SAN RAFAEL, Calif.,
June 30, 2017 /PRNewswire/
-- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced
that the company submitted a Biologics License Application (BLA) on
Friday, June 30, 2017 to the U.S.
Food and Drug Administration (FDA) for pegvaliase, a PEGylated
recombinant phenylalanine ammonia lyase enzyme product, to reduce
blood phenylalanine (Phe) levels in adult patients with PKU who
have uncontrolled blood Phe levels on existing management.
Following receipt of the BLA, the FDA conducts an initial
assessment of the application to determine its fileability.
The FDA typically notifies the applicant of their filing decision
and planned Prescription Drug User Fee Act (PDUFA) action date
within 60 to 74 days after receipt of the application. The company
also intends to submit an application for registration in the
European Union (EU) by year end 2017.
"We believe that pegvaliase offers the promise of an important
new treatment option for those adult patients with PKU unable to
manage their condition with existing treatments. Pegvaliase
has been shown to lower blood Phe levels, which was the primary
endpoint for registration of the only therapy currently approved to
treat PKU," said Hank Fuchs, M.D.,
President Worldwide Research and Development. "The current
medical guidelines highlight that the primary goal of therapy is to
lower Phe, and pegvaliase represents an important advance in
achieving that goal for adult PKU patients. We look forward
to working with the FDA to bring this treatment to patients."
"Not all PKU patients experience the same symptoms, but we know
that these symptoms improve with a reduction in blood Phe levels,"
said Nicola Longo, M.D., Ph.D.,
Professor at the University of Utah
School of Medicine and investigator for the pegvaliase Phase 3
studies. "For those adult patients who cannot control Phe
levels on existing management, pegvaliase can reduce Phe levels and
produce meaningful benefits. I look forward to working with
the FDA to convey my enthusiasm for a therapy that can improve upon
what's currently available."
About Pegvaliase
Pegvaliase is an investigational study drug that substitutes the
deficient PAH enzyme in PKU with the PEGylated version of the
enzyme phenylalanine lyase, to break down Phe. It is being
developed as a potential treatment for adults with inadequately
controlled blood Phe levels. 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 or Phenylalanine Hydroxylase
Deficiency
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 or PAH deficiency under
the age of 40 in developed countries are diagnosed at birth and
treatment is implemented soon after. PAH deficiency 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 and PAH deficiency, please visit
www.PKU.com. Information on this website is not incorporated by
reference into this press release.
About ACMG Guidelines
Practice guidelines issued by the American College of Medical
Genetics and Genomics (ACMG) support the need for lifelong
management of Phe levels in patients with phenylketonuria or PKU.
The new diagnosis and management guidelines were published online
in Genetics In Medicine's Advance Online Publication (AOP) service
and provide the first update to recommendations for therapy of PKU
since the 2001 National Institutes of Health Consensus
statement.
The guidelines state that treatment of PKU should be initiated
as early as possible and must be continued throughout adulthood
with a goal of maintaining blood levels of Phe for all patients
between 120-360 umol/L. Patients treated from the early weeks of
life with initial good metabolic control, but who lose that control
in later childhood or adult life, may experience both reversible
and irreversible neuropsychiatric consequences.
According to the guidelines "the primary goal of therapy is to
lower blood Phe, and any interventions, including medications, or
combination of therapies that help to achieve that goal in an
individual, without other negative consequences, should be
considered appropriate therapy."
Evidence for the guidelines are drawn from two previous
independent review processes from the National Institutes of Health
(2001) and the Agency for Health Research and Quality (2012). The
guidelines can be accessed online at:
https://www.acmg.net/docs/Phenylalanine_Hydrosylase_Deficiency_Practice_Guideline_AOP_Jan_2013.pdf
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 EMA; the potential outcome of the review of such filings; and
the possible approval of such product candidates. 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 clinical trials
of its product candidates; the content and timing of decisions by
the FDA, the EMA and other regulatory authorities concerning its
product candidates; our ability to manufacture sufficient
quantities of pegvaliase for clinical trials, commercial launch and
other preapproval requirements; and those other risks detailed from
time to time 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 March 31, 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:
|
|
Investors:
|
Media:
|
Traci
McCarty
|
Debra
Charlesworth
|
BioMarin
Pharmaceutical Inc.
|
BioMarin
Pharmaceutical Inc.
|
(415)
455-7558
|
(415)
455-7451
|
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/biomarin-submits-pegvaliase-biologics-license-application-bla-to-the-us-fda-for-treatment-of-phenylketonuria-pku-300482373.html
SOURCE BioMarin Pharmaceutical Inc.