Zurich (AFP)

The Swiss pharmaceutical group Roche has obtained approval in the United States of Evrysdi, a new treatment for spinal muscular atrophy, he announced on Monday.

The US drug agency, the Food and Drug Administration (FDA), has given the green light to this drug for children aged two months and older as well as for adult patients affected by this severe neurodegenerative disease, has it stated in a statement.

Also called risdiplam, this drug is "the first and only" treatment that can be given at home, Roche said. In liquid form, it can be administered directly orally or through a food probe.

The approval was granted based on the results of two studies involving 450 patients at different ages and stages of the disease, which demonstrated significant improvement in important motor functions.

Spinal muscular atrophy is a rare but serious neurodegenerative disease linked to a defective gene that causes muscle atrophy. Babies affected by this disease quickly lose motor neurons for essential muscle functions, such as breathing, swallowing, speaking and walking.

Without treatment, the baby's muscles weaken and gradually progress to paralysis or death. The disease affects about one in 10,000 babies, Roche said. The treatment will be available in the United States within two weeks, said the Swiss laboratory.

Long considered incurable, the treatment of this disease has made significant progress with the arrival on the market of new classes of drugs, promising but very expensive. At the end of 2016, the American Medicines Agency first approved the treatment from the American laboratory Biogen, called spinraza, then gave the green light last year to gene therapy from its Swiss competitor Novartis, called Zolgensma.

"Although it is arriving late on the market," Roche's treatment should find its place among its competitors, especially thanks to its oral formula, said Stefan Schneider, analyst at Vontobel, in a stock market commentary. He estimates for the moment the potential annual turnover of this drug, at its peak of sales, at around 500 million Swiss francs (463 million euros).

Despite this approval, the title yielded 0.08% to 313.95 Swiss francs at 10:30 GMT, against the trend of the SMI, the flagship index of the Swiss Stock Exchange, up 0.51%, weighed down by a setback during clinical trials on another treatment.

In a separate statement, Roche said "mixed" results for its investigational drug etrolizumab in clinical trials for bowel disease.

© 2020 AFP