Breakthrough for kids with genetic condition in Scotland
The NHS in Scotland has agreed to provide treatment for those with a severe form of spinal muscular atrophy
Last updated 8th May 2018
Children suffering from the most severe form of a genetic condition will be given access to a "life changing" treatment on the NHS in Scotland.
The charity Muscular Dystrophy UK says that Spinraza is the first and only treatment for patients with spinal muscular atrophy (SMA), a rare and inherited muscle-wasting condition that can cause patients to lose the ability to move, breathe and swallow.
Without access to it, life expectancy for those with SMA type 1 - the most severe form - is rarely longer than two years.
The Scottish Medicines Consortium (SMC) recommended on Monday that the drug be made available on the NHS in Scotland for children with type 1 SMA.
It did not, however, approve the treatment for patients with type 2 and 3 of the condition.
Muscular Dystrophy UK, which has been campaigning for access to Spinraza for all patients with the condition, welcomed the news but said it will keep fighting until all patients with SMA have access to the treatment.
Robert Meadowcroft, chief executive of the charity, said: "Spinraza brings hope to many families, and we welcome the news that it will be made available by NHS Scotland to children with SMA type 1.
"But we won't rest until all patients with SMA have access to this treatment, and it is now vital that robust data is provided by (manufacturer) Biogen to support the argument for access for all people with SMA, as we turn our focus to encouraging the SMC to reassess the drug for other types.
"SMA is a devastating and cruel condition, and while it is not a cure, Spinraza can buy families more time.''
Biogen said the "landmark'' approval of the drug, also known as nusinersen, is the first in the UK for this treatment.
Terry O'Regan, managing director and vice president of Biogen UK and Ireland, said: "Biogen is extremely proud to bring nusinersen to patients in Scotland.''
The drug is one of five approved by the SMC on Monday.
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 undergone 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: "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.
"Unfortunately, the evidence presented suggested that nusinersen was substantially less cost effective when used in types 2 and 3 and therefore could not be accepted by the committee.
"These are incredibly 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."