A Roche drug for spinal muscular atrophy is now on the market officially recognized For the treatment of infants under 2 years of age, to provide those born with this rare neuromuscular disorder a way to receive this treatment option at a younger age. The regulatory decision announced Tuesday also puts Roche drug Evrysdi on par with treatment options from Novartis and Biogen.
Evrysdi was first approved in 2020, becoming the third product approved for the treatment of SMA. The drug is a once-daily solution, making it the first approved oral SMA drug. But Biogen’s Spiraza, which is injected into the spine every four months, and Novartis’ gene therapy Zolgensma, a one-time infusion, are approved to treat young infants and adults. Evrysdi’s initial regulatory nod covers people aged 2 and older. With the expanded approval, Evrysdi could reach patients at a younger age, providing benefits to patients before their disease progresses.
SMA arises from defects in the SMN1 gene, which encodes a protein important for keeping motor neurons healthy. The disease causes progressive muscle weakness. Babies begin to miss key motor milestones, such as the ability to sit up and roll over. As the disease progresses, patients may need breathing assistance. Evrysdi is a small molecule designed to acquire the SMN2 gene to increase production of key motor neuron proteins in the central nervous system and peripheral tissues.
The latest regulatory nod to Roche’s SMA drug is based on interim efficacy and safety data from a neonatal clinical study. Roche reported that of six infants with two or three copies of the SMN2 gene, they were all able to sit after a year of treatment. 67% of infants can stand and 50% can walk independently. All infants in the study were alive at 12 months without permanent ventilation. The most common adverse reactions reported in the study were upper respiratory tract infection, lower respiratory tract infection, constipation, vomiting and cough.
“Due to Evrysdi’s efficacy in multiple settings, it can now be used in patients with SMA, from presymptomatic neonates to the elderly,” Levi Garraway, Roche’s chief medical officer and head of global product development, said in a prepared statement. “We are proud of this achievement, which has the potential to make a real difference for SMA patients and their caregivers.”
Evrysdi was originally developed by PTC Therapeutics of South Plainfield, NJ. In 2011, the company began a partnership with Roche, the pharmaceutical giant that will be responsible for the clinical development and commercialization of the SMA program. The drug is currently approved in 81 countries and is under review in another 27. According to Roche’s 2021 annual report, the company had revenue of 604 million Swiss francs ($629 million) last year. By contrast, Biogen’s Spiraza, also long-term, had $1.9 billion in sales last year. Zolgensma generated $1.3 billion in revenue for Novartis in 2021.
By the end of 2021, PTC reported receiving $160 million in milestone payments from Roche and $59.4 million in royalties from Evrysdi sales.
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