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BioMarin (BMRN) Gets CHMP Nod for Hemophilia Drug Roctavian
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BioMarin Pharmaceutical Inc. (BMRN - Free Report) receives a positive opinion from the Committee for Medicinal Products for Human Use (“CHMP”), recommending a conditional marketing authorization (CMA) for its gene therapy candidate, valoctocogene roxaparvovec, to treat adults with severe hemophilia A.
The company is awaiting the final decision from the European Commission (EC), typically coherent with the CHMP recommendation, expected by third-quarter 2022.
The single-dose infusion gene therapy (valoctocogene roxaparvovec) will be marketed with the tradename of Roctavian. The treatment will be available for adult patients with severe hemophilia A who were not previously treated with factor VII inhibitors and do not have detectable antibodies to adeno-associated virus serotype 5. Roctavian will be the first gene therapy for patients with hemophilia A in Europe following a potential conditional approval.
Hemophilia A is a hereditary bleeding disorder caused by the lack of blood clotting Factor VIII. According to BioMarin, a single dose of Roctavian can maintain effective levels of Factor VIII, a protein that is deficient in patients with severe hemophilia, per data from clinical studies. The current standard of care for these patients is lifelong injectable therapy to help maintain enough clotting factors in the bloodstream to prevent bleeds.
If EC approves Roctavian, it will bring access to more than 20,000 severe hemophilia A patients across Europe, the Middle East and Africa.
Shares of BioMarin have declined 1.7% in the year-to-date period compared with the industry’s 21.2% decrease.
In the United States, BioMarin had initially filed a biologics license application (BLA) seeking approval for valoctocogene roxaparvovec as a potential treatment for hemophilia A, in 2020. However, the FDA issued a complete response letter (CRL) in August 2020. The regulatory authority requested additional data on the annualized bleed rates from the GENEr8-1 study to provide more evidence of a long-lasting effect.
Back then, BioMarin also withdrew its MAA filed in Europe. BioMarin resubmitted the MAA and its new one-year data to the European Medicines Agency (EMA) in June 2021. BioMarin announced two-year follow-up safety and efficacy data from the phase III GENEr8-1 study in January 2022 and submitted the same to the EMA.
BioMarin plans to re-fille the BLA with the FDA in June 2022. But last month, the company announced that they had decided to delay the BLA resubmission for Roctavian.
The management took the decision based on the FDA’s request for additional information and data analyses to be included in the BLA before resubmission. The BLA is now expected to be re-filled by September end.
Aridis Pharmaceuticals’ loss per share estimates for 2022 have narrowed from $1.78 to 34 cents in the past 30 days. The same for 2023 has narrowed from 75 cents to 60 cents in the same time frame. Shares of ARDS have plunged 46.3% in the year-to-date period.
Earnings of Aridis missed estimates in two of the trailing four quarters and beat the same on the remaining two occasions, the average negative surprise being 75.16%.
Anavex Life Sciences’ loss per share estimates for 2022 have narrowed from 85 cents to 60 cents in the past 30 days. Shares of AVXL have plunged 41.3% year to date.
Earnings of Anavex beat estimates in two of the last four quarters and missed the mark on the other two occasions, the average surprise being 0.48%.
Scholar Rock’sloss per share estimates for 2022 have narrowed from $2.94 to $2.59 in the past 30 days. The same for 2023 has narrowed from $3.27 cents to $2.85 in the same time frame. Shares of SRRK have plunged 76% in the year-to-date period.
Earnings of Scholar Rock missed estimates in two of the trailing four quarters and beat the same on the remaining two occasions, the average surprise being 13.12%.
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BioMarin (BMRN) Gets CHMP Nod for Hemophilia Drug Roctavian
BioMarin Pharmaceutical Inc. (BMRN - Free Report) receives a positive opinion from the Committee for Medicinal Products for Human Use (“CHMP”), recommending a conditional marketing authorization (CMA) for its gene therapy candidate, valoctocogene roxaparvovec, to treat adults with severe hemophilia A.
The company is awaiting the final decision from the European Commission (EC), typically coherent with the CHMP recommendation, expected by third-quarter 2022.
The single-dose infusion gene therapy (valoctocogene roxaparvovec) will be marketed with the tradename of Roctavian. The treatment will be available for adult patients with severe hemophilia A who were not previously treated with factor VII inhibitors and do not have detectable antibodies to adeno-associated virus serotype 5. Roctavian will be the first gene therapy for patients with hemophilia A in Europe following a potential conditional approval.
Hemophilia A is a hereditary bleeding disorder caused by the lack of blood clotting Factor VIII. According to BioMarin, a single dose of Roctavian can maintain effective levels of Factor VIII, a protein that is deficient in patients with severe hemophilia, per data from clinical studies. The current standard of care for these patients is lifelong injectable therapy to help maintain enough clotting factors in the bloodstream to prevent bleeds.
If EC approves Roctavian, it will bring access to more than 20,000 severe hemophilia A patients across Europe, the Middle East and Africa.
Shares of BioMarin have declined 1.7% in the year-to-date period compared with the industry’s 21.2% decrease.
BioMarin Pharmaceutical Inc. Price and Consensus
BioMarin Pharmaceutical Inc. price-consensus-chart | BioMarin Pharmaceutical Inc. Quote
In the United States, BioMarin had initially filed a biologics license application (BLA) seeking approval for valoctocogene roxaparvovec as a potential treatment for hemophilia A, in 2020. However, the FDA issued a complete response letter (CRL) in August 2020. The regulatory authority requested additional data on the annualized bleed rates from the GENEr8-1 study to provide more evidence of a long-lasting effect.
Back then, BioMarin also withdrew its MAA filed in Europe. BioMarin resubmitted the MAA and its new one-year data to the European Medicines Agency (EMA) in June 2021. BioMarin announced two-year follow-up safety and efficacy data from the phase III GENEr8-1 study in January 2022 and submitted the same to the EMA.
BioMarin plans to re-fille the BLA with the FDA in June 2022. But last month, the company announced that they had decided to delay the BLA resubmission for Roctavian.
The management took the decision based on the FDA’s request for additional information and data analyses to be included in the BLA before resubmission. The BLA is now expected to be re-filled by September end.
Zacks Rank & Stocks to Consider
BioMarin currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the same sector are Aridis Pharmaceuticals (ARDS - Free Report) , Anavex Life Sciences (AVXL - Free Report) and Scholar Rock (SRRK - Free Report) , each carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Aridis Pharmaceuticals’ loss per share estimates for 2022 have narrowed from $1.78 to 34 cents in the past 30 days. The same for 2023 has narrowed from 75 cents to 60 cents in the same time frame. Shares of ARDS have plunged 46.3% in the year-to-date period.
Earnings of Aridis missed estimates in two of the trailing four quarters and beat the same on the remaining two occasions, the average negative surprise being 75.16%.
Anavex Life Sciences’ loss per share estimates for 2022 have narrowed from 85 cents to 60 cents in the past 30 days. Shares of AVXL have plunged 41.3% year to date.
Earnings of Anavex beat estimates in two of the last four quarters and missed the mark on the other two occasions, the average surprise being 0.48%.
Scholar Rock’sloss per share estimates for 2022 have narrowed from $2.94 to $2.59 in the past 30 days. The same for 2023 has narrowed from $3.27 cents to $2.85 in the same time frame. Shares of SRRK have plunged 76% in the year-to-date period.
Earnings of Scholar Rock missed estimates in two of the trailing four quarters and beat the same on the remaining two occasions, the average surprise being 13.12%.