For now, Roche’s oral spinal muscular atrophy (SMA) drug is too costly to gain the go ahead from England’s drug-price watchdogs.
The National Institutes for Health and Care Excellence (NICE) published draft guidance on Wednesday that recommends against the use of Roche’s risdiplam, marketed as Evrysdi, for people with types 1, 2 and 3 SMA.
SMA is a progressive neuromuscular condition affecting the nerves in the spinal cord controlling movement, leading to muscle weakness, progressive loss of movement and difficulty breathing and swallowing, NICE says. If recommended, roughly 1,500 people would’ve been eligible for the medicine.
Evrysdi’s ability to help patients wasn’t the problem. In turning the drug away, the agency said the treatment has uncertain long-term benefits and that its cost-effectiveness estimates were "substantially higher than the range normally considered" by the agency. The drug’s list price is £7,900 per 60 mg vial, according to an appraisal consultation document.
Still, the agency recognized that Evrysdi’s oral dosing makes it a better option for some patients. Evrysdi is the third SMA drug to win an approval in recent years behind Biogen’s blockbuster Spinraza and Novartis’ gene therapy Zolgensma. Both of those treatment options have scored NICE backing.
While Roche’s drug can be dosed at home, Spinraza is injected into the spinal fluid every four months after an initial four loading doses that occur closer together. Zolgensma is a one-time treatment given through an infusion.
“We are disappointed not to be able to recommend this innovative new treatment as an option for people with this devastating condition, particularly where there remains a high unmet clinical need,” Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said in a statement.
The agency’s decision will likely lead to further discussions. NICE said it’s open to comments until June 23 and will meet again on July 13. Until then, it will “work closely with the company to help them address the committee’s concerns.”
While disappointing, the NICE verdict wasn’t unexpected, according to SMA UK. The charity group said it expects Roche “to be working hard on their response” and to negotiate with the NHS to gain approval.
NICE currently recommends Spinraza for presymptomatic SMA patients or patients with type 1, 2 or 3 disease under a managed access deal. That drug costs £450,000 for the first year and £225,000 for subsequent years, but NICE and Biogen inked a deal for a confidential discount.
In March, the agency also endorsed Zolgensma, which costs £1.79 million per dose, for babies aged up to 12 months with type 1 SMA. Novartis also has also agreed to provide a discount for its gene therapy.