SAN RAFAEL, Calif.,
May 31, 2020 /PRNewswire/ -- BioMarin
Pharmaceutical Inc. (NASDAQ: BMRN) announced today an update to its
previously reported results of an open-label Phase 1/2 study of
valoctocogene roxaparvovec, an investigational gene therapy
treatment for adults with severe hemophilia A. The data have
been submitted as a late-breaking abstract to the World Federation
of Hemophilia (WFH) Virtual Summit to be held June 14-19, 2020.
The four-year update for the 6e13 vg/kg and three-year update
for the 4e13 vg/kg cohorts demonstrated that all subjects in both
cohorts remain off prophylactic Factor VIII treatment since
receiving their single dose of valoctocogene roxaparvovec.
Cumulative mean annualized bleed rates (ABR) remain less than one
(1) in both cohorts and below pre-treatment baseline levels.
The mean ABR in year four for the 6e13 vg/kg cohort was 1.3, and
the mean ABR in year three for the 4e13 vg/kg cohort was 0.5.
Over the past year, six of the seven participants in the 6e13 vg/kg
cohort and five of the six participants in the 4e13 vg/kg cohort
remain free of spontaneous bleeds. Factor VIII activity
levels declined commensurate with the most recent years'
observations and remain in a range to provide hemostatic
efficacy.
"It's been a privilege to participate in this pioneering
research and to observe how the patients on the study have done so
much to improve our understanding of gene therapy research for
hemophilia A. This additional data is an important step
toward a potential first treatment of its kind for this devastating
disease," said Professor John Pasi,
M.B., Ch.B., Ph.D., from Barts and the London School of Medicine
and Dentistry; chief investigator for the valoctocogene
roxaparvovec Phase 1/2 study, and a principal investigator for the
Phase 3 study. "Each year of data increases our knowledge of
safety and efficacy and contributes to the growing body of
scientific data on gene therapies in general and hemophilia A in
particular."
"BioMarin is proud to have advanced the community's knowledge of
the potential for gene therapy to transform lives, and we are
grateful for the support of the bleeding disorders community in
this endeavor. In just over four years since starting clinical
trials in patients, we've submitted applications for marketing
authorizations globally, and we continue to contribute to the
growing body of scientific data in gene therapy for hemophilia A
with five studies underway," said Hank
Fuchs, M.D., President, Worldwide Research and Development
at BioMarin. "We continue to move forward with health
authorities to make this treatment available for people with severe
hemophilia A. We are committed to pioneering this field
and advancing the standard of care for patients."
Safety Summary
Overall, the safety profile of valoctocogene roxaparvovec
remains consistent with previously reported data with no
delayed-onset treatment related events. No participants
developed inhibitors to Factor VIII, and no participants withdrew
from the study. No participants have developed thrombotic
events. The most common adverse events associated with
valoctocogene roxaparvovec occurred early and included transient
infusion-associated reactions and transient, asymptomatic, and mild
to moderate rise in the levels of certain proteins and enzymes
measured in liver function tests with no long-lasting clinical
sequelae.
Regulatory Status
The Food and Drug Administration (FDA) is reviewing the
biologics license application, under Priority Review, for
valoctocogene roxaparvovec with a PDUFA action date of August 21, 2020. The FDA also granted
valoctocogene roxaparvovec Breakthrough Therapy
designation.
The European Medicines Agency (EMA) validated the Company's
Marketing Authorization Application (MAA) for valoctocogene
roxaparvovec which has been in review under accelerated assessment
since January. Recognizing valoctocogene roxaparvovec for its
potential to benefit patients with unmet medical needs, EMA granted
access to its Priority Medicines (PRIME) regulatory
initiative. Although the MAA remains under accelerated
assessment at this time, the Company expects the review procedure
to be extended by at least three months due to COVID-19
delays. Further, the Company believes there is a high
possibility that the MAA will revert to the standard review
procedure, as is the case with most filings that initially receive
accelerated assessment. Because of the combination of these
events, the Company expects an opinion from the Committee for
Medicinal Products for Human Use (CHMP) in late 2020/early
2021.
BioMarin's valoctocogene roxaparvovec has also 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.
The Company believes that both submissions represent the first
time a gene therapy product for any type of hemophilia indication
is under review for marketing authorization by health
authorities.
Gene Therapy Manufacturing
The Company recently received EMA cGMP certification for its
gene therapy manufacturing facility and quality systems for the
production of valoctocogene roxaparvovec, an important step in
obtaining regulatory approval of the product in the European Union.
The Health Products Regulatory Authority (HPRA)
of Ireland conducted, on behalf of EMA, a pre-approval
inspection in the first quarter and issued a cGMP certification in
the second quarter. The inspection of the facility by FDA is
expected to be complete during the second quarter, which would
allow for potential licensure of the facility in the U.S.
consistent with the August 21st PDUFA date.
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 patients with 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 Statement
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including
without limitation, statements about: (i) the development of
BioMarin's valoctocogene roxaparvovec program generally, (ii) the
impact of valoctocogene roxaparvovec gene therapy for treating
patients with severe hemophilia A, (iii) that Factor VIII activity
levels declined commensurate with prior year's observations and
remain in a range to provide hemostatic efficacy, (iv) the planned
updates of the Phase 1/2 study including the Company's submission
of a late-breaking abstract to share four years of data at the WFH
Virtual Summit, (v) the inspection of its gene therapy
manufacturing facility by the FDA to be completed during the second
quarter of 2020, (vi) the licensure of its gene therapy
manufacturing facility, including the timing of such licensure, and
(vii) the potential approval and commercialization of valoctocogene
roxaparvovec for the treatment of severe hemophilia A, including
timing of such approval decisions.
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 the above interim data;
any potential adverse events observed in the continuing monitoring
of the patients in the Phase 1/2 trial; the content and timing of
decisions by the FDA, the European Commission and other regulatory
authorities, including the potential impact of the COVID-19
pandemic on the regulatory authorities' abilities to issue such
decisions and the timing of such decisions; the content and timing
of decisions by local and central ethics committees regarding the
clinical trials; BioMarin's ability to successfully manufacture
valoctocogene roxaparvovec; 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 BioMarin's Quarterly Report on Form 10-Q for the quarter
ended March 31, 2020, and future
filings and reports by BioMarin. 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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Traci
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Debra
Charlesworth
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BioMarin
Pharmaceutical Inc.
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BioMarin
Pharmaceutical Inc.
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455-7451
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SOURCE BioMarin Pharmaceutical Inc.