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BioMarin (BMRN) Gets FDA Nod for Hemophilia A Gene Therapy
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BioMarin (BMRN - Free Report) announced that the FDA has approved Roctavian (valoctocogene roxaparvovec or valrox), an adeno-associated virus-based gene therapy to treat adult patients with severe hemophilia A.
Following the FDA’s approval, Roctavian is the first one-shot gene therapy approved for treating severe hemophilia A in adults. The gene therapy was granted conditional approval in the European Union for adults last August.
Multiple reports suggest that BioMarin will price Roctavian at $2.9 million.
The FDA approval is based on three-year follow-up safety and efficacy data from the phase III GENEr8-1 study, which evaluated Roctavian in adults aged 18 through 70 with severe hemophilia A. Study participants, who were administered the gene therapy, experienced a mean annualized bleeding rate (“ABR”) reduction of 52% compared with those who were administered the current standard-of-care (“SOC”) of treatment, i.e., a prophylaxis regimen with Factor VIII (“FVIII”).
In the year so far, BioMarin’s shares have lost 14.1% compared with the industry’s 9.8% decline.
Image Source: Zacks Investment Research
A rare genetic blood disorder, hemophilia B is caused by a missing or defective clotting protein FVIII, which prevents normal blood clotting. If the blood does not clot properly, it can lead to painful bleeding inside the joints that can cause scarring and damage.
Roctavian has been designed to deliver a functional gene that enables the body to produce the FVIII protein on its own, reducing the need for ongoing prophylaxis.
Similar to Roctavian, Pfizer (PFE - Free Report) and partner Sangamo Therapeutics (SGMO - Free Report) are developing their own gene therapy — giroctocogene fitelparvovec — to treat hemophilia A. Sangamo and Pfizer are evaluating giroctocogene fitelparvovec in the phase III AFFINE study. Pfizer/Sangamo expects to report pivotal data from the AFFINE study by mid-2024. Based on this data, Sangamo/Pfizer expects to start regulatory submissions for the gene therapy candidate in Europe and the United States during second-half 2024.
In the past 60 days, the estimate for AbbVie’s 2023 and 2024 EPS have increased from $10.95 to $10.99 and $11.02 to $11.04, respectively. Shares of AbbVie are down 17.6% in the year-to-date period.
Earnings of AbbVie beat estimates in each of the last four quarters, witnessing an average earnings surprise of 1.78%.
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BioMarin (BMRN) Gets FDA Nod for Hemophilia A Gene Therapy
BioMarin (BMRN - Free Report) announced that the FDA has approved Roctavian (valoctocogene roxaparvovec or valrox), an adeno-associated virus-based gene therapy to treat adult patients with severe hemophilia A.
Following the FDA’s approval, Roctavian is the first one-shot gene therapy approved for treating severe hemophilia A in adults. The gene therapy was granted conditional approval in the European Union for adults last August.
Multiple reports suggest that BioMarin will price Roctavian at $2.9 million.
The FDA approval is based on three-year follow-up safety and efficacy data from the phase III GENEr8-1 study, which evaluated Roctavian in adults aged 18 through 70 with severe hemophilia A. Study participants, who were administered the gene therapy, experienced a mean annualized bleeding rate (“ABR”) reduction of 52% compared with those who were administered the current standard-of-care (“SOC”) of treatment, i.e., a prophylaxis regimen with Factor VIII (“FVIII”).
In the year so far, BioMarin’s shares have lost 14.1% compared with the industry’s 9.8% decline.
Image Source: Zacks Investment Research
A rare genetic blood disorder, hemophilia B is caused by a missing or defective clotting protein FVIII, which prevents normal blood clotting. If the blood does not clot properly, it can lead to painful bleeding inside the joints that can cause scarring and damage.
Roctavian has been designed to deliver a functional gene that enables the body to produce the FVIII protein on its own, reducing the need for ongoing prophylaxis.
Similar to Roctavian, Pfizer (PFE - Free Report) and partner Sangamo Therapeutics (SGMO - Free Report) are developing their own gene therapy — giroctocogene fitelparvovec — to treat hemophilia A. Sangamo and Pfizer are evaluating giroctocogene fitelparvovec in the phase III AFFINE study. Pfizer/Sangamo expects to report pivotal data from the AFFINE study by mid-2024. Based on this data, Sangamo/Pfizer expects to start regulatory submissions for the gene therapy candidate in Europe and the United States during second-half 2024.
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). A better-ranked stock in the overall healthcare sector is AbbVie (ABBV - Free Report) , which also carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 60 days, the estimate for AbbVie’s 2023 and 2024 EPS have increased from $10.95 to $10.99 and $11.02 to $11.04, respectively. Shares of AbbVie are down 17.6% in the year-to-date period.
Earnings of AbbVie beat estimates in each of the last four quarters, witnessing an average earnings surprise of 1.78%.