High-priced orphan gene therapies are expected to put health systems under pressure

The FDA has approved Novartis-subsidiary AveXis Inc’s spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec-xioi). The company wants to set the price tag at $2,125m per dose.

Most patients with the inheritable genetic defect in the SMN1 (survival motor neuron 1) gene, leading to progressive muscle weakness and paralysis, die before they are two years old. Novartis’ subsidiary AveXis Inc on Friday received FDA approval for Zolgensma (AVXS-101) as a treatment of SMA patients less than 2 years of age, even with presymptomatic disease. According to claims of Novartis a single injection can cure the disease. A long term follow up study (15 years) is ongoing. The company announced to set the price at $2.125m per dose in the US, making it the world’s most expensive drug.

High-priced orphan gene therapies are expected to put health systems under pressure as their producers demand high prices for potentially curative treatments which have only been validated in a low number of patients upon approval.

Source: European Biotechnology