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Sanofi (SNY) Gets FDA Nod for Rare Disease Drug Xenpozyme
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Sanofi (SNY - Free Report) announced that the FDA has approved Xenpozyme (olipudase alfa) for the treatment of adult and pediatric patients with non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare, progressive, and potentially life-threatening genetic disease.
The FDA approval was based on data from two clinical studies — ASCEND and ASCEND-Peds — in which treatment with Xenpozyme led to clinically relevant improvements in lung function and reduction in spleen and liver volumes in the given patient population.
Enlarged spleen or liver, difficulty in breathing, lung infections, and unusual bruising or bleeding are some of the signs and symptoms of this rare disease, which particularly affects infants and children and is associated with substantial morbidity and risk of premature death.
Xenpozyme was under priority review in the United States while the FDA previously granted Breakthrough Therapy designation to the drug.
Following the nod, Xenpozyme became the first disease-specific therapy to be approved by the FDA for the treatment of ASMD. Xenpozyme has currently become the only approved treatment for the given disease.
Per the company, fewer than 120 patients are diagnosed with ASMD in the United States, with almost two-thirds of them being pediatric patients. Supportive care and careful monitoring to detect potential disease complications were the only way to manage ASMD until now.
Hence, the approval for Xenpozyme offers a new treatment option for the given patient community to treat this rare disease.
Sanofi plans to launch Xenpozyme in the upcoming weeks in the United States. The wholesale acquisition cost of Xenpozyme has been fixed at $7,142 per vial in the country.
Shares of Sanofi have plunged 18.1% so far this year compared with the industry’s decrease of 3.8%.
Image Source: Zacks Investment Research
The European Commission approved Xenpozyme for the treatment of ASMD in June 2022. The drug was previously approved in Japan for the same indication in March.
Achilles Therapeutics’ loss per share estimates have narrowed 6.4% for 2022 and 9.6% for 2023 in the past 60 days.
Earnings of Achilles Therapeutics surpassed estimates in three of the trailing four quarters and missed on the remaining occasion. ACHL delivered an earnings surprise of 12.45%, on average.
Atara Biotherapeutics’ loss per share estimates have narrowed 43.2% for 2022 and 31.8% for 2023 in the past 60 days.
Earnings of Atara Biotherapeutics surpassed estimates in three of the trailing four quarters and missed on the other occasion. ATRA delivered an earnings surprise of 4.83%, on average.
ORIC Pharmaceuticals’ loss per share estimates have narrowed 8.6% for 2022 and 22% for 2023 in the past 60 days.
Earnings of ORIC Pharmaceuticals surpassed estimates in three of the trailing four quarters and missed on the remaining occasion. ORIC delivered an earnings surprise of 8.85%, on average.
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Sanofi (SNY) Gets FDA Nod for Rare Disease Drug Xenpozyme
Sanofi (SNY - Free Report) announced that the FDA has approved Xenpozyme (olipudase alfa) for the treatment of adult and pediatric patients with non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare, progressive, and potentially life-threatening genetic disease.
The FDA approval was based on data from two clinical studies — ASCEND and ASCEND-Peds — in which treatment with Xenpozyme led to clinically relevant improvements in lung function and reduction in spleen and liver volumes in the given patient population.
Enlarged spleen or liver, difficulty in breathing, lung infections, and unusual bruising or bleeding are some of the signs and symptoms of this rare disease, which particularly affects infants and children and is associated with substantial morbidity and risk of premature death.
Xenpozyme was under priority review in the United States while the FDA previously granted Breakthrough Therapy designation to the drug.
Following the nod, Xenpozyme became the first disease-specific therapy to be approved by the FDA for the treatment of ASMD. Xenpozyme has currently become the only approved treatment for the given disease.
Per the company, fewer than 120 patients are diagnosed with ASMD in the United States, with almost two-thirds of them being pediatric patients. Supportive care and careful monitoring to detect potential disease complications were the only way to manage ASMD until now.
Hence, the approval for Xenpozyme offers a new treatment option for the given patient community to treat this rare disease.
Sanofi plans to launch Xenpozyme in the upcoming weeks in the United States. The wholesale acquisition cost of Xenpozyme has been fixed at $7,142 per vial in the country.
Shares of Sanofi have plunged 18.1% so far this year compared with the industry’s decrease of 3.8%.
Image Source: Zacks Investment Research
The European Commission approved Xenpozyme for the treatment of ASMD in June 2022. The drug was previously approved in Japan for the same indication in March.
Zacks Rank & Stocks to Consider
Sanofi currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector include Achilles Therapeutics plc (ACHL - Free Report) , Atara Biotherapeutics, Inc. (ATRA - Free Report) and ORIC Pharmaceuticals, Inc. (ORIC - Free Report) , all carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Achilles Therapeutics’ loss per share estimates have narrowed 6.4% for 2022 and 9.6% for 2023 in the past 60 days.
Earnings of Achilles Therapeutics surpassed estimates in three of the trailing four quarters and missed on the remaining occasion. ACHL delivered an earnings surprise of 12.45%, on average.
Atara Biotherapeutics’ loss per share estimates have narrowed 43.2% for 2022 and 31.8% for 2023 in the past 60 days.
Earnings of Atara Biotherapeutics surpassed estimates in three of the trailing four quarters and missed on the other occasion. ATRA delivered an earnings surprise of 4.83%, on average.
ORIC Pharmaceuticals’ loss per share estimates have narrowed 8.6% for 2022 and 22% for 2023 in the past 60 days.
Earnings of ORIC Pharmaceuticals surpassed estimates in three of the trailing four quarters and missed on the remaining occasion. ORIC delivered an earnings surprise of 8.85%, on average.