Lottery Determining Which Babies Will Receive The Most Expensive Treatment In The World

Zolgensma, which became the most expensive medicine in the world last May, will be offered to one hundred babies under two years of age, who will be chosen by lot on January 2. The Swiss laboratory Novartis, owner of the treatment, last May fixed the price of this single injection against spinal muscular atrophy (SMA), whose results are particularly encouraging. The salary amounts to almost 380,000 euros per year, for 5 years. It therefore amounts to almost 2 million euros.

Deliveries of the precious gene therapy drug to the first children will take place in February 2020, . As a first step, 50 doses will be offered by June and the rest will be distributed in 2020.

The disease affects between one in 6,000 newborns and one in 10,000. SMA causes muscle wasting of the limbs and trunk. When it occurs very early, pathology can shorten a baby’s lifespan to just a few months.

In November 2017, a study had shown the benefits of a gene therapy developed by French researchers and finalized by the American start-up AveXis. The latter was then bought by Novartis in May 2018. Research by French specialists was notably funded by public money and AFM-Telethon funds.