European Commission approves expensive treatment of rare muscular diseases | Right away

The European Commission has approved the costly treatment of sMA muscle diseases in small children using the drug Zolgensma. Pharmaceutical company Novartis said on Tuesday it hoped the treatment would be available soon.

Previously, the drug was approved by the European Medicines Agency (EMA).

A one-off treatment costs more than 1.9 million euros. The drug is manufactured for the treatment of spinal muscular atrophy, a progressive muscle disease. This condition can weaken the muscles of patients with SMA type 1 at a very fast pace, preventing them from breathing or moving.

In January of this year, there was excitement because Novartis had created a kind of lottery to give away the drug among patients. The company did so because the product was not yet approved for the European market.

“Lotteries are indeed gambling, and this model is absolutely not in line with health care,” said the health ministers of Belgium, the Netherlands, Luxembourg, Austria and Ireland.