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BioMarin (BMRN) Hemophilia Gene Therapy BLA Gets FDA Acceptance
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BioMarin Pharmaceutical Inc. (BMRN - Free Report) recently announced that the FDA has accepted the resubmitted biologics license application (BLA) for its investigational AAV gene therapy, valoctocogene roxaparvovec to treat adult patients with severe hemophilia A.
A decision from the regulatory body is expected on Mar 31, 2023. Currently, the FDA is not planning to hold an advisory committee meeting.
If approved, valoctocogene roxaparvovec would become the first gene therapy for treating severe hemophilia A in the United States.
Last month, BMRN resubmitted the BLA for valoctocogene roxaparvovec to treat adult patients with severe hemophilia A.
The resubmitted BLA was based on two-year outcomes data from the phase III study, GENEr8-1, evaluating valoctocogene roxaparvovec in patients with hemophilia A. Data from the GENEr8-1 study showed that treatment with valoctocogene roxaparvovec led to a stable and durable bleed control. Participants, who were treated with the gene therapy, achieved significantly reduced annualized bleeding rate and the mean annualized factor VIII (a blood clotting protein) infusion rate. Treatment with the gene therapy also demonstrated superiority to the current standard of care, Factor VIII prophylactic therapy.
BioMarin had submitted a BLA in 2019 for valoctocogene roxaparvovec for hemophilia A. However, the FDA issued a complete response letter to the BLA ahead of the PDUFA date in August 2020 as the FDA was not satisfied with the available data and asked for two-year follow-up data on annualized bleed rates from the ongoing phase III study to provide additional evidence of a durable effect.
Shares of BioMarin have risen 0.6% in the year-to-date period against the industry’s 27.8% decline.
Image Source: Zacks Investment Research
A one-time infusion, valoctocogene roxaparvovec is designed to enable the body to produce Factor VIII on its own. Moreover, the patients are also not required to be administered continued hemophilia prophylaxis, which is required in other marketed therapies.
In August 2022, the European Commission granted conditional approval to valoctocogene roxaparvovec for treating adult patients with severe hemophilia A. The therapy is being marketed by BioMarin under the trade name Roctavian.
Loss per share estimates for Acadia have narrowed 0.8% for 2022 and 11.7% for 2023 in the past 60 days.
Earnings of Acadia surpassed estimates in two of the trailing four quarters and missed on the remaining two occasions. ACAD delivered an earnings surprise of 6.83%, on average.
Loss per share estimates for ORIC Pharmaceuticals have narrowed 5.9% for 2022 and 7.3% for 2023 in the past 60 days.
Earnings of ORIC Pharmaceuticals surpassed estimates in three of the trailing four quarters and missed on the other occasion. ORIC delivered an earnings surprise of 8.85%, on average.
Loss per share estimates for Atara Biotherapeutics have narrowed 21.3% for 2022 and 8.3% 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.
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BioMarin (BMRN) Hemophilia Gene Therapy BLA Gets FDA Acceptance
BioMarin Pharmaceutical Inc. (BMRN - Free Report) recently announced that the FDA has accepted the resubmitted biologics license application (BLA) for its investigational AAV gene therapy, valoctocogene roxaparvovec to treat adult patients with severe hemophilia A.
A decision from the regulatory body is expected on Mar 31, 2023. Currently, the FDA is not planning to hold an advisory committee meeting.
If approved, valoctocogene roxaparvovec would become the first gene therapy for treating severe hemophilia A in the United States.
Last month, BMRN resubmitted the BLA for valoctocogene roxaparvovec to treat adult patients with severe hemophilia A.
The resubmitted BLA was based on two-year outcomes data from the phase III study, GENEr8-1, evaluating valoctocogene roxaparvovec in patients with hemophilia A. Data from the GENEr8-1 study showed that treatment with valoctocogene roxaparvovec led to a stable and durable bleed control. Participants, who were treated with the gene therapy, achieved significantly reduced annualized bleeding rate and the mean annualized factor VIII (a blood clotting protein) infusion rate. Treatment with the gene therapy also demonstrated superiority to the current standard of care, Factor VIII prophylactic therapy.
BioMarin had submitted a BLA in 2019 for valoctocogene roxaparvovec for hemophilia A. However, the FDA issued a complete response letter to the BLA ahead of the PDUFA date in August 2020 as the FDA was not satisfied with the available data and asked for two-year follow-up data on annualized bleed rates from the ongoing phase III study to provide additional evidence of a durable effect.
Shares of BioMarin have risen 0.6% in the year-to-date period against the industry’s 27.8% decline.
Image Source: Zacks Investment Research
A one-time infusion, valoctocogene roxaparvovec is designed to enable the body to produce Factor VIII on its own. Moreover, the patients are also not required to be administered continued hemophilia prophylaxis, which is required in other marketed therapies.
In August 2022, the European Commission granted conditional approval to valoctocogene roxaparvovec for treating adult patients with severe hemophilia A. The therapy is being marketed by BioMarin under the trade name Roctavian.
Zacks Rank & Other Stocks to Consider
BioMarin currently carries a Zacks Rank #2 (Buy). Other stocks worth considering in the biotech sector are Acadia Pharmaceuticals Inc. (ACAD - Free Report) , ORIC Pharmaceuticals, Inc. (ORIC - Free Report) and Atara Biotherapeutics, Inc. (ATRA - Free Report) , all carrying a Zacks Rank #2 at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Loss per share estimates for Acadia have narrowed 0.8% for 2022 and 11.7% for 2023 in the past 60 days.
Earnings of Acadia surpassed estimates in two of the trailing four quarters and missed on the remaining two occasions. ACAD delivered an earnings surprise of 6.83%, on average.
Loss per share estimates for ORIC Pharmaceuticals have narrowed 5.9% for 2022 and 7.3% for 2023 in the past 60 days.
Earnings of ORIC Pharmaceuticals surpassed estimates in three of the trailing four quarters and missed on the other occasion. ORIC delivered an earnings surprise of 8.85%, on average.
Loss per share estimates for Atara Biotherapeutics have narrowed 21.3% for 2022 and 8.3% 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.