BioMarin Resubmits Marketing Authorization Application (MAA) to European Medicines Agency for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A

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SAN RAFAEL, CALIFORNIA., June 28, 2021 / PRNewswire / – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that it has re-submitted a MAA from the European Medicines Agency (EMA) for its investigational therapy Valoctocogen roxaparvovec for adults with severe hemophilia A. submitted. in the May 2021, the EMA has granted the company’s accelerated assessment request. An accelerated assessment may shorten the time frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and the Committee on Advanced Therapies (CAT) to consider an Advanced Therapy Medicinal Product (ATMP). A CHMP opinion is expected in the first half of 2022.

The application includes data on the safety and efficacy of the 134 study participants in the phase 3 GENEr8-1 study, all of whom were observed for at least one year after treatment with Valoctocogen Roxaparvovec, as well as four and three years of follow-up from the dose cohorts 6e13 vg / kg and 4e13 vg / kg in the ongoing phase 1/2 dose escalation study.

“This is an important step in delivering what is potentially the first gene therapy for people with haemophilia A. The data package for this submission includes the largest phase 3 study in gene therapy for hemophilia A, supported by up to four years of observation in a phase 1 study. “/ 2 study,” said Hank Fuchs, MD, President, Global Research and Development at BioMarin. “We look forward to the EMA reviewing the robust dataset. We believe Valoctocogene Roxaparvovec represents a scientific innovation that could become an important treatment option for people with unmet medical needs.”

“This application is important to the bleeding disorder community as it requires continued scientific and medical innovation to provide a treatment option to meet the unmet needs of many people with Haemophilia A,” said Brian O Mahony FACSLM, Chief Executive of the Irish Haemophilia Society . “We believe that therapeutic choices are critical to our community in order to provide access to alternative treatment approaches with the potential to meet individual patient needs.”

Applications will be eligible for expedited evaluation if the CHMP and CAT decide that the product is of great public health interest, particularly with regard to therapeutic innovations. It can take up to 210 days to evaluate an application for authorization under the EMA’s centralized process, not counting the stops when applicants are asked to provide additional information. Upon request, the CHMP and CAT may reduce the timeframe to 150 days if the applicant provides sufficient reasons for an accelerated evaluation, although an application originally intended for accelerated evaluation may, for various reasons, fall back on the standard procedure during the review. The decision to give an expedited assessment does not affect the possible opinion of the CHMP and CAT on whether a marketing authorization should be granted.

Regulatory status

in the The United States, BioMarin intends to provide two-year follow-up safety and efficacy data for all participants in the Phase 3 GENEr8-1 study to support the benefit-risk assessment of Valoctocogen Roxaparvovec, as previously approved by the Food and Drug Administration ( FDA) required. . BioMarin is aiming to re-submit the Biologics License Application (BLA) in the second quarter of 2022, assuming positive study results, followed by an expected six-month review by the FDA.

The FDA granted the valoctocogen Roxaparvovec Regenerative Medicine Advanced Therapy (RMAT) status. March 2021. RMAT is an accelerated program designed to facilitate the development and review of regenerative medicine therapies, such as Valoctocogene Roxaparvovec, that are expected to address an unmet medical need in patients with severe illness. The RMAT marking complements the Breakthrough Therapy marking that the company received in 2017.

In addition to the RMAT designation and the Breakthrough Therapy Designation, BioMarin’s Valoctocogen Roxaparvovec has also received orphan drug status from the FDA and EMA for the treatment of severe hemophilia A promise for the diagnosis and / or treatment of rare diseases or conditions.

Robust clinical program

BioMarin has several clinical trials underway as part of its comprehensive gene therapy program for the treatment of hemophilia A. In addition to the global phase 3 study GENEr8-1 and the ongoing phase 1/2 dose escalation study, the company is actively recruiting participants for a phase 3b, single-arm, open-label study to evaluate the efficacy and safety of Valoctocogen Roxaparvovec at a dose of 6e13 µg / kg with prophylactic corticosteroids in patients with haemophilia A. The company is also conducting a phase 1/2 study with 6e13 µg / kg dose Valoctocogen Roxaparvovec in patients with haemophilia A with pre-existing AAV5 antibodies and another phase 1/2 study with the dose of 6e13 µg / kg Valoctocogen Roxaparvovec in patients with haemophilia A with active or previous FVIII inhibitors.

About hemophilia A.

People living with haemophilia A lack enough functioning Factor VIII protein to help their blood clot, and they are at risk of painful and / or potentially life-threatening bleeding, even from minor injuries. In addition, people with the most severe form of haemophilia A (FVIII level <1%) often experience painful, spontaneous bleeding in muscles or joints. Individuals with the most severe form of hemophilia A make approximately 45 percent the hemophilia A population. People with haemophilia A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease have a greatly reduced tendency to bleed. The standard of care for adults with severe haemophilia A is prophylactic treatment with replacement factor VIII infusions given intravenously up to two to three times per week or 100 to 150 infusions per year. Despite these therapies, many people continue to experience breakthrough bleeding, which leads to progressive and debilitating joint damage that can severely affect their quality of life.

Hemophilia A, also known as factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective factor VIII, a coagulation protein. Although it is passed on from parents to children, about 1/3 of the cases are caused by a spontaneous mutation, a new mutation that was not inherited. About 1 in 10,000 people has haemophilia A.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for severe and life-threatening rare and extremely rare genetic diseases. The company’s portfolio consists of six commercialized products and several clinical and preclinical product candidates. More information is available at Information on BioMarin’s website is not incorporated by reference in this press release.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, but not limited to, statements about (i) the development of BioMarin’s Valoctocogen-Roxaparvovec program in general, (ii) the anticipated timing of a CHMP meeting Opinion in the first half of 2022, (iii) BioMarin’s intention to provide the US Food and Drug Administration (FDA) with two-year follow-up data on the safety and efficacy of all study participants from the GENEr8-1 study in order to facilitate the benefit-risk assessment to be supported by Valoctocogene roxaparvovec, (iv) BioMarin with the aim of resubmitting a registration application for biologics in the second quarter of 2022 assuming positive study results, followed by an expected six-month review process by the FDA, and (v) the timing of regulatory activities in the US and Europeincluding the validation and timing of potential permits and expected review procedures. These forward-looking statements are predictions and involve risks and uncertainties, so that actual results could differ materially from these statements. These risks and uncertainties include, but are not limited to: the results and timing of current and planned preclinical and clinical studies with Valoctocogen Roxaparvovec; additional data from the continuation of the Phase 1/2 study and the Phase 3 study, any possible adverse events observed during the ongoing monitoring of the participants in the clinical studies; The content and timing of the decisions made by the FDA, the European Commission, and other regulatory authorities; Content and timing of decisions by local and central ethics committees regarding clinical trials; our ability to successfully manufacture Valoctocogene Roxaparvovec for clinical trials and if approved, commercially; and the other risks listed from time to time under the heading “Risk Factors” and elsewhere in BioMarin’s Securities and Exchange Commission (SEC) filings, including BioMarin’s Quarterly Report on Form 10-Q for the past quarter March 31, 2021, as well as future filings and reports from BioMarin. BioMarin assumes no obligation or obligation to update any forward-looking statements contained in this press release as a result of new information, future events, or changes in its expectations.

BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.




Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

SOURCE BioMarin Pharmaceutical Inc.

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