Orchard follows buyout with FDA approval of rare disease gene therapy
The decision makes available in the U.S. a gene therapy GSK first began working on more than a decade ago. Orchard Therapeutics on Monday won Food and Drug Administration approval for the first gene therapy to treat a deadly and devastating rare disease in children called metachromatic leukodystrophy.
The condition is caused by a faulty gene that robs the body of the ability to make enough of a crucial enzyme called arylsulfatase A. It most often strikes infants between the ages of 6 months and 2 years, resulting in nerve damage that affects motor and cognitive function. The disease kills about half of children with the “late infantile” form within five years of onset.
Orchard, recently acquired by Japanese drugmaker Kyowa Kirin, will sell the treatment in the U.S. under the brand name Lenmeldy. The therapy is already available in Europe, where it’s known as Libmeldy.
For Kyowa Kirin, the approval offers a measure of validation for its Orchard acquisition, which closed in January. But access to the world’s biggest pharmaceutical market is only part of the battle when it comes to selling gene therapies.
Founded in 2015, Orchard acquired GSK’s gene therapy portfolio in 2018 and instantly became one of the few companies with a marketed gene therapy, Strimvelis. The company then went public in a sizable IPO, raising $200 million. The next year, Orchard’s shares traded at $190.
But faced with the complications inherent in selling costly and difficult-to-make medications, the company abandoned plans to develop several therapies and stopped investing in Strimvelis, which had only been given to 16 people. It restructured twice.
Finally in December, it agreed to a takeover by Kyowa Kirin for $16 per American Depositary Share, just above its $14 debut price in 2018. The purchase price included an additional $1 per share payout that was tied to by FDA approval of Lenmeldy.
Kyowa Kirin and Orchard are now looking to the U.S. market for a boost. Orchard won European Commission approval for Libmeldy in December 2020 and has slowly won reimbursement from payers for the costly therapy. Still, third-quarter sales of the treatment reached just $5.6 million.
The FDA cleared Lenmeldy for presymptomatic late infantile patients as well as those considered “early juvenile” who either have early symptoms or haven’t yet displayed any. A company spokesperson said U.S. pricing and launch details will be announced later this week. The Institute for Clinical and Economic Review says the product would be cost-effective at a price of $2.3 million to $3.9 million a patient.
For families coping with the devastating condition, the FDA approval offers new hope. In studies, Orchard found that its treatment could stop or slow disease progression and has up to 12 years of follow-up on patients.
In its release on Monday, the FDA highlighted data for pre-symptomatic late infantile patients. All of those treated with Lenmeldy in Orchard’s studies were still alive at age six, compared with the 58% survival rate historically seen with untreated children. In the treatment group, 71% of patients were able to walk without help at age 5 and 85% had normal language and performance IQ scores.
Lenmeldy is one of the drugs Orchard acquired in its GSK portfolio purchase. As part of that agreement, Orchard is transferring a priority review voucher it won from the FDA approval — a tool that can speed up a drug’s evaluation — to GSK.
March 20, 2024
