to the onset of symptoms,” said Bobby Gaspar, M.D., Ph.D., co-founder and chief executive officer of Orchard Therapeutics. “We are committed to enabling broad, expedient and sustainable access to this important therapy for eligible patients with early-onset MLD in the U.S.”