Biotech sets record price for gene therapy for rare disease
A new gene therapy for an ultrarare fatal genetic disorder will carry a list price of $4.25 million for the one-time treatment, according to its manufacturer Orchard Therapeutics, whose US headquarters is located in Boston. That makes the drug the most expensive ever approved.
The gene therapy, called Lenmeldy, won the approval of the Food and Drug Administration on Monday. It treats early-onset metachromatic leukodystrophy, also known as MLD, a devastating disease whose most serious form kills the majority of children within five years of the onset of symptoms, according to Orchard.
The disease is characterized by accumulation of fats called sulfatides, said the National Organization for Rare Disorders. This causes the destruction of the protective fatty layer surrounding the nerves in both the central and peripheral nervous systems. Children lose the ability to walk, talk, and interact with the world around them and eventually deteriorate into a vegetative state.
About 40 children are born with MLD in the United States each year. Over half of them show symptoms in the first three years of life, according to the rare disorders organization.
Lenmeldy treats the underlying genetic cause of the disease similar to other gene therapies approved for inherited conditions in recent years. Doctors take stem cells from a patient, insert a functioning copy of the defective gene that causes MLD into the cells, and then infuse them back into the patient. Before the infusion, patients must undergo high-dose chemotherapy to make room in their bone marrow for the genetically modified cells.
“Lenmeldy is truly a paradigmshifting medicine and has the potential to stop or slow the progression of this devastating childhood disease with a single treatment,” said Bobby Gaspar, Orchard’s cofounder and chief executive.
Orchard is based in London and was recently acquired by Kyowa Kirin, a Japanese pharmaceutical company, for almost $478 million. Orchard’s US headquarters is located at 101 Seaport Blvd. in Boston’s Seaport District.