BioMarin Announces EMA Grants Accelerated Assessment for Cerliponase Alfa, Experimental Treatment for a Form of Batten Disea...
May 03 2016 - 8:25AM
EU and U.S. Marketing Application Submissions
Planned for Mid-Year 2016
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced
that the European Medicines Agency (EMA) has granted BioMarin's
request for accelerated assessment for the planned cerliponase alfa
Marketing Authorization Application (MAA). Accelerated
assessments are granted on the grounds that a product may satisfy
an unmet medical need and is of major interest from the point of
view of therapeutic innovation and public health. Accelerated
assessment has the potential to shorten EMA's review
procedure. However, at any time during the MAA assessment, the
EMA may decide to continue the assessment under standard assessment
timelines, and most applications that initially qualify for
accelerated assessment are ultimately reviewed on a standard
timeline.
Cerliponase alfa is a recombinant human tripeptidyl peptidase 1
(rhTPP1) intended for the treatment of children with CLN2 disease,
a form of Batten disease. CLN2 disease is a rapidly
progressing, fatal neurodegenerative disease with no approved
treatments.
The company expects to submit the cerliponase alfa MAA to the
EMA and the Biologics License Application to the U.S. Food and Drug
Administration (FDA) by mid-year. If the cerliponase alfa MAA
is accepted by the EMA, then an opinion from the Committee for
Medicinal Products for Human Use (CHMP) is anticipated in the first
quarter of 2017, and, if positive, a decision from the European
Commission could be received in the first half of 2017.
"We are pleased that the EMA has recognized the need to bring a
therapy to children with this particular form of Batten disease as
quickly as possible. This could be the first therapeutic
option available for these children,” said Jean-Jacques Bienaimé,
Chairman and Chief Executive Officer of BioMarin. "We look forward
to entering the regulatory review phase for cerliponase alfa on a
global basis. CLN2 disease represents a significant unmet
medical need for those affected, and our hope is to offer a
life-altering treatment option to patients worldwide."
In the U.S., BioMarin is seeking to shorten the regulatory
review time by requesting Priority Review. Priority Review status
is designated by the FDA to drugs that offer major advances in
treatment or provide a treatment where no adequate therapy exists.
The FDA will evaluate this request following submission of
the planned Biologics License Application (BLA). Cerliponase
alfa has been granted Orphan Drug Designation by the FDA and EMA
and Breakthrough Therapy designation by the FDA.
Early Access Program
BioMarin is planning to implement an early access program to
provide limited access to treatment for additional CLN2 patients
prior to obtaining marketing approval. An early access
program will be limited in scope and number of participants, and
will be conducted under a protocol. We expect that the
program will be conducted at centers that have participated in the
cerliponase alfa study. Those sites have experience
administering this drug directly to the brain and would ensure
continued patient monitoring. The program is expected to
begin in at least one site by the end of Q3 2016, and the timing of
additional sites will vary by country and individual site.
The overall scope, eligibility criteria and details of this program
are still being determined. BioMarin must
adhere to specific legal procedures for each country and has begun
these preparations at risk with the goal of being ready to dose
patients by the end of Q3 2016. BioMarin will provide
additional details about the scope and timing of this program as
they become available.
About Cerliponase Alfa
Cerliponase alfa is a recombinant form of human 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
cerliponase alfa, please contact BioMarin Medical Information at
medinfo@bmrn.com.
About CLN2 Disease
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/CLN2 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 contribute to the progressive and relentless
neurodegeneration which manifests as loss of cognitive, motor, and
visual functions. Disease progression is rapid. The onset of
symptoms is typically between ages two and four. Patients typically
present initially with language delay and seizures, followed by
movement disorders, motor deterioration, dementia, blindness and
early death. During the later stages of the disease, feeding and
tending to everyday needs become very difficult and death typically
occurs between ten and 16 years of age.
There is no approved treatment that can prevent, stop, or
reverse 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.
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, the expectations regarding
the impact of EMA Accelerated Assessment program, timing of
regulatory filings with the FDA and EMA and its global regulatory
process in general. 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 FDA and EMA 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 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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