A medication used to treat a very rare condition, metachromatic leukodystrophy (MLD) is now believed to be the most expensive drug. Gene therapy, Libmeldy (atidarsagene autotemcel), also called OTL-200, is a one-off treatment and has a price of about £2.8 million (GBP).
MLD is a very rare hereditary condition resulting from a mutation in the gene needed to make arylsulfatase A (ARSA). ARSA breaks down fats, called sulfatides. This will cause the accumulation of sulfatides leading to the destruction of the protective fatty layers of the peripheral and central nervous systems.
The disease has three types – late-infantile MLD (symptoms appears in the first 3 years of life), juvenile MLD (less common, mostly from 4 to 12 or 14 years), and adult MLD. MLD affects one in 100,000 births.
The disease is progressive, and the symptoms are seizures, difficulty walking, personality changes, behavioural changes, speech difficulty. In the late stage, other symptoms such as blindness, unresponsiveness, and inability to work can occur.
About Libmeldy NHS deal
In the UK, the NHS negotiated a deal that will see a confidential discount for libmeldy to help parents of children with early-onset MLD.
This is coming after the National Institute of Health Care Excellence (NICE) rejected the drug price last year due to the exorbitant price, and doubt about the long-term effect. The long term effect of the drug is not yet known, though medical experts are hoping the drug will result in a permanent cure for the disease.
Libmeldy, made by Orchard Therapeutics, in partnership with San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. It has been approved by the European Medicines Agency, but is still under investigation by the U.S. Food and Drug Administration.
It is an ‘orphan medicine’ since it is used for a rare disease, and it might not result in profit for the pharmaceutical company except there is a government subvention.
Five European sites will administer the drug. Two of the sites – Tübingen University Hospital, in Germany, Hôpital Debré in Paris, have already treated two patients. Another patient from the Middle East is scheduled to be treated at the Ospedale San Raffaele in Milan, Italy. In England, a specialist service at the Centre of Genomic Medicine at Saint Mary’s Hospital, Manchester will administer the drug.