A Roche drug that treats spinal muscular atrophy is now approved for treating babies younger than 2 months, providing 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 the Roche drug, Evrysdi, on equal competitive footing versus treatment options from Novartis and Biogen.
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Evrysdi was first approved in 2020, making it the third product authorized for treating SMA. The drug is a solution that’s taken once daily, making it the first approved oral SMA drug. But Biogen’s Spinraza, given as an injection into the spine every four months, and Novartis gene therapy Zolgensma, given as one-time infusion, were approved to treat young babies as well as adults. Evrysdi’s initial regulatory nod covered those age 2 months and older. With the expanded approval, Evrysdi can reach patients at a younger age, providing benefit to patients before their disease can progress.
SMA stems from a defect in the SMN1 gene, which encodes a protein that is important for keeping motor neurons healthy. The disease leads to progressive muscle weakness. Babies start to miss key movement milestones, such as the ability to sit up and roll over. As the disease worsens, patients may need breathing assistance. Evrysdi is a small molecule intended to get the SMN2 gene to increase the production of the key motor neuron protein in the central nervous system and peripheral tissues.
The latest regulatory nod for Roche’s SMA drug was based on interim efficacy and safety data from a clinical study in newborns. Of the six babies with two or three copies of the SMN2 gene, Roche reported all of them were able to sit with after one year of treatment; 67% could stand and 50% of infants could walk independently. All infants in the study were alive at 12 months without the need for permanent ventilation. The most common adverse reactions reported in the study were upper respiratory tract infection, lower respiratory tract infection, constipation, vomiting, and cough.
“Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA from pre-symptomatic newborns to older adults,” 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 to those living with SMA and their caregivers.”
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Evrysdi was initially developed by South Plainfield, New Jersey-based PTC Therapeutics. In 2011, the company began a partnership with Roche under which the pharmaceutical giant would take handle clinical development and commercialization of the SMA program. The drug is currently approved in 81 countries and is under review in 27 more countries. It accounted for 604 million Swiss francs (about $629 million) in revenue last year, according to Roche’s 2021 annual report. By comparison, Biogen’s Spinraza, which is also taken chronically, generated $1.9 billion in sales last year. Zolgensma produced $1.3 billion in revenue for Novartis in 2021.
As of the end of 2021, PTC reported receiving $160 million in milestone payments from Roche and $59.4 million in royalties from Evrysdi sales.
Photo: Giuseppe Aresu/Bloomberg, via Getty Images
