A third spinal muscular atrophy treatment is set to debut with the FDA’s approval for Roche’s Evrysdi, formerly known as risdiplam. The medicine offers at-home dosing for patients, potentially giving it an edge versus the competition during the COVID-19 pandemic.
Another potential edge? Price. Genentech set Evrysdi’s list price at up to $340,000 per year, depending on a patient’s weight. For some younger patients, the price could be under $100,000, a spokesman said.
“We carefully considered a number of factors, including input from the SMA community, and took a long-term, thoughtful approach in determining the price of Evrysdi to help ensure those who are prescribed can access this first and only at-home SMA treatment, shown to be safe and effective across a wide range of ages and SMA types in our clinical trials,” he added.
The new med is set to enter a competitive SMA field after launches in recent years for Biogen’s Spinraza and Novartis’ Zolgensma. Those companies have been battling for market share and working to grow sales. Spinraza sales reached $2.1 billion last year, while Zolgensma generated $361 million during its launch year.
Those meds also carry high price tags of $750,000 for the first year of Spinraza and $2.125 million for Zolgensma, which is intended to be a one-time treatment. Spinraza’s list price drops to $375,000 for the second and subsequent years of treatment.
Despite the fact there are already two approved medications in SMA, Dalia Moawad, Genentech’s head of neuro rare disease U.S. medical affairs, said most patients aren't getting treatment.
“Even though the innovation has certainly happened in this field over the last several years … we know that in the U.S. alone, the majority of SMA patients go untreated,” she said in an interview.
And with its at-home dosing, the new medicine is “emerging as a very important therapeutic option and coming at a very critical juncture for the SMA community,” she added.
The FDA approved Evrysdi based on data from two phase 3 studies, the first being a trial in symptomatic patients aged 2 to 7 months, and the other in children and adults aged 2 to 25 years. The latter trial, Sunfish, is the only placebo-controlled trial of an SMA therapy in adults with type 2 and 3 disease, according to Roche's biotech outfit Genentech.
In the studies, patients taking the drug showed improvement in motor function, including the ability to sit without support for 5 seconds for infants, a development not normally seen in the natural course of the disease. The drug also improved survival without permanent ventilation.
Evrysdi is the result of a collaboration between Roche’s Genentech, the SMA Foundation and PTC Therapeutics. The liquid drug is administered daily by mouth or feeding tube. It's set to launch in the U.S. within two weeks.