SAN RAFAEL, Calif.,
Dec. 19, 2017 /PRNewswire/
-- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today
that it has dosed the first patient in the global GENEr8-1 Phase 3
study with the 6e13 vg/kg dose for valoctocogene roxaparvovec
(formerly BMN 270), an investigational gene therapy for the
treatment of patients with severe hemophilia A. This is the
first of two Phase 3 studies in the global Phase 3 program to dose
a first patient.
"We are thrilled to have enrolled the first patient in this
potential registration-enabling study for gene therapy in severe
hemophilia A," said Hank Fuchs,
M.D., President, Worldwide Research and Development at
BioMarin. "I am proud of the effort that BioMarin has made to
move this breakthrough therapy rapidly into Phase 3 bringing this
one-time treatment one step closer to patients."
The global Phase 3 program includes two studies with
valoctocogene roxaparvovec, one with the 6e13 vg/kg dose (GENEr8-1)
and one with the 4e13 vg/kg dose (GENEr8-2). Both Phase
3 GENEr8 studies will be open-label single-arm studies to evaluate
the efficacy and safety of valoctocogene roxaparvovec.
GENEr8-2 will enroll the first patient at the start of 2018.
The primary endpoint in both studies will be based on the FVIII
activity level achieved following valoctocogene roxaparvovec, and
the secondary endpoints will measure annualized FVIII replacement
therapy use rate and annualized bleed rate.
BioMarin will also begin a Phase 1/2 Study with the 6e13kg/vg
dose and with approximately 10 patients who are AAV5
positive. The first patient is expected to enroll in the
first half of 2018.
Regulatory Status
The U.S. Food and Drug Administration (FDA) granted
valoctocogene roxaparvovec Breakthrough Therapy
Designation. The FDA's Breakthrough Therapy Designation
program is intended to facilitate and expedite development and
review of new drugs to address unmet medical need in the treatment
of a serious condition. To qualify for Breakthrough Therapy
Designation, preliminary clinical evidence must show that that the
drug may demonstrate substantial improvement over existing
therapies.
The European Medicines Agency (EMA) has granted access to
its Priority Medicines (PRIME) regulatory initiative for
valoctocogene roxaparvovec. To be accepted for PRIME, an
investigational therapy has to show its potential to benefit
patients with unmet medical needs based on early clinical data.
PRIME focuses on medicines that may offer a major therapeutic
advantage over existing treatments, or benefit patients with no
treatment options. These medicines are considered priority
medicines within the European Union (EU).
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.
Gene Therapy Manufacturing
BioMarin has constructed one of the largest gene therapy
manufacturing facilities in the world, which is located in
Novato, California. Good
Manufacturing Practices (GMP) production of valoctocogene
roxaparvovec has commenced and will support pivotal clinical
development activities and anticipated commercial demand. This
facility is capable of supporting approximately 2,000 patients per
year, and the production process was developed in accordance with
International Conference on Harmonisation guidance for
Pharmaceuticals for Human Use facilitating worldwide registration
with health authorities.
About Hemophilia A
Hemophilia A, also called factor VIII (FVIII) deficiency or
classic hemophilia, is a 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 is born with Hemophilia A.
People living with the disease are not able to form blood
clots efficiently and are at risk for excessive bleeding from
modest injuries, potentially endangering their life. People with
severe hemophilia often bleed spontaneously into their muscles or
joints. The standard of care for the 43 percent of hemophilia A
patients, who are severely affected, is a prophylactic regimen of
Factor VIII infusions three times per week. Even with prophylactic
regimens, many patients still experience spontaneous bleeding
events that result in progressive and debilitating joint
damage.
About Gene Therapy
Gene therapy is a form of treatment designed to fix a genetic
problem by adding a corrected copy of the defective gene. The
functional gene is inserted into a vector, containing a small DNA
sequence, that acts as a delivery mechanism, providing the ability
to deliver the functional gene to cells. The cells can then use the
information to build the functional proteins that the body needs,
potentially reducing or eliminating the cause of the disease.
Currently, in the United States,
gene therapy is available only as part of a clinical trial.
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 five commercialized products and
multiple clinical and pre-clinical product candidates. For
additional information, please visit www.BMRN.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 the development of BioMarin's
valoctocogene roxaparvovec program generally, the timing and
results of the planned clinical trials of valoctocogene
roxaparvovec and statements regarding the company's gene therapy
manufacturing facility and its ability to support anticipated
commercial demand. 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; the content and timing of decisions by
the U.S. Food and Drug Administration, the European Commission and
other regulatory authorities; 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 BioMarin's Quarterly
Report on Form 10-Q for the quarter ended September 30, 2017, 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 trademarks of BioMarin Pharmaceutical
Inc.
Contact:
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Investors:
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Media:
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Traci
McCarty
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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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(415)
455-7451
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SOURCE BioMarin Pharmaceutical Inc.