FDA approves first oral treatment for a rare muscle-destroying disease

The Food and Drug Administration on Friday approved the first oral therapy for spinal muscular atrophy, a rare and often devastating disease, adding a new option for patients who now rely on costly injected treatments.

The drug, from partners Genentech and PTC Therapeutics, is approved for adults and children with SMA, regardless of the severity of their disease. The once-a-day treatment will compete with a one-time gene therapy from Novartis and a drug from Biogen that is injected into the spine every four months.

Genentech and PTC’s treatment, branded Evrysdi, is dosed based upon weight, meaning its cost will vary patient by patient. In a statement, Genentech said Evrysdi’s maximum price would be $340,000 per year. For patients who weigh less than 44 pounds, the cost would be less. Treatment for patients under 2 years old would generally cost less than $100,000 a year, according to the company.

By contrast, Novartis’s gene therapy, Zolgensma, costs $2.1 million for a single administration whose benefits have proved to last for years and could be lifelong. Biogen’s treatment, Spinraza, costs $750,000 in the first year and then $375,000 each year thereafter.

Genentech believes Evrysdi is priced at a discount to its competitors. The drug, a liquid administered by mouth, is also the only approved SMA treatment that can be taken at home.

SMA, which had no approved treatments until 2016, has become a lucrative market for the drug industry. Zolgensma, approved in 2018, brought in $361 million in revenue for Novartis last year and amassed $375 million in the first half of 2020. Biogen made roughly $2.1 billion from Spinraza in 2019 and expects the drug to drive its growth for years to come.

In clinical trials, Evrysdi improved motor function and led to an 81% survival rate in infants with the most severe form of SMA, which is almost universally fatal by age 2 when left untreated. In older patients with milder forms of the disease, Evrysdi showed a statistically significant improvement over placebo on a measure of patients’ motor function.

Read original article here