Programme starts for 100 ill infants

Swiss drugmaker’s scheme draws criticism from patient groups

GENEVA: Swiss drugmaker Novartis started a lottery yesterday to give the world’s most expensive medication to 100 ill babies and toddlers for free.

The one-dose gene therapy Zolgensma, which treats a rare degenerative muscle disease in infants, costs US$2.1 million (RM8.6 million) and has only been approved by health authorities in the United States so far.

While Novartis argues that a lottery will help parents of 100 children around the world, patient lobby groups have said the scheme does not solve the underlying problems of high cost and lacking regulatory approval.

When Novartis reported a US$7.1 billion profit for 2019 last week, it cited Zolgensma as one of its key growth drivers.

However, the company says it cannot produce more doses of the drug at lower prices because there are no additional production capacities beyond the regular US orders and the 100 ones that will be drawn.

“Despite all the criticism against this procedure, there is a lack of alternative proposals,” a Novartis spokesman told dpa.

Starting yesterday, one child will be drawn every few weeks to receive Zolgensma for free to treat spinal muscular atrophy (SMA).

The disease, estimated to affect one in 8,000 to 10,000 people around the world, weakens and shrinks muscles, which can lead to paralysis or respiratory failure.

Novartis has not revealed the number of lottery applications.

If authorities in the winner’s country allow Zolgensma on an exceptional basis, the infant will receive it within weeks, according to the drug company.