Drug hope for rare child muscle disease

CHILDREN with the most severe form of spinal muscular atrophy are to be given access to a ‘life-changing’ treatment. The charity Muscular Dystrophy UK says Spinraza is the first and only treatment for the inherited muscle-wasting condition.

The disorder can cause patients to lose the ability to move, breathe and swallow.

Without access to the drug, life expectancy for children with spinal muscular atrophy (SMA) type 1 – the most severe form – is rarely longer than two years.

The Scottish Medicines Consortium (SMC) yesterday recommended that the drug be made available on the NHS in Scotland for children with type 1 SMA. It is not available on the NHS in England.

It did not approve the treatment for patients with types 2 and 3 – which affect older children and are less severe – saying it was not cost effective in these cases.

Muscular Dystrophy UK, which has been campaigning for access to Spinraza, welcomed the news but said it would keep fighting for all patients.

There are an estimated 100 children in Scotland with SMA – but only a handful with the most severe form. The drug costs up to £450,000 a year per patient, but the SMC negotiated an undisclosed discount.

Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said the drug brought hope to many families.

He added: ‘We won’t rest until all patients have access to this treatment, and it is now vital that robust data is provided to support the argument for access for all people with SMA.

‘SMA is a devastating and cruel condition, and while it is not a cure, Spinraza can buy families more time.’

The drug’s manufacturer, Biogen, said the ‘landmark’ approval of the medication, also known as nusinersen, was the first in the UK for this treatment.

The drug is one of five approved by the SMC yesterday. Avelumab (Bavencio) was accepted for the treatment of Merkel cell carcinoma, an aggressive, rare form of skin cancer, while Regorafenib (Stivarga) was accepted for the treatment of advanced liver cancer in patients who have already had other treatment.

Selexipag (Uptravi) can now be used in Scotland to treat abnormally high blood pressure in the arteries of the lungs, while brodalumab (Kyntheum) was accepted for the treatment of severe plaque psoriasis, an inflammatory skin condition.

On Spinraza, SMC chairman Dr Alan MacDonald said: ‘I am pleased the committee was able to accept these five medicines for use by NHS Scotland.

‘The committee heard compelling evidence on the benefits of nusinersen, supported by the views of experts and patient groups, and was able to apply a very high degree of flexibility in accepting this medicine for type 1 SMA.

‘The evidence presented suggested that nusinersen was substantially less cost effective when used in types 2 and 3.

‘These are difficult decisions. The medicine is extremely expensive and there is a need to consider all those who need treatment by NHS Scotland.

‘We would welcome a resubmission from the company which addresses the issues raised.’

‘Devastating condition’