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Zolgensma°: the drug of extremes

For some years now, the escalating cost of new drugs has increasingly threatened access to health care. In 2019, Zolgensma° (onasemnogene abeparvovec-xioi) reached the absurd price of about 2 million euros for the single injection that is required to treat each patient.

At the beginning, it was a great story of hope for children suffering from spinal muscular atrophy, a rare genetic disease which is very disabling and carries a grim prognosis. The defective gene was identified in 1995 by a team from the Necker hospital in Paris. A group at Généthon (a French non-profit research laboratory funded by public donations through a telethon and by grants) then developed gene therapy enabling the defective gene to be modified, and filed a patent in 2007, together with the French National Centre for Scientific Research (CNRS). In 2011, the same team published encouraging results obtained in mice. A US start-up company, AveXis, tested this treatment in children and signed a licencing agreement with Généthon. Following encouraging trial results, Novartis bought AveXis in 2018 for 8.7 billion dollars.

The single injection will be billed at 1.9 million euros per child in the United States. Novartis downplayed the price by pointing out that it is "50% of the 10-year cost of chronic SMA treatment", referring to the exorbitant price of Spinraza° (nusinersen), which is authorised for the same disease. As for Généthon and the CNRS, they are expected to be sharing just "several tens of millions of euros over time".

This whole affair speaks volumes about the practices of the stakeholders involved in this outrageously profitoriented drug development programme. Effective action can only come from countries that present a united front, from citizen engagement, and from the courage of leaders convinced of the need to preserve access to health care and the collective resources destined for the community. And who are prepared, to that end, to begin by demanding transparency from companies and start-ups as regards the costs of research and production.

©Prescrire 1 April 2020

"Zolgensma°: the drug of extremes" Prescrire Int 2020; 29 (214): 107. (Pdf, free).

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