NHS approves new drug for muscle-wasting disease

As reported by The Guardian, a drug that could prolong and improve the lives of those suffering with a muscle-wasting disease has finally been approved by the NHS

A drug that may prolong the lives of children with spinal muscular atrophy (SMA) has been approved by the NHS.

The drug – Spinraza – will now be available after a long negotiation between the NHS and the manufacturer over its price.

Spinraza could help between 600 and 1,200 children and adults in England and Wales with SMA.

As a muscle-wasting disease, SMA can shorten the life expectancy of babies and toddlers in particular.

There is limited data regarding Spinraza’s long-term effectiveness, though it is proven to help slow the progress of the disease.

NICE previously turned down the drug due to its price.

Simon Stevens, chief executive of the NHS said that an agreement had been reached and children would shortly get access to Spinraza.

“This promising treatment has the potential to be life changing for children and their families,” said Stevens.

“The NHS has now reached one of the most comprehensive deals in the world, which allows us to assess real-world evidence of its long-term benefits.”

Stevens also hinted that other drug companies should consider compromising on price in future.

He said: “This latest deal, coming on the heels of a number of other recent successful negotiations, demonstrates that there is no reason for other companies not to show equivalent flexibility in order to benefit NHS patients, taxpayers and indeed themselves.”

Catherine Woodhead, the chief executive of Muscular Dystrophy UK, which has been campaigning for Spinraza to be made available on the NHS, said: “Today’s announcement is fantastic news that gives families hope. Children already receiving treatment are reaching milestones never thought possible and living longer, and now hundreds of others will be given that same chance.

“Every day counts for people with SMA, and we need to ensure this decision is implemented as soon as possible and made available UK-wide. Our work here is not done. The lengthy, frustrating delays, which we have seen throughout this process must not be allowed to happen again.

“During this time, there will have been irreversible decline in SMA patients’ health. The appraisal system for new treatments for rare diseases needs to be urgently overhauled to make it fit for purpose for future treatments for rare conditions.”

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