BioMarin Resubmits Biologics License Software (BLA) for Valoctocogene Roxaparvovec AAV Gene Remedy for Extreme Hemophilia A to the FDA

BioMarin Resubmits Biologics License Software (BLA) for Valoctocogene Roxaparvovec AAV Gene Remedy for Extreme Hemophilia A to the FDA

BLA Consists of Substantial Physique of Knowledge from Pivotal Part 3 and Ongoing Part 1/2 Research

If Authorized, Would Be 1st Gene Remedy in U.S. for Remedy of Extreme Hemophilia A

SAN RAFAEL, Calif., Sept. 29, 2022 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) introduced at this time that the Firm resubmitted a Biologics License Software (BLA) to the U.S. Meals and Drug Administration (FDA) for its investigational AAV gene remedy, valoctocogene roxaparvovec, for adults with extreme hemophilia A. The resubmission incorporates the Firm’s response to the FDA Full Response (CR) Letter for valoctocogene roxaparvovec gene remedy issued on August 18, 2020, and subsequent suggestions, together with two-year outcomes from the worldwide GENEr8-1 Part 3 research and supportive information from 5 years of follow-up from the continuing Part 1/2 dose escalation research.

BioMarin anticipates an FDA response by the top of October on whether or not the BLA resubmission is full and acceptable for evaluate. Sometimes, BLA resubmissions are adopted by a six-month evaluate process. Nonetheless, the Firm anticipates three extra months of evaluate could also be vital based mostly on the variety of information read-outs that can emerge through the process. If accredited, valoctocogene roxaparvovec can be the primary commercially-available gene remedy within the U.S. for the therapy of extreme hemophilia A.

The FDA granted Regenerative Drugs Superior Remedy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program supposed to facilitate improvement and evaluate of regenerative medication therapies, similar to valoctocogene roxaparvovec, which might be anticipated to deal with an unmet medical want in sufferers with severe situations. The RMAT designation is complementary to Breakthrough Remedy Designation, which the Firm obtained for valoctocogene roxaparvovec in 2017.

Along with the RMAT Designation and Breakthrough Remedy Designation, BioMarin’s valoctocogene roxaparvovec additionally obtained orphan drug designation from the EMA and FDA for the therapy of extreme hemophilia A. Orphan drug designation is reserved for medicines treating uncommon, life-threatening or chronically debilitating illnesses. The European Fee (EC) granted conditional advertising authorization to valoctocogene roxaparvovec gene remedy underneath the model title ROCTAVIAN™ on August 24, 2022 and endorsed the advice from the European Medicines Company (EMA) to take care of orphan drug designation, thereby granting a 10-year interval of market exclusivity within the European Union.

“We’re happy to succeed in this level within the improvement program for valoctocogene roxaparvovec and look ahead to working with the FDA with the objective of bringing a doubtlessly transformative remedy to individuals with extreme hemophilia A in the USA,” stated Hank Fuchs, M.D., President of Worldwide Analysis and Growth at BioMarin. “This massive and sturdy information set supplied on this BLA resubmission exhibits an encouraging efficacy profile. We stay dedicated to sharing these information with the general public, together with even longer-term information generated by way of our ongoing medical trials and any post-approval research, to additional our understanding of AAV gene remedy in extreme hemophilia A and of gene therapies extra broadly.”

The resubmission features a substantial physique of information from the valoctocogene roxaparvovec medical improvement program, probably the most extensively studied gene remedy for extreme hemophilia A, together with two-year outcomes from the worldwide GENEr8-1 Part 3 research. The GENEr8-1 Part 3 research demonstrated steady and sturdy bleed management, together with a discount within the imply annualized bleeding fee (ABR) and the imply annualized Issue VIII infusion fee. As well as, the information bundle included supportive proof from 5 years of follow-up from the 6e13 vg/kg dose cohort within the ongoing Part 1/2 dose escalation research.  The resubmission additionally consists of a proposed long-term extension research following all medical trial individuals for as much as 15 years, in addition to two post-approval registry research. 

Sturdy Medical Program

BioMarin has a number of medical research underway in its complete gene remedy program for the therapy of extreme hemophilia A. Along with the worldwide Part 3 research GENEr8-1 and the continuing Part 1/2 dose escalation research, the Firm can also be conducting a Part 3, single arm, open-label research to judge the efficacy and security of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in individuals with extreme hemophilia A (Research 270-303). Additionally ongoing are a Part 1/2 Research with the 6e13 vg/kg dose of valoctocogene roxaparvovec in individuals with extreme hemophilia A with pre-existing AAV5 antibodies (Research 270-203) and a Part 1/2 Research with the 6e13 vg/kg dose of valoctocogene roxaparvovec in individuals with extreme hemophilia A with energetic or prior Issue VIII inhibitors (Research 270-205).

Security Abstract

General, thus far, a single 6e13 vg/kg dose of valoctocogene roxaparvovec has been effectively tolerated with no delayed-onset therapy associated opposed occasions. The commonest opposed occasions (AE) related to valoctocogene roxaparvovec have occurred early and included transient infusion related reactions and delicate to reasonable rise in liver enzymes with no long-lasting medical sequelae. Alanine aminotransferase (ALT) elevation, a laboratory check of liver operate, has remained the commonest opposed drug response. Different opposed reactions have included aspartate aminotransferase (AST) elevation (101 individuals, 63%), nausea (55 individuals, 34%), headache (54 individuals, 34%), and fatigue (44 individuals, 28%). No individuals have developed inhibitors to Issue VIII, thromboembolic occasions or malignancy related to valoctocogene roxaparvovec.

About Hemophilia A

Folks dwelling with hemophilia A scarcity ample functioning Issue VIII protein to assist their blood clot and are in danger for painful and/or doubtlessly life-threatening bleeds from even modest accidents. Moreover, individuals with probably the most extreme type of hemophilia A (Issue VIII ranges <1%) typically expertise painful, spontaneous bleeds into their muscle mass or joints.  People with probably the most extreme type of hemophilia A make up roughly 50 % of the hemophilia A inhabitants. Folks with hemophilia A with reasonable (Issue VIII 1-5%) or delicate (Issue VIII 5-40%) illness present a much-reduced propensity to bleed.  People with extreme hemophilia A are handled with a prophylactic routine of intravenous Issue VIII infusions administered 2-3 occasions per week (100-150 infusions per 12 months) or a bispecific monoclonal antibody that mimics the exercise of Issue VIII administered 1-4 occasions per 30 days (12-48 injections or photographs per 12 months). Regardless of these regimens, many individuals proceed to expertise breakthrough bleeds, leading to progressive and debilitating joint harm, which might have a significant influence on their high quality of life.

Hemophilia A, additionally known as Issue VIII deficiency or basic hemophilia, is an X-linked genetic dysfunction brought on by lacking or faulty Issue VIII, a clotting protein. Though it’s handed down from mother and father to kids, about 1/3 of circumstances are brought on by a spontaneous mutation, a brand new mutation that was not inherited. Roughly 1 in 10,000 individuals have hemophilia A.

About BioMarin

BioMarin is a world biotechnology firm that develops and commercializes modern therapies for individuals with severe and life-threatening genetic illnesses and medical situations. The Firm selects product candidates for illnesses and situations that symbolize a major unmet medical want, have well-understood biology and supply a possibility to be first-to-market or provide a major profit over current merchandise. The Firm’s portfolio consists of eight industrial merchandise and a number of medical and preclinical product candidates for the therapy of assorted illnesses. For extra info, please go to www.biomarin.com.

Ahead-Wanting Statements

This press launch comprises forward-looking statements concerning the enterprise prospects of BioMarin Pharmaceutical Inc. (BioMarin), together with with out limitation, statements about: BioMarin anticipating an FDA response by the top of October on whether or not the BLA resubmission is full and acceptable for evaluate, BioMarin’s expectations concerning the period of the evaluate process, valoctocogene roxaparvovec being the primary commercially-available gene remedy within the U.S. for the therapy of extreme hemophilia A, if accredited, BioMarin’s dedication to sharing longer-term information generated by way of its ongoing medical trials and any post-approval research.  These forward-looking statements are predictions and contain dangers and uncertainties such that precise outcomes could differ materially from these statements. These dangers and uncertainties embrace, amongst others: the outcomes and timing of present and deliberate preclinical research and medical trials of valoctocogene roxaparvovec; extra information from the continuation of the medical trials of valoctocogene roxaparvovec, any potential opposed occasions noticed within the persevering with monitoring of the individuals within the medical trials; the content material and timing of selections by the FDA and different regulatory authorities, together with choices to grant extra advertising registrations based mostly on an EMA license; the content material and timing of selections by native and central ethics committees concerning the medical trials; our potential to efficiently manufacture valoctocogene roxaparvovec for the medical trials and commercially; and people and people components detailed in BioMarin’s filings with the Securities and Change Fee (SEC), together with, with out limitation, the components contained underneath the caption “Threat Components” in BioMarin’s Quarterly Report on Kind 10-Q for the quarter ended June 30, 2022 as such components could also be up to date by any subsequent studies. Stockholders are urged to not place undue reliance on forward-looking statements, which communicate solely as of the date hereof. BioMarin is underneath no obligation, and expressly disclaims any obligation to replace or alter any forward-looking assertion, whether or not because of new info, future occasions or in any other case.

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

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BioMarin Resubmits Biologics License Software (BLA) for Valoctocogene Roxaparvovec AAV Gene Remedy for Extreme Hemophilia A to the FDA