Roche, PTC will now get an FDA decision on their SMA drug in 6 months — facing off with Biogen, Novartis
A major competitor to Biogen’s Spinraza franchise could arrive as early as this coming May, as Roche and its biotech partner obtained a priority review for risdiplam.
FDA regulators will make a decision on the spinal muscular atrophy drug by May 24, 2020, according to PTC Therapeutics, who is bagging $15 million in milestone payment for the NDA filing acceptance.
The move comes on the heels of topline data from the Phase III SUNFISH trial, in which risdiplam met the primary endpoint on change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment, compared to placebo. The results confirmed earlier findings from part one of the study.
SUNFISH enrolled 180 patients with type 2 or 3 SMA between 2 and 25 years old. An earlier, open label trial dubbed FIREFISH covered those with the type 1 variation of the neuromuscular disease.
“The FIREFISH and SUNFISH trials were designed to represent the real world spectrum of people living with SMA and include many people previously underrepresented in clinical trials,” noted Levi Garraway, Roche’s newly minted head of global product development, in a statement.
In PTC’s release, CEO Stuart Peltz added that the NDA submission includes all three types of SMA patients “demonstrating improvements in motor functions and developmental milestones, and a compelling safety profile.”
Unlike Spinraza, an antisense oligonucleotide administered intrathecally every four months following initial loading doses, risdiplam is an oral medication to be taken daily. By modifying how the SMN2 gene is spliced, the drug supposedly increases functional SMN protein levels in both the central nervous system and peripheral tissues.
The drug is already decorated with the FDA’s orphan and fast track designations.
The filing could lead to a broader label than expected, according to a note from Credit Suisse analyst Evan Seigerman, who’s previously warned that risdiplam spells an even bigger threat to Biogen’s Spinraza franchise than Novartis’ gene therapy. Since gaining approval in May, Zolgensma has been plagued by reimbursement issues as payers push back against its $2.1 million price tag (although Novartis stresses that the payment is delivered in a 5-year installment plan for a supposed once-and-done cure). It’s also only approved for infants under the age of 2.
Although Spinraza has earned Biogen more than $1.5 billion in the first three quarters of 2019, Seigerman said he is “increasingly concerned” about its durability. Having launched late 2016 (and accrued more than $4.1 billion in recorded sales so far), the franchise may begin to wane come H2 2020.
And Roche is going all-in for the three-rival rivalry. Together with PTC and the SMA Foundation, the pharma giant is running more clinical trials with patients ranging from newborns to 60 years old, including those previously treated with other SMA therapies.