No child in Poland suffering from spinal muscular atrophy will take part in the draw for the Zolgensma gene therapy, the most expensive drug in the world. The therapy has so far been registered only in the USA, and is still experimental in Europe.
AveXis, a therapy producer belonging to the Swiss concern Novartis, has organized a lottery in which 100 children from outside the USA will get the medicine for free. However, the application to the lottery cannot be made by a parent, but only by a hospital or a doctor who has the possibility to administer a gene therapy preparation. On the other hand, in order to administer experimental therapy in Poland and in EU countries, the consent of the Minister of Health is necessary. The Ministry, however, has restrictions imposed and cannot give its consent to administer such therapy in case of other effective methods of treatment available on the market.
Zolgensma registered only in the USA
Zolgensma gene therapy costs over $2m and is aimed at children with type I spinal atrophy (SMA). The preparation is intended for children up to two years old.
Currently, the drug is only registered in the USA. This is due to the lengthy approval process by the European Medicines Agency. According to the SMA foundation, there is no evidence of a higher effectiveness of Zolgensma therapy compared to Spinraza, which is approved for the European market and reimbursed in Poland.