NEW YORK, May 15 — England's National Health Service said it had reached a deal to pay for US drugmaker Biogen Inc's expensive spinal muscle atrophy treatment Spinraza.
NHS England said it reached a “managed access agreement” with Biogen under which it will fund treatment for a limited time and collect data on the drug's effectiveness.
NHS England did not disclose the price it will pay for the drug.
Spinraza, which is administered by spinal injection every four months, has a US list price of US$750,000 (RM3.1 million) for the first year and US$375,000 annually thereafter.
Britain's healthcare cost agency, NICE, said in August it could not recommend Spinraza as a cost
effective treatment. The rejection came despite a lower British price tag of £450,000 (RM2.44 million) for the first year and Biogen offering an undisclosed discount to the National Health Service.
Spinal muscular atrophy (SMA) is the leading genetic cause of infant mortality, affecting one in every 10,000 live births. About 60 per cent of patients have the most severe Type 1 form of the disease, which often leads to paralysis, impaired breathing and death by the second birthday.
Between 600 and 1,200 children and adults are currently living with the condition in England and Wales, according to NHS England. Spinraza will be made available immediately to Type 1 patients by Biogen, it said.
It said that Spinraza will be made available for other patients — including adults and siblings who have yet to show symptoms — in a few weeks, after NICE publishes guidance.
Spinraza was the first approved treatment for SMA, but is expected to soon face new competition. The US Food and Drug Administration is due to decide on approval for Novartis' gene therapy Zolgensma this month, with other regulators expected to follow suit later this year.
Novartis says the one-time treatment could be a cure for SMA and is pushing for a price in the range of US$1.5 million to US$5 million. That would make Zolgensma the most expensive new therapy. — Reuters
