Unconventional drug ‘lottery’ will hand out 50 doses of Zolgensma, world’s most expensive gene therapy

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Wynter, a little girl with SMA. Image: Jamie Clarkson

The lottery that began [recently] was not about money, or about choosing a school, or about obtaining a visa. It was about a child’s life. In this case, the children selected would receive a drug that otherwise was not available.

The treatment, a gene therapy called Zolgensma, is designed for children like Wynter who have a neuromuscular disease called spinal muscular atrophy, or SMA.

The lottery was devised by the drug’s manufacturer, Novartis, to give families in those places a chance to get it through a novel form of compassionate use — a way to get medications that have not been approved — while they wait. Fifty doses are slotted to be given away for free in the first half of the year, with up to 100 total.

Related article:  ALS may soon be treatable with new drug combo dreamed up by a college student

Ethicists and advocates have debated the merits and the design of the unusual arrangement. Parents said that it was uncomfortable to cast their child’s fate into what felt like a sweepstakes — a kind of bizarre Willy Wonka contest in which, as Maura Blair, a Canadian mother of a child with SMA put it, “we’re talking about lives.” …

Zolgensma costs $2.1 million in the United States — the world’s most expensive drug.

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