Roche Holding AG RHHBY announced that the FDA has accepted the new drug application (NDA) for its investigational agent risdiplam. The company is seeking approval of the candidate for the treatment of patients with spinal muscular atrophy (SMA).
Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN-2) splicing modifier for SMA.
With the FDA granting a priority review to the NDA, a decision from the regulatory body is expected on May 24, 2020. If approved, risdiplam will become the first at-home administered medicine for people with SMA.
We note that a Priority Review designation is granted to drugs with potential to provide significant improvements in the safety and effectiveness of the treatment plus prevention or diagnosis of a serious disease.
The NDA was based on 12-month data from the pivotal FIREFISH and SUNFISH studies, which evaluated risdiplam across a broad patient population suffering type 1, 2 or 3 SMA.
In November, Roche announced positive data from the part 2 of the SUNFISH study on risdiplam. The study met its primary endpoint of change from baseline in the MFM-32 scale after one-year regimen with risdiplam compared to placebo, as data showed statistically significant improvements in the overall study population with type 2 or 3 SMA.
Moreover, no treatment-related safety findings causing study withdrawal have been seen in any risdiplam study to date.
Shares of Roche have rallied 22.5% so far this year compared with the industry’s increase of 5.1%.
We remind investors that Roche leads the clinical development of risdiplam in collaboration with the SMA Foundation and PTC Therapeutics PTCT.
Risdiplam is also being evaluated in a broad clinical program for SMA in patients ranging from newborns to 60 year-olds. It includes those who are previously treated with SMA-targeting therapies. Additionally, the company has two ongoing studies, namely JEWELFISH and RAINBOWFISH.
Successful development of risdiplam will boost Roche’s growth prospects. However, competition is stiff in the SMA market from the likes of Biogen’s BIIB Spinraza and Novartis’ NVS gene therapy Zolgensma.
Roche currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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