BioMarin Announces EMA Validation of Brineura™ (Cerliponase Alfa) Marketing Authorization Application for Treatment of CLN2...
September 15 2016 - 8:38AM
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the
European Medicines Agency (EMA) validated the Marketing
Authorization Application (MAA) for Brineura™ (cerliponase alfa) to
treat children with CLN2 disease, a form of Batten
disease. Validation of the MAA confirms that the submission is
accepted and starts the formal review process by the EMA's
Committee for Human Medicinal Products (CHMP).
Earlier this year, the CHMP accepted BioMarin's request for
accelerated assessment of the MAA on the grounds that Brineura is
of major public health interest with the potential to have a major
impact on medical practice for CLN2 patients in
Europe. Accelerated assessment has the potential to shorten
the EMA's review procedure. However, applications which are
initially granted accelerated assessment frequently revert to
standard assessment timelines. Assuming a positive opinion from the
CHMP and standard assessment timing, a decision from the European
Commission is anticipated by the third quarter of 2017. The EMA
previously granted Brineura Orphan Drug Designation.
“CLN2 disease is a rapidly progressing, fatal neurodegenerative
disease with no approved treatments. Based on the positive results
of the pivotal study, we are working to bring this meaningful
therapeutic option to patients and families in Europe as soon as
possible," said Hank Fuchs, MD, Chief Medical Officer of BioMarin.
"We greatly appreciate the CLN2 community’s ongoing support and
look forward to continuing to work with regulatory authorities in
the coming months.”
Children with CLN2 disease typically begin to present symptoms
between the ages of two and four, with the majority of affected
children losing their ability to walk and talk by approximately six
years of age. Initial symptoms can include language delay and
seizures, followed by movement disorders, motor deterioration,
dementia and blindness. During the later stages of the disease,
feeding and tending to everyday needs become very difficult, and
death often occurs between eight and 12 years of age.
Marketing Applications
On July 27, 2016, the U.S. Food and Drug Administration (FDA)
accepted for review the submission of a Biologics License
Application (BLA) for cerliponase alfa. The Prescription Drug
User Fee Act (PDUFA) goal date for a decision is April 27, 2017.
The FDA granted cerliponase alfa Priority Review status, which is
designated to drugs that offer major advances in treatment or
provide a treatment where no adequate therapy exists. Cerliponase
alfa was previously granted Orphan Drug Designation and
Breakthrough Therapy Designation by the FDA. The Agency has
advised that they plan to hold an advisory committee meeting at a
date to be confirmed per their usual practice of notification in
the Federal Register.
Early Access Program
BioMarin has implemented an early access (compassionate use)
program as planned to provide experimental drug for additional CLN2
patients prior to obtaining marketing approval. The program
is limited in scope and number of participants, and is being
conducted under a protocol. The program initially is being
conducted at centers that have participated in the cerliponase alfa
study. The program began in August 2016 in Hamburg, Germany
and Columbus, OH, U.S.A. We continue to work on opening the
other sites, while adhering to specific legal and regulatory
procedures for each country. In order to assure fairness in
inclusion, enrollment decisions will be made independent of
BioMarin. In addition, the identities of participants are
confidential to protect the privacy of the patients and
families.
About Cerliponase Alfa
Brineura is a recombinant form of human tripeptidyl peptidase 1
(TPP1), the enzyme deficient in patients with CLN2 disease. It is
an enzyme replacement therapy designed to restore TPP1 enzyme
activity and break down the storage materials that cause CLN2
disease. In order to reach the cells of the brain and central
nervous system, the treatment is delivered directly to the fluid
surrounding the brain (cerebrospinal fluid) using BioMarin's
patented technology.
For additional information regarding the investigational product
Brineura, please contact BioMarin Medical Information
at medinfo@bmrn.com.
About CLN2 Disease
CLN2 disease is caused by mutations in the TPP1/CLN2 gene,
resulting in deficient activity of the enzyme TPP1. In the absence
of TPP1, lysosomal storage materials normally metabolized by this
enzyme accumulate in many organs, particularly in the brain and
retina. Buildup of these storage materials in the cells of the
nervous system contribute to progressive and relentless
neurodegeneration, which manifests as loss of cognitive, motor and
visual functions.
There is no approved treatment that can prevent, stop or reverse
CLN2 disease. Symptomatic care to treat disease symptoms, prevent
and treat complications, and attempt to preserve quality of life is
the only currently available option for patients with this rare
disease.
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: BioMarin's development
programs for cerliponase alfa generally, and specifically about
regulatory filings for commercial approval of the product
candidate, the results of the Phase 1/2 pivotal trial and an
ongoing extension study of cerliponase alfa. 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 cerliponase alfa; 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
cerliponase alfa 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" in BioMarin's 2015 Annual Report on Form 10-K, as amended,
and the factors contained in BioMarin's reports on Form
8-K. 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 and Brineura™ is a trademark
of BioMarin Pharmaceutical Inc.
Contacts:
Investors
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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