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UK price watchdog turns down Biogen's rare muscle disease treatment for NHS use

The U.K.'s National Institute for Health and Care Excellence, or NICE, did not recommend Biogen Inc.'s rare muscle disease treatment Spinraza for use under the National Health Service.

The U.K. price watchdog said there were significant uncertainties regarding Spinraza's long-term benefits, and cited an extremely high cost for turning down Spinraza in its draft guidance, adding that even after the proposed confidential discount the therapy wasn't found to be cost-effective.

Spinraza, also known as nusinersen, treats a rare genetic disorder called spinal muscular atrophy, or SMA. The disease affects the nerves in the spinal cord leading to muscle weakness, progressive loss of movement, and difficulty breathing and swallowing.

There are between 1,200 and 2,500 children and adults in the U.K. living with SMA. Those with the most severe forms of SMA usually die before reaching the age of 2, and there are currently no active treatments targeting the underlying cause of SMA, NICE said in its news release.

Spinraza is the first treatment to address the cause of motor neuron degeneration in SMA — when nerves in the brain and spine progressively lose function. The drug is injected directly into the spine and is a lifelong treatment.

Spinraza has a list price of £75,000 per vial, NICE said.

The U.K. regulator added that Cambridge, Mass.-based Biogen intends to develop a proposal for a managed access arrangement regarding Spinraza. A managed access arrangement is a process in which patients can receive new treatments with uncertain long-term data at discounted cost and before final funding decisions are taken.

NICE added that the managed access arrangement is rarely used for treatments other than cancer drugs or certain new and existing highly specialized medicines.

Biogen received U.S. Food and Drug Administration approval for Spinraza in end 2016. The company also received European Medicines Agency recommendation for the therapy in 2017.