Patient groups are criticizing Novartis (NVS +0.9%) over its lottery-style program aimed at providing pricey gene therapy Zolgensma (onasemnogene abeparvovec-xioi) free-of-charge on a limited basis to SMA patients outside of the U.S. Patient advocates regard the program as inappropriate for a life-saving therapy for babies.
The company’s decision was based on advice from bioethicists concerned that creating complicated criteria would unfairly discriminate against some patients. It plans to distribute ~100 free doses each year contingent on production capacity.
About 1,600 children with SMA Type 1 live in Europe alone so global ex-U.S. demand dwarfs availability via lottery.
Zolgensma, approved by the FDA in March, is the world’s most expensive drug at $2.1M per dose.
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