SAN RAFAEL, Calif.,
Dec. 22, 2017 /PRNewswire/
-- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today
announced that the U.S. Food and Drug Administration (FDA) will
require additional time to complete its review of the Biologics
License Application (BLA) for its investigational therapy
pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase
enzyme product, to reduce blood phenylalanine (Phe) levels in adult
patients with phenylketonuria (PKU) who have uncontrolled blood Phe
levels on existing management. In a notice received from the FDA,
the Prescription Drug User Fee Act (PDUFA) Goal Date for pegvaliase
has been extended by three months to May
28, 2018. Due to the Memorial Day weekend, the Action
Goal Date will be May 25, 2018.
On Aug. 29, 2017, when the FDA
accepted BioMarin's BLA and granted priority review status, the
company announced that the FDA had requested additional information
on Chemistry, Manufacturing and Controls (CMC), which was likely to
be classified as a major amendment to the BLA and result in a three
month extension of the PDUFA date. As expected, the FDA
designated the receipt of this additional information as a major
amendment to the application thus extending the PDUFA action date
by three months.
"We are pleased with our ongoing interactions with the FDA on
the pegvaliase BLA. We appreciate the FDA's ability to
expedite review through priority designation, particularly for this
complex disease and treatment," said Hank
Fuchs, M.D., President Worldwide Research and Development at
BioMarin. "We continue to work closely with the FDA and look
forward to the possibility of bringing this important treatment to
patients."
The FDA has granted priority review designation to pegvaliase,
which is granted to drugs that treat a serious condition and, if
approved, would provide a significant improvement in safety or
effectiveness of the treatment, prevention, or diagnosis of a
serious condition.
About Pegvaliase
Pegvaliase is an investigational study drug that substitutes the
deficient PAH enzyme in PKU with the PEGylated version of the
enzyme phenylalanine ammonia lyase, to break down Phe. It is being
developed as a potential treatment for adults with inadequately
controlled blood Phe levels in the study. In clinical
studies, treatment with subcutaneous pegvaliase substantially
reduced blood Phe compared to placebo using a randomized withdrawal
study design, and led to long-term maintenance of Phe reduction in
the majority of adult patients with PKU. Pegvaliase was
administered using a dosing regimen that achieved a manageable
safety profile, consisting primarily of immune-mediated responses,
including anaphylaxis, for which robust risk management measures
effective in clinical trials will be proposed.
For additional information regarding the investigational product
pegvaliase, please contact BioMarin Medical Information at
medinfo@bmrn.com.
About Phenylketonuria
Phenylketonuria (PKU), or phenylalanine hydroxylase (PAH)
deficiency, is a genetic disorder affecting approximately 50,000
diagnosed patients in the developed world and is caused by a
deficiency of the enzyme PAH. This enzyme is required for the
metabolism of Phe, an essential amino acid found in most
protein-containing foods. If the active enzyme is not present in
sufficient quantities, Phe accumulates to abnormally high levels in
the blood and becomes toxic to the brain, resulting in a variety of
complications including severe intellectual disability, seizures,
tremors, behavioral problems and psychiatric symptoms. As a result
of newborn screening efforts implemented in the 1960s and early
1970s, virtually all individuals with PKU under the age of 40 in
developed countries are diagnosed at birth and treatment is
implemented soon after. PKU can be managed with a Phe-restricted
diet, which is supplemented by low-protein modified foods and
Phe-free medical foods; however, the strict diet is difficult for
most patients to adhere to the extent needed for achieving adequate
control of blood Phe levels.
To learn more about PKU, please visit www.PKU.com. Information
on this website is not incorporated by reference into this press
release.
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 six 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: BioMarin's development
programs for pegvaliase generally, and specifically about
expectations regarding the BLA filing for pegvaliase with the FDA
and the FDA's evaluation of such filing; the potential outcome of
the review of such filings; and the possible approval of such
product candidate. 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 of current and
planned clinical trials of pegvaliase; the content and timing of
decisions by the U.S. Food and Drug Administration, the European
Medicines Agency and other regulatory authorities; our ability to
manufacture sufficient quantities of pegvaliase for clinical
trials, commercial launch and other preapproval requirements; 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" and elsewhere in the
Company's Securities and Exchange Commission (SEC) filings,
including the Company's Quarterly Report on Form 10-Q for the
quarter ended September 30, 2017, and
future filings and reports by the Company. The Company 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.
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.