SAN RAFAEL, Calif. - BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that its gene therapy product, ROCTAVIAN® (valoctocogene roxaparvovec-rvox), has maintained its efficacy in preventing bleeding in patients with severe hemophilia A for four years post-treatment.
The data, derived from the Phase 3 GENEr8-1 trial, will be presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis in Bangkok, Thailand, scheduled for June 22-26, 2024.
The trial results showed that 73.6% of participants experienced zero treated bleeds in the fourth year. Additionally, the average annualized bleeding rate (ABR) for treated bleeds was 0.8 per year, and for all bleeds was 1.3 per year. The therapy was also associated with a reduction in the need for factor VIII infusions, with a mean annualized infusion rate of 6.1 post-prophylaxis cessation.
In terms of safety, no new signals were observed, and factor VIII levels remained stable compared to the three-year data. These findings suggest that ROCTAVIAN could offer an alternative to the current standard of care, which typically involves frequent infusions.
The study also assessed the impact of ROCTAVIAN on health-related quality of life (HRQoL) using the Haemo-QOL-A assessment. Results indicated improvements in HRQoL scores, particularly in areas related to physical and role functioning, as well as bleeding consequences.
ROCTAVIAN received conditional marketing authorization from the European Commission on August 24, 2022, and approval from the U.S. Food and Drug Administration on June 29, 2023. It is designed for adults with severe hemophilia A who lack antibodies to adeno-associated virus serotype 5 (AAV5).
The therapy works by delivering a functional gene that enables the body to produce factor VIII on its own, potentially reducing the need for ongoing prophylaxis. However, it is contraindicated in patients with certain conditions, such as active infections or significant hepatic fibrosis, and those with known hypersensitivity to mannitol.
This news is based on a press release statement.
InvestingPro Insights
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) has shown promising results with its gene therapy product, ROCTAVIAN®, which is making waves in the treatment of severe hemophilia A. From an investor's perspective, the company's financial health and market performance are key factors to consider alongside these medical advancements.
InvestingPro data reveals that BioMarin has a market capitalization of 15.33 billion USD, indicating a robust presence in the biopharmaceutical industry. The company's P/E ratio stands at 74.17, which, while high, is seen in a different light when considering the near-term earnings growth, as suggested by one of the InvestingPro Tips. The adjusted P/E ratio for the last twelve months as of Q1 2024 is slightly lower at 69.64, reflecting a more favorable valuation in the eyes of some investors.
The revenue growth also presents a positive outlook, with a 13.74% increase over the last twelve months as of Q1 2024. This financial metric underscores the company's ability to increase its earnings over time, which could be a testament to the success of its products and operational efficiency.
Investors looking for short-term gains might be interested in another InvestingPro Tip, which notes a significant return over the last week, with a 7.7% price total return. This could be indicative of the market's reaction to recent developments, such as the promising data from the ROCTAVIAN® trial.
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