SAN RAFAEL, Calif.,
April 21, 2017 /PRNewswire/ --
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the
Committee for Medicinal Products for Human Use (CHMP), the
scientific committee of the European Medicines Agency (EMA), has
adopted a positive opinion for the company's Marketing
Authorization Application (MAA) for Brineura™
(cerliponase alfa) to treat children with Neuronal Ceroid
Lipofuscinosis Type 2 (CLN2) disease, a form of Batten disease,
which is also known as tripeptidyl peptidase 1 (TPP1)
deficiency.
The CHMP's recommendation is now referred to the European
Commission (EC), which is expected to render its final decision by
the second quarter of 2017. The EC typically adheres to the
recommendation of the CHMP, but is not obligated to do so. If
approved by the EC, BioMarin will receive marketing authorization
for Brineura in all 28 countries of the European Union,
Norway, Iceland and Liechtenstein.
The CHMP positive opinion was adopted following an accelerated
review procedure, reserved for medicinal products expected to be of
major public health interest. The EMA revised process for
accelerated assessment came into effect June
1, 2016, and Brineura is one of the first therapies to go
through this process.
"A little less than four years ago, the first child was treated
in a clinical trial, and today marks another important step forward
in providing the first treatment option for children affected by
CLN2 disease, a rapidly progressing and fatal pediatric brain
disorder. We thank the CHMP and the CLN2 community for their
continued support, including the children and families who gave
their time to participate in the clinical trials with the goal of
making treatment a reality for patients," said Hank Fuchs, M.D., President of Worldwide
Research and Development at BioMarin. "It is a privilege and
an honor to pioneer the successful delivery of an enzyme
replacement therapy delivered directly to the brain and to show
that the treatment can slow or stabilize the progression of this
degenerative brain disease."
"I have dedicated my career to studying Batten disease and
participated in an ongoing effort to collect natural history data
in the hope of being able to use that information to study
potential treatments. To be the principal investigator in
these clinical trials using that natural history data, which has
led to a potential therapy, is both personally and professionally
fulfilling," said Angela Schulz,
M.D. Ph.D., Department of Paediatrics, Children's Hospital,
University Medical Center Hamburg-Eppendorf. "I am hopeful
that soon physicians will be able to offer to children an approved
medicine that has the potential to change the course of this
relentless disease."
Regulatory Submissions
The Brineura MAA was based on an open-label, dose-escalation
study for Brineura in 24 patients with CLN2 disease between 3 and 8
years of age, as well as an open-label extension study. The primary
objectives were to evaluate the safety and tolerability of
intracerebroventricular-administered Brineura and to evaluate
effectiveness using a CLN2 disease-specific rating scale score in
comparison with natural history data after 48 and 72 weeks of
treatment.
In 2016, the CHMP accepted BioMarin's request for accelerated
assessment. The EMA previously granted Brineura Orphan Drug
Designation.
The CHMP is a scientific committee composed of representatives
from the 28-member states of the EU, and Iceland, Norway and Liechtenstein. The committee
reviews medical product applications on their scientific and
clinical merit and provides advice to the EC, which has the
authority to approve medicines for the EU. The EC, which
typically adheres to the recommendation of the CHMP, is expected to
make its final decision in about 67 days.
Brineura is also currently under review by the U.S. Food and
Drug Administration (FDA) as a treatment for children three years
and older with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
disease, a form of Batten disease, which is also known
as tripeptidyl peptidase 1 (TPP1) deficiency. The FDA's
Prescription Drug User Fee Act (PDUFA) goal date for a decision is
April 27, 2017.
About Brineura
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 into the fluid
surrounding the brain (cerebrospinal fluid) using BioMarin's
patented technology. Brineura is an investigational enzyme
replacement therapy that has been shown to help stabilize or slow
the progression of CLN2 disease.
Brineura administered via intracerebroventricular infusion every
14 days was well tolerated, and no patients discontinued treatment
due to adverse events (AEs). Most AEs were Grade 1 or 2, and
the majority are consistent with severe, chronic neurologic disease
in pediatric patients. The most common events associated with
treatment included: pyrexia, hypersensitivity, seizure,
epilepsy, vomiting and headache.
For additional information regarding this investigational
product, please contact BioMarin Medical Information at
medinfo@bmrn.com.
About CLN2 Disease
Children with CLN2 disease typically begin experiencing seizures
between the ages of 2 and 4 years old, preceded in the majority of
cases by language development delay. The disease progresses
rapidly with most affected children losing the ability to walk and
talk by approximately 6 years of age. Initial symptoms are
followed by movement disorders, motor deterioration, dementia,
blindness, and death usually occurring between the ages of 8 and 12
years of age. During the later stages of the disease, feeding
and tending to everyday needs become very difficult. BioMarin
estimates the incidence of CLN2 disease is approximately one in
200,000 with up to 1,200 to 1,600 children in the regions of the
world where BioMarin operates, many of whom are undiagnosed.
The neuronal ceroid lipofuscinoses (NCLs) are a heterogeneous
group of lysosomal storage disorders that includes the autosomal
recessive neurodegenerative disorder CLN2 disease. CLN2 disease is
caused by mutations in the TPP1 gene resulting in deficient
activity of the enzyme tripeptidyl peptidase 1 (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 contributes to the progressive and relentless
neurodegeneration which manifests as loss of cognitive, motor, and
visual functions.
Currently, there is no approved therapy to treat CLN2 disease.
Symptomatic care to treat the symptoms of the disease, prevent and
treat complications, and attempt to preserve quality of life is the
only available treatment options for patients with this rare
disease.
Conference Call and Webcast to be Held Friday, April 21 at 4:05
pm ET
Interested parties may access a live webcast that will accompany
the conference call by going here. A replay of the call
will be archived on the site for one week following the call.
U.S. / Canada Dial-in Number: (866) 502-9859
International Dial-in Number: (574) 990-1362
Conference ID: 3969755
Replay Dial-in Number: (855) 859-2056
Replay International Dial-in Number: (404) 537-3406
Conference ID: 3969755
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.BioMarin.com.
Forward-Looking Statement
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including,
without limitation, statements about: regulatory filings for the
commercial approval of Brineura, including the expected timing of
the EC's final decision on Brineura. 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: the risk that
the EC or other regulatory agencies, including the FDA, may not
approve Brineura for the treatment of children with Neuronal Ceroid
Lipofuscinosis Type 2 (CLN2) disease; the results and timing of
current and future clinical trials related to Brineura; the risks
related to commercialization of Brineura and our ability to
manufacture sufficient quantities of Brineura for the 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 Annual Report on Form 10-K for the year ended
December 31, 2016, and future filings
and reports by the Company. The Company undertakes no duty or
obligation to update any forward-looking statements contained in
this Current Report on Form 8-K as a result of new information,
future events or changes in its expectations.
BioMarin® is a registered trademark and Brineura™ is
a trademark of BioMarin Pharmaceutical Inc.
Contacts:
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Debra
Charlesworth
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BioMarin
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BioMarin
Pharmaceutical Inc.
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(415)
455-7558
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455-7451
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SOURCE BioMarin Pharmaceutical Inc.