- The Food and Drug Administration will give Roche's experimental therapy for spinal muscular atrophy a speedy review and will decide on approval within six months, the Swiss pharma said Monday.
- If approved, Roche's risdiplam would be the third SMA therapy to reach market in the U.S., following approvals for Biogen's Spinraza and Novartis' Zolgensma. Less than three years ago, there were no FDA-approved medicines for SMA, a rare disease that is the most common genetic cause of infant mortality.
- Roche executives are hoping for a broad label, submitting clinical results to the FDA from patients with the most severe as well as more moderate forms of the neurodegenerative disease. The agency is expected to make a decision by May 24, 2020, the pharma said.
As therapeutic options for SMA expand, treatment may become more specific to the disease's various types, which are classified by when symptoms materialize as well as by the number of copies of an SMA-related gene.
Type 1 is the most common and severe form, with symptoms starting to appear in newborns within their first few months of life. On the other end of the spectrum, Type 4 is considered an adult-onset disease, bringing with it mild or moderate symptoms and little effect on life expectancy.
Biogen won the first FDA approval for an SMA therapy with Spinraza (nusinersen) in December 2016. Spinraza was approved for all types of the disease and has become a multi-billion dollar drug for the Cambridge, Massachusetts-based biotech.
Earlier this year, the FDA approved a gene therapy for SMA patients under the age of two. Zolgensma (onasemnogene abeparvovec) has already exceeded initial sales expectations since launching, earning $160 million in the months of July, August and September.
Now, pending FDA approval, risdiplam is poised to enter the market in 2020. Wall Street analysts expect the oral SMA therapy to grow into a blockbuster therapy, too, with several banks estimating $2 billion to $3 billion in peak annual sales.
By aiming for a broad patient population, Roche's drug will initially pose more of a challenge to Spinraza than Zolgensma, Wall Street analysts noted Monday. Cantor Fitzgerald analyst Alethia Young said the oral delivery is "a huge advantage" over Spinraza, which requires an injection into the spinal canal several times per year.
Risdiplam's filing includes clinical data from FIREFISH, an open-label study of Type 1 infants, and SUNFISH, which tested children and adults from ages two to 25 with the less severe Types 2 or 3.
Earlier this month, Roche announced topline results from SUNFISH, saying risdiplam beat placebo on the study's primary goal of improving motor function from baseline.
Additional trials are ongoing that test the oral medication in an even larger SMA patient population, ranging from asymptomatic newborns to 60-year-olds.
None of the three drugmakers developed the SMA therapies themselves. Biogen and Roche both acquired their drugs through licensing deals with Ionis Pharmaceuticals and PTC Therapeutics, respectively. And Novartis paid nearly $9 billion last year to acquire AveXis, which developed Zolgensma.
Even as treatment advances hold out new hope for SMA patients previously without options, the drugs have come under criticism for their high costs.
Novartis set a $2.1 million list price for Zolgensma, making it the world's most expensive medicine. The company justified that cost with the gene therapy's potential to act as a cure via a one-time treatment.
Biogen's Spinraza, meanwhile, carries a $750,000 list price for the first year of treatment, followed by $375,000 each following year. ICER, an influential nonprofit that analyzes drug pricing, concluded in April that Spinraza exceeds common thresholds for cost-effectiveness.
Roche has yet to say how it will approach pricing for risdiplam.
The FDA filing triggered a $15 million milestone payment from Roche to PTC. The New Jersey-based biotech's stock jumped as much as 8% Monday morning.