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BioMarin's (BMRN) Hemophilia Gene Therapy Gets Nod in EU
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BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that the European Commission (EC) has granted conditional approval to Roctavian (valoctocogene roxaparvovec, or valrox) gene therapy for treating adult patients with severe hemophilia A (congenital Factor VIII deficiency).
The EC has granted conditional marketing authorization (CMA) to use Roctavian in adults who have not been treated with Factor VIII inhibitors and do not have detectable antibodies to adeno-associated virus serotype 5 (AAV5).
The approval follows a positive review by the Committee for Medicinal Products for Human Use (CHMP), which recommended the approval of the gene therapy in June. The decision by the EC and CHMP was based on two-year outcomes data fromthe phase III study, GENEr8-1, evaluating Roctavian in patients with hemophilia A.
Data from the GENEr8-1 study showed that treatment with Roctavianled to stable and durable bleed control. In fact, participants who were treated with the therapy achieved significantly reduced annualized bleeding rate and the mean annualized factor VIII (a blood clotting protein) infusion rate. Treatment with Roctavian also demonstrated superiority to the current standard of care, Factor VIIIprophylactic therapy.
Shares of BioMarin have risen 5.2% in the year-to-date period against the industry’s 22.3% decline.
Image Source: Zacks Investment Research
The EC decision makes Roctavian the first approved gene therapy for hemophilia A. Per the EC, the use of Roctavian carries significant benefits over currently marketed therapies in treating hemophilia A. In fact, the EC has also endorsed the EMA’s recommendation and granted Roctavian orphan drug designation (ODD). Following the grant of ODD, the drug now has a 10-year market exclusivity in the European Union.
A one-time infusion, Roctavian is designed to enable the body to produce Factor VIII on its own. Moreover, the patients are also not required to be administered a continued hemophilia prophylaxis, which in other marketed therapies is required.
In the United States, BMRN had previously submitted a biologics license application (BLA) in 2019 for valrox to address hemophilia A. However, the FDA issued a complete response letter (CRL) to the BLA ahead of the PDUFA date in August 2020. This was due to the regulatory agency’s dissatisfaction with the available data. As a result, it asked for two-year follow-up information on the annualized bleed rates from the GENEr8-1 study only to provide additional evidence of a durable effect. BioMarin expects the BLA to be refiled next month.
BioMarin currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the overall healthcare sector include Assertio (ASRT - Free Report) , Morphic (MORF - Free Report) and Sesen Bio . While Morphic and Sesen Bio sport a Zacks Rank #1 (Strong Buy) at present, Assertio carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Estimates for Sesen Bio’s 2023 bottom line have narrowed from 27 cents to 1 cent in the past 30 days. Share prices of Sesen Bio have fallen 19.4% in the year-to-date period.
Earnings of Sesen Bio beat estimates in each of the last four quarters, the average surprise being 89.49%. In the last reported quarter, Sesen Bio delivered an earnings surprise of 61.54%.
In the past 30 days, estimates for Morphic’s 2022 loss per share have narrowed from $3.38 to $1.75. Loss estimates for 2023 have narrowed from $3.91 to $3.77 during the same period. Shares of Morphic have lost 33.7% in the year-to-date period.
Earnings of Morphic beat estimates in three of the last four quarters and missed the mark just once, witnessing a surprise of 48.29%, on average. In the last reported quarter, MORF delivered an earnings surprise of 183.95%.
In the past 30 days, estimates for Assertio’s 2022 earnings per share have risen from 40 cents to 49 cents. Earnings estimates for 2023 have risen from 29 cents to 30 cents during the same period. Shares of Assertio have risen 32.6% in the year-to-date period.
Earnings of Assertio beat estimates in three of the last four quarters and missed the mark just once, witnessing a surprise of 126.39%, on average. In the last reported quarter, ASRT delivered an earnings surprise of 100.00%.
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BioMarin's (BMRN) Hemophilia Gene Therapy Gets Nod in EU
BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that the European Commission (EC) has granted conditional approval to Roctavian (valoctocogene roxaparvovec, or valrox) gene therapy for treating adult patients with severe hemophilia A (congenital Factor VIII deficiency).
The EC has granted conditional marketing authorization (CMA) to use Roctavian in adults who have not been treated with Factor VIII inhibitors and do not have detectable antibodies to adeno-associated virus serotype 5 (AAV5).
The approval follows a positive review by the Committee for Medicinal Products for Human Use (CHMP), which recommended the approval of the gene therapy in June. The decision by the EC and CHMP was based on two-year outcomes data fromthe phase III study, GENEr8-1, evaluating Roctavian in patients with hemophilia A.
Data from the GENEr8-1 study showed that treatment with Roctavianled to stable and durable bleed control. In fact, participants who were treated with the therapy achieved significantly reduced annualized bleeding rate and the mean annualized factor VIII (a blood clotting protein) infusion rate. Treatment with Roctavian also demonstrated superiority to the current standard of care, Factor VIIIprophylactic therapy.
Shares of BioMarin have risen 5.2% in the year-to-date period against the industry’s 22.3% decline.
Image Source: Zacks Investment Research
The EC decision makes Roctavian the first approved gene therapy for hemophilia A. Per the EC, the use of Roctavian carries significant benefits over currently marketed therapies in treating hemophilia A. In fact, the EC has also endorsed the EMA’s recommendation and granted Roctavian orphan drug designation (ODD). Following the grant of ODD, the drug now has a 10-year market exclusivity in the European Union.
A one-time infusion, Roctavian is designed to enable the body to produce Factor VIII on its own. Moreover, the patients are also not required to be administered a continued hemophilia prophylaxis, which in other marketed therapies is required.
In the United States, BMRN had previously submitted a biologics license application (BLA) in 2019 for valrox to address hemophilia A. However, the FDA issued a complete response letter (CRL) to the BLA ahead of the PDUFA date in August 2020. This was due to the regulatory agency’s dissatisfaction with the available data. As a result, it asked for two-year follow-up information on the annualized bleed rates from the GENEr8-1 study only to provide additional evidence of a durable effect. BioMarin expects the BLA to be refiled next month.
BioMarin Pharmaceutical Inc. Price
BioMarin Pharmaceutical Inc. price | BioMarin Pharmaceutical Inc. Quote
Zacks Rank & Stocks to Consider
BioMarin currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the overall healthcare sector include Assertio (ASRT - Free Report) , Morphic (MORF - Free Report) and Sesen Bio . While Morphic and Sesen Bio sport a Zacks Rank #1 (Strong Buy) at present, Assertio carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Estimates for Sesen Bio’s 2023 bottom line have narrowed from 27 cents to 1 cent in the past 30 days. Share prices of Sesen Bio have fallen 19.4% in the year-to-date period.
Earnings of Sesen Bio beat estimates in each of the last four quarters, the average surprise being 89.49%. In the last reported quarter, Sesen Bio delivered an earnings surprise of 61.54%.
In the past 30 days, estimates for Morphic’s 2022 loss per share have narrowed from $3.38 to $1.75. Loss estimates for 2023 have narrowed from $3.91 to $3.77 during the same period. Shares of Morphic have lost 33.7% in the year-to-date period.
Earnings of Morphic beat estimates in three of the last four quarters and missed the mark just once, witnessing a surprise of 48.29%, on average. In the last reported quarter, MORF delivered an earnings surprise of 183.95%.
In the past 30 days, estimates for Assertio’s 2022 earnings per share have risen from 40 cents to 49 cents. Earnings estimates for 2023 have risen from 29 cents to 30 cents during the same period. Shares of Assertio have risen 32.6% in the year-to-date period.
Earnings of Assertio beat estimates in three of the last four quarters and missed the mark just once, witnessing a surprise of 126.39%, on average. In the last reported quarter, ASRT delivered an earnings surprise of 100.00%.