NICE rejects risdiplam for treating spinal muscular atrophy

pharmafile | June 2, 2021 | News story | Research and Development  

NICE has issued draft guidance today which does not recommend Roche’s risdiplam (Evrysdi) for treating the rare genetic disorder spinal muscular atrophy (SMA).

SMA is a progressive neuromuscular condition affecting the nerves in the spinal cord controlling movement. This leads to muscle weakness, progressive loss of movement, and difficulty breathing and swallowing.

SMA can be grouped into five main types (types 0 to 4), based on the age of onset and the maximum motor function reached.

SMA type 1 is one of the most severe forms, and people with it usually die before the age of two if untreated. Due to the progressive nature of SMA, everyone will experience more severe symptoms over time regardless of type.

Risdiplam is licensed to treat people with types 1, 2 and 3 SMA. Had risdiplam been recommended, around 1,500 people would have been eligible for treatment.

The committee agreed that the clinical trials demonstrate that risdiplam meaningfully improves motor function for people with type 1, 2, and 3 SMA, but the long-term benefits are uncertain.

Therefore, the committee could not currently recommend the drug, due to the cost-effectiveness estimates being substantially higher than the range normally considered an effective use of NHS resources.

In the case of type 1, this was despite NICE agreeing that risdiplam could be considered a life-extending treatment at the end of life. The committee also acknowledged the rarity and severity of the disease in its decision making.

Meindert Boysen, Deputy Chief Executive and Director of the Centre for Health Technology Evaluation at NICE, said: “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.

“The committee considered that there may be some benefits that have not been captured in the company’s economic model, such as improvements in respiratory function, fine motor skills, swallowing, and ability to communicate.

“We will continue to work closely with the company to help them address the committee’s concerns ahead of its next meeting.”

This news comes shortly after a five-month-old baby was one of the first patients to receive Zolgensma for SMA on the NHS.

NICE currently recommends nusinersen for some people with types 1, 2, and 3 SMA as part of a managed access agreement.

NICE has also published draft guidance which recommends onasemnogene abeparvovec for some babies aged up to 12 months with type 1 SMA.

Lilly Subbotin

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