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On May 24th, 2019 the pharmaceutical company Novartis received approval to commercialize Zolgensma, its new treatment, on the US market.
Zolgensma treats spinal muscular atrophy (SMA) a genetic disease that affects nerve cells causing muscle atrophy or even paralysis for pediatric patients. In the most serious cases, this disease could cause the deaths of babies in their first two years.
The treatment resulting from long-term scientific research, is administered in a single injection costing 2.125 million USD. In order to facilitate the patients’ access to the new treatment, Novartis has negotiated its payment with insurers and mutuals to create 5-year outcomes-based agreements.
While the cost of Zolgensma seems highly expensive, it is only half that of Spinraza (Nusinersen), the first SMA treatment prescribed for 10 years. The first year costs 750 000 USD, the nine remaining years reach 375 000 USD per year.
Just like in the US, Zolgensma should be approved in Europe and Asia to allow a maximum number of patients to access the treatment.
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