Kids with spinal muscular atrophy to receive costly drug on compassionate grounds

80 Israeli youngsters suffer from the rare genetic disease.

November 15, 2017 03:25
1 minute read.
Program for children to observe animals

A child looking at the monkeys at the Ramat Gan safari from her hospital bed . (photo credit: RAMAT GAN SAFARI)

Up to 15 children with severe cases of debilitating spinal muscular atrophy will receive the drug Spinraza, which costs NIS 2 million a year per patient.

The orphan drug will be supplied by its developer, Biogen Inc., for compassionate reasons, Health Minister Ya’acov Litzman told the Finance Committee on Tuesday.

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A total of 80 Israeli youngsters suffer from the rare genetic disease, which can significantly improve with the help of the drug. The European Commission approved the drug for marketing six months after the FDA approved it to treat children with SMA. Litzman has instructed all hospitals to provide the drug as an immediate response to SMA for all the serious (type 1) patients suffering from the disease.

SMA is a rare neuromuscular disorder that causes progressive muscle wasting, which often leads to early death. Spinraza (nusinersen) has been found to significantly improve the patient’s functioning. The minister told the committee that he is working on a way to provide treatment to every patient who needs the drug, but did not explain how.

It the drug had to be supplied by the basket of health services for 2018, whose contents are being decided on now, almost one third would have gone to pay for Spinraza. The health basket committee learned that Britain faces a similar dilemma and will supply the drug to some of its affected children.

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