Roche Holding AG (RHHBY - Free Report) 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 - Free Report) .
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 - Free Report) Spinraza and Novartis’ (NVS - Free Report) gene therapy Zolgensma.
Zacks Rank
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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Roche Holding AG (RHHBY - Free Report) 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 - Free Report) .
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 - Free Report) Spinraza and Novartis’ (NVS - Free Report) gene therapy Zolgensma.
Zacks Rank
Roche currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Today's Best Stocks from Zacks
Would you like to see the updated picks from our best market-beating strategies? From 2017 through Q3 2019, while the S&P 500 gained +39.6%, five of our strategies returned +51.8%, +57.5%, +96.9%, +119.0% and even +158.9%.
This outperformance has not just been a recent phenomenon. From 2000 – Q3 2019, while the S&P averaged +5.6% per year, our top strategies averaged up to +54.1% per year.
See their latest picks free >>
Zacks Names "Single Best Pick to Double"
From thousands of stocks, 5 Zacks experts each have chosen their favorite to skyrocket +100% or more in months to come. From those 5, Director of Research Sheraz Mian hand-picks one to have the most explosive upside of all.
It’s a little-known chemical company that’s up 65% over last year, yet still dirt cheap. With unrelenting demand, soaring 2022 earnings estimates, and $1.5 billion for repurchasing shares, retail investors could jump in at any time.
This company could rival or surpass other recent Zacks’ Stocks Set to Double like Boston Beer Company which shot up +143.0% in little more than 9 months and NVIDIA which boomed +175.9% in one year.
Free: See Our Top Stock and 4 Runners Up >>
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