SAN RAFAEL, Calif.,
Feb. 1, 2017 /PRNewswire/
-- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today
that the European Medicines Agency (EMA) has granted access to
its Priority Medicines (PRIME) regulatory initiative for the
company's investigational gene therapy treatment for severe
hemophilia A, BMN 270. 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.
The Committee for Medicinal Products for Human Use (CHMP), which
is the EMA's committee responsible for human medicines, determined
to provide access to the PRIME scheme based on findings by the CHMP
that:
- the unmet need is justified in particular on the basis of
breakthrough bleeds in prophylactic treatment settings, leading to
sequelae (negative after effect of the disease), including
hemophilic arthropathy (permanent joint disease as a result of
repeated bleeding in the joints); and
- the potential to address the need is justified on the basis of
preliminary clinical data in affected patients, supporting that a
single IV administration results in sustained restoration of factor
VIII activity, reduction of Annualized Bleeding Rates and improved
quality of life.
"We are thrilled that the EMA has recognized the potential of
BMN 270 to change the course of hemophilia A and provide a
treatment paradigm currently unmet by available therapies," said
Hank Fuchs, M.D., President
Worldwide Research and Development at BioMarin. "We look
forward to preparing to enroll patients in a registration enbabling
study in the third quarter of this year, as we work closely with
the EMA to accelerate development and hopefully to facilitate
earlier access to the first gene therapy treatment for patients
with severe hemophilia A, the most common form of the disease."
PRIME is a scheme to strengthen support to medicines that target
an unmet medical need. The scheme 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).
Through PRIME, EMA offers early, proactive and enhanced support
to medicine developers to optimize the generation of robust data on
a medicine's benefits and risks and enable accelerated assessment
of medicine applications. This will help patients to benefit as
early as possible from therapies that may significantly improve
their quality of life.
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.1 As an X-linked disorder, hemophilia A mostly
affects males, occurring in approximately 1 in 5,000 male
births.2 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% of hemophilia A
patients who are severely affected, is a prophylactic regimen of
factor VIII infusions three times per week.3 Even with
prophylactic regimens, many patients still experience microbleeds
and spontaneous bleeding events that result in progressive joint
damage.
About Gene Therapy
Gene therapy is a treatment designed to alter a genetic problem
by adding a corrected copy of the defective gene. The functional
gene is inserted into a vector – containing a DNA sequence coding
for a specific protein – 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 protein
that the body needs, potentially reducing or eliminating the cause
of the disease. Currently, gene therapy for the treatment of
hemophilia A is available only as part of a clinical trial.
The AAV approach to gene therapy has been advanced at the
University College London (UCL) in the treatment of Hemophilia B.
At UCL, this technology has shown promising evidence of both safety
and efficacy, correcting bleeding for greater than four years in a
continuing clinical trial.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for people with serious and
life-threatening rare disorders. 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
BMN 270 program generally and the timing and results of the
clinical trial of BMN 270; the impact of the access to the EMA
PRIME scheme and the associated potential for accelerated
assessment. 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 BMN 270, including final
analysis of the Phase 1/2 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 U.S. Food and
Drug Administration, the European Commission and other regulatory
authorities; a potential decision by the EMA to remove BMN 270 from
the PRIME scheme or the accelerated assessment framework; 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 factors detailed in BioMarin's
filings with the Securities and Exchange Commission, including,
without limitation, the factors contained under the caption "Risk
Factors" in BioMarin's 2015 Annual Report on Form 10-K, and the
factors contained in BioMarin's reports on Form 10-Q. Stockholders
are urged 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 alter any forward-looking statement, whether as a result
of new information, future events or otherwise.
BioMarin® is a registered trademark of BioMarin
Pharmaceutical Inc.
1 Source: National Hemophilia Foundation
http://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A
2 Source: CDC
http://www.cdc.gov/ncbddd/hemophilia/data.html
3 Source: World Federation of Hemophilia
http://www.wfh.org/en/resources/annual-global-survey
http://www.wfh.org/en/abd/prophylaxis/prophylaxis-administration-and-dosing-schedules
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.