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BioMarin Announces Advances in FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A


The FDA no longer plans to hold an advisory committee meeting, as previously scheduled, to discuss the Biologics License Application (BLA)

BioMarin remains on track to conduct scheduled FDA manufacturing inspections in the coming weeks

SAN RAFAEL, Calif., November 23, 2022 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced progress in the U.S. Food and Drug Administration’s (FDA) review of the Biologics License Application (BLA) for ROCTAVIAN™ (gene therapy valoctocogene roxaparvovec AAV) for adults with severe hemophilia R. The company was recently advised by the FDA that after further review, at this time the agency no longer plans to hold an advisory committee meeting to discuss of the BLA for ROCTAVIAN which is currently under review. Previously, the FDA communicated to the Company that it intended to hold an advisory committee meeting but did not specify a date. The company also remains on track to host the FDA’s planned pre-clearance inspection (PLI) of BioMarin’s gene therapy manufacturing facility located in NovatoCALIFORNIA.

“Reviewing a BLA is a dynamic process, and we appreciate the FDA’s continued commitment as we strive to provide a potentially transformative treatment choice for patients with severe hemophilia A,” said Hank Fuchs, MD, president of global research and development at BioMarin. “We look forward to continuing the dialogue with the Agency as they review our application.”

About valoctocogene roxaparvovec (ROCTAVIAN™)

The FDA granted RMAT (Regenerative Medicine Advanced Therapy) designation to valoctocogene roxaparvovec in March 2021. RMAT is an accelerated program intended to facilitate the development and investigation of regenerative medicine therapies, such as valoctocogene roxaparvovec, which are expected to address an unmet medical need in patients with serious diseases. The RMAT designation is complementary to the Breakthrough Therapy designation, which the Company received for valoctocogene roxaparvovec in 2017.

In addition to RMAT designation and Breakthrough Therapy designation, BioMarin’s valoctocogene roxaparvovec has also received orphan drug designation from the EMA and FDA for the treatment of severe hemophilia A. Orphan drug designation is reserved for drugs that treat rare, life-threatening, or chronically debilitating diseases. . The European Commission (EC) has granted conditional marketing authorization to valoctocogene gene therapy roxaparvovec under the brand name ROCTAVIAN™ on August 24, 2022.

Robust clinical program

BioMarin has several ongoing clinical studies in its comprehensive gene therapy program for the treatment of severe hemophilia A. In addition to the global Phase 3 GENEr8-1 study and the ongoing Phase 1/2 study, the company is also conducting a Phase 3, open-label, single-arm study to assess the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A (study 270-303). A phase 1/2 study is also underway with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with pre-existing AAV5 antibodies (study 270-203) and a phase 1/2 study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe haemophilia A with active or prior factor VIII inhibitors (study 270-205).

Security Summary

Overall, to date, a single dose of 6e13 vg/kg valoctocogene roxaparvovec has been well tolerated with no late-onset treatment-related adverse events. The most common adverse events (AEs) associated with valoctocogene roxaparvovec occurred early and included transient infusion-associated reactions and mild to moderate elevation of liver enzymes without long-term clinical sequelae. Elevated alanine aminotransferase (ALT), a laboratory test of liver function, remained the most common side effect. Other adverse events included aspartate aminotransferase (AST) elevation (101 participants, 63%), nausea (55 participants, 34%), headache (54 participants, 34%) and fatigue (44 attendees, 28%). No participants developed factor VIII inhibitors, thromboembolic events, or malignancy associated with valoctocogene roxaparvovec.

About Hemophilia A

People living with hemophilia A do not have enough functional factor VIII protein to help their blood clot and are at risk of painful and/or life-threatening bleeding from even minor injuries. In addition, people with the most severe form of hemophilia A (factor VIII levels

Hemophilia A, also called factor VIII deficiency or classic hemophilia, is an X-linked genetic disease caused by missing or defective factor VIII, a clotting protein. Although passed from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that has not been inherited. About 1 in 10,000 people have hemophilia A.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have a well-understood biology and offer an opportunity to be first to market or offer a significant advantage over existing products. The Company’s portfolio consists of eight commercial products and several clinical and preclinical product candidates for the treatment of various diseases. For more information, please visit

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 regarding: the planned FDA inspection of the valoctocogene roxaparvovec manufacturing facility , the FDA is planning an advisory committee meeting and the possibility of regulatory approval. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the results and timing of ongoing and planned preclinical studies and clinical trials of valoctocogene roxaparvovec; additional data from ongoing clinical trials of valoctocogene roxaparvovec, including the 3-year results of the phase 3 GENEr8-1 study; any potential adverse events observed during ongoing monitoring of clinical trial participants; the content and timing of decisions by the FDA and other regulatory authorities, particularly with respect to inspection and determination of any additional submissions as “major changes”; the possibility for the FDA to require a meeting of the advisory committee, in particular after receipt of the additional data requested; and factors detailed in BioMarin’s filings with the Securities and Exchange Commission (SEC), including, without limitation, factors contained under the heading “Risk Factors” in BioMarin’s Quarterly Report on Form 10 -Q for the completed quarter September 30, 2022 as these factors may be updated by any subsequent report. Shareholders are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation and expressly disclaims any obligation to update or change any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc and ROCTAVIAN™ is a trademark of BioMarin Pharmaceutical Inc.




Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc..

(415) 455-7558

415) 455-7451

SOURCEBioMarin Pharmaceutical Inc.

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