Single treatment with the drug Zolgensma costs € 1.9 million per patient in the United States. The European price of the drug from pharmaceutical company Avexis-Novartis is not yet known.
Spinal muscular atrophy (SMA) is a serious genetic muscle disease, which is sometimes fatal before the first year of life. In our country, there are about ten children with this rare disease, which leads to increasingly severe paralysis. Worldwide, this involves an estimated 15,000 children.
Admission in the Netherlands
It may take a while before the drug is available for Dutch patients. The Healthcare Institute must first issue an opinion on admission to the basic package, after which the manufacturer must negotiate with Minister Van Rijn (Medical Care) about the price.
Gene therapy makes it possible to halt the development of SMA in children up to two years of age through one-off treatment. Babies are injected with a missing gene through an injection, with the aim of stopping the progression of the disease.
In the Netherlands, a medicine is already available, Spinraza, but it must be administered for life. Parents also fear that Spinraza does not work sufficiently or not fast enough.
Pharmaceutical Avexis-Novartis caused quite a stir early this year with a “raffle” of a hundred doses of Zolgensma in countries where the drug is not yet on the market. Minister of Care at the time, Bruins, expressed his dissatisfaction with the “lottery”. He stated that the manufacturer is “giving false hopes to false parents,” and urged a medically and ethically appropriate way to deliver the drug to all patients who need it.
The Inspectorate for Health and Youth granted the University Medical Center Utrecht permission to register two patients for the ‘draw’. UMCU called for a direct decision on the approval of the product to be issued quickly, “even though Zolgensma does not have sufficient scientific evidence of its potential effects and its long-term risk.”
After approval by the European Commission, according to the manufacturer, the product will in any case be immediately available in France and soon also in Germany.
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