SAN RAFAEL, Calif.,
March 8, 2021 /PRNewswire/
-- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced
that the U.S. Food and Drug Administration (FDA) granted
Regenerative Medicine Advanced Therapy (RMAT) designation to
valoctocogene roxaparvovec, an investigational gene therapy for the
treatment of adults with severe hemophilia A. The FDA granted
RMAT designation based on the potential of the valoctocogene
roxaparvovec clinical data to address the unmet medical need
within this population.
RMAT is an expedited program intended to facilitate development
and review of regenerative medicine therapies, such as
valoctocogene roxaparvovec, that are intended to address an unmet
medical need in patients with serious conditions. The RMAT
designation is complementary to Breakthrough Therapy Designation,
which the Company received in 2017, allowing early, close, and
frequent interactions with the FDA. One additional feature of the
RMAT program is that sponsors of products that have been granted
RMAT designation and which receive accelerated approval may be able
to fulfill the post-approval requirements from clinical evidence
obtained from sources other than the traditional confirmatory
clinical trials.
The RMAT designation comes coincidently during Bleeding
Disorders Awareness Month initiated by the National Hemophilia
Foundation to celebrate and honor the bleeding disorders
community.
"We are encouraged that the FDA granted RMAT Designation to
valoctocogene roxaparvovec. This designation confirms our
belief in this treatment's potential to address unmet medical needs
for people with hemophilia A at this time," said Hank Fuchs, M.D., President of Worldwide
Research and Development at BioMarin. "We look forward to
continuing a productive dialogue with the FDA around the RMAT
designation, which provides options for the Agency to leverage data
post approval, while also recognizing the agency's initial request
to see two years of data from the Phase 3 study to evaluate the
safety and efficacy of this investigational treatment option that
could potentially provide a transformational treatment choice for
the hemophilia community."
"During Bleeding Disorders Awareness Month, we applaud the FDA
for its efforts to recognize the potential of cell and gene
therapies to help people with hemophilia who have medical needs not
currently addressed," said Leonard A.
Valentino, MD, President & CEO of the National
Hemophilia Foundation (NHF). "Established under the 21st
Century Cures Act, RMAT designation has the potential to provide
more treatment choices to people with bleeding disorders at a
faster pace, which benefits the whole community. The FDA's RMAT
designation is a critical program to advance the efficient
development and regulatory review of regenerative medicine products
that have the potential to address unmet needs," said the Alliance
for Regenerative Medicine (ARM). "As the global voice of the
regenerative medicine sector, ARM played a critical role in the
creation of this pathway. The FDA has granted more than 50 RMAT
designations to investigational products and in February approved
an RMAT-designated product for the first time, illustrating the
agency's commitment to advancing the development of regenerative
medicines."
Regulatory Status
In the Complete Response Letter of August
18, 2020 to the Company's Biologics License Application
(BLA) for valoctocogene roxaparvovec, the FDA recommended that the
Company complete the Phase 3 GENEr8-1 study and submit two-year
follow-up safety and efficacy data on all study participants. The
Company plans to meet with FDA to review the two-year data request
and share the Phase 3 GENEr8-1 results announced on January 10, 2021. In the EU, BioMarin is
targeting submission of the Marketing Authorization Application
(MAA) with these results to the EMA in the second quarter of 2021
pending confirmation in planned pre-submission meetings.
In addition to the RMAT Designation and Breakthrough Therapy
Designation, BioMarin's valoctocogene roxaparvovec also has
received orphan drug designation from the FDA and EMA for the
treatment of severe hemophilia A. The Orphan Drug Designation
program is intended to advance the evaluation and development of
products that demonstrate promise for the diagnosis and/or
treatment of rare diseases or conditions.
Robust Clinical Program
BioMarin has multiple clinical studies underway in its
comprehensive gene therapy program for the treatment of severe
hemophilia A. In addition to the global Phase 3 study GENEr8-1
and the ongoing Phase 1/2 dose escalation study, the Company
recently began enrolling participants in a Phase 3b, single arm, open-label study to evaluate the
efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13
vg/kg with prophylactic corticosteroids in people with hemophilia
A. The Company is running a Phase 1/2 Study with the 6e13kg/vg
dose of valoctocogene roxaparvovec in approximately 10 participants
with pre-existing AAV5 antibodies, as well as another Phase 1/2
Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in
people with hemophilia A with active or prior FVIII
inhibitors.
About Hemophilia A
People living with hemophilia A lack sufficient functioning
Factor VIII protein to help their blood clot and are at risk for
painful and/or potentially life-threatening bleeds from even modest
injuries. Additionally, people with the most severe form of
hemophilia A (FVIII levels <1%) often experience painful,
spontaneous bleeds into their muscles or joints. Individuals with
the most severe form of hemophilia A make up approximately 50
percent of the hemophilia A population. People with hemophilia
A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease show a
much-reduced propensity to bleed. The standard of care for
individuals with severe hemophilia A is a prophylactic regimen of
replacement Factor VIII infusions administered intravenously up to
two to three times per week or 100 to 150 infusions per
year. Despite these regimens, many people continue to
experience breakthrough bleeds, resulting in progressive and
debilitating joint damage, which can have a major impact on their
quality of life.
Hemophilia A, also called Factor VIII deficiency or classic
hemophilia, is an X-linked genetic disorder caused by missing or
defective Factor VIII, a clotting protein. Although it is passed
down from parents to children, about 1/3 of cases are caused by a
spontaneous mutation, a new mutation that was not inherited.
Approximately 1 in 10,000 people have Hemophilia A.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for serious and
life-threatening rare and ultra-rare genetic diseases. The
Company's portfolio consists of six commercialized products and
multiple clinical and pre-clinical product candidates. For
additional information, please visit www.biomarin.com.
Information on BioMarin's website is not incorporated by reference
into this press release.
Forward Looking Statements
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including
without limitation, statements about the development of BioMarin's
valoctocogene roxaparvovec program generally, the RMAT designation,
the Company's plans to meet with the FDA to review the 2-year data
request and share the Phase 3 GENEr8-1 results announced on
January 10, 2021, BioMarin targeting
submission of the MAA with these results to the EMA in the second
quarter of 2021; the potential of valoctocogene roxaparvovec to
address the unmet medical need within this hemophilia A
population. 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: results and timing of current
and planned preclinical studies and clinical trials of
valoctocogene roxaparvovec, including final analysis of data from
the continuation of these trials; any potential adverse events
observed in the continuing monitoring of the patients in the
clinical trials; the content and timing of decisions by the FDA,
the EMA and other regulatory authorities; the content and timing of
decisions by local and central ethics committees regarding the
clinical trials; our ability to successfully manufacture the
product candidate for the preclinical and clinical trials;
and those other risks detailed from time to time under the caption
"Risk Factors" and elsewhere in BioMarin's Securities and Exchange
Commission (SEC) filings, including without limitation, BioMarin's
Annual Report on Form 10-K for the year ended December 31, 2020 as such factors may be updated
by any subsequent reports. BioMarin undertakes no duty 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.
Contacts:
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Debra
Charlesworth
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BioMarin
Pharmaceutical Inc.
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BioMarin
Pharmaceutical Inc.
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(415)
455-7558
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455-7451
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SOURCE BioMarin Pharmaceutical Inc.