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On June 3, according to foreign media reports, Roche’s oral spinal muscular atrophy (SMA) drug Evrysdi was too expensive to be approved by the UK drug price regulator
.
The United Kingdom's Drug Cost and Efficacy Management Agency (NICE) has issued a draft guideline that does not recommend the use of Roche's risdiplam (marketed as Evrysdi) for patients with type 1, 2 and 3 SMA
NICE stated that SMA is a progressive neuromuscular disease that affects the spinal nerves that control movement.
It can cause muscle weakness, progressive loss of exercise capacity, and difficulty breathing and swallowing
.
If recommended, approximately 1,500 people will be eligible for the drug
Nevertheless, the agency recognizes that oral administration of Evrysdi makes it a better choice for some patients
.
Evrysdi is the third SMA drug approved in recent years, second only to Biogen's Spinraza and Novartis' gene therapy Zolgensma
NICE currently recommends Spinraza for pre-symptomatic SMA patients or patients with type 1, 2 or 3 disease
.
The cost of the drug was 450,000 pounds in the first year and 225,000 pounds in subsequent years, but NICE and Biogen signed a confidentiality discount agreement
The agency's decision may trigger further discussion
.
NICE said it will listen to opinions before June 23 and will meet again on July 13
Reference source: Roche's oral SMA med Evrysdi turned down by UK cost watchdog—despite prior nods for Zolgensma and Spinraza