May 4, 2020
Positive opinion for gene therapy with Zolgensma in the Netherlands
Netherland's Medicines Evaluation Board conducted the assessment on Zolgensma - new gene therapy for treatment spinal muscular atrophy in young children. The European medicines agency gave a positive advice for Zolgensma's marketing authorization under certain conditions.

Spinal muscular atrophy (SMA) is a hereditary disease with the defect in Survival Motor Neuron-1 (SMN-1) gene. This leads to the lack of the SMN protein. Certain nerve cells that control muscles, the so-called 'motor neurons', die without SMN protein.

Zolgensma is the brand name for gene therapy whose active ingredient is called onasemnogenic abeparvovec. European medicines agency (EMA) gave a positive advice 'under conditions' for this therapy. With this type of approval, the EMA can recommend treatment with less evidence than normally needed, for diseases that have no good treatment options yet. Additional results from the ongoing researches on Zolgensma will be assessed by EMA.

Zolgensma treatment is now approved for patients with symptoms of the most severe form of SMA (SMA type 1) and for patients with only 1, 2, or 3 copies of the SMN-2 gene.

The European Commission is planning to discuss whether the positive opinion of the EMA can be accepted. After that, each EU country has to decide whether the cost of this treatment ($ 2.1 million per patient in the United States) associated with the health benefits.

See the full details here.

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