FDA Accepts BLA for BioMarin’s Cerliponase Alfa for CLN2 Disease, Form of Batten Disease
July 27 2016 - 6:02PM
Potential First Treatment for Fatal, Rare, Brain
Disease in Children
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the
U.S. Food and Drug Administration (FDA) accepted for review the
submission of a Biologics License Application (BLA) for cerliponase
alfa, an investigational therapy to treat children with CLN2
disease, a form of Batten disease. The Prescription Drug User
Fee Act (PDUFA) goal date for a decision is January 27, 2017.
BioMarin also has submitted a Marketing Authorization Application
(MAA) to the European Medicines Agency (EMA) for cerliponase alfa,
and it is undergoing validation at the Agency.
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 company also announced that the preliminary approved brand
name for cerliponase alfa is Brineura™.
“CLN2 disease is a rapidly progressing, fatal neurodegenerative
disease with no approved treatments. The FDA recognized the
potential of cerliponase alfa to help address this devastating
condition, and we look forward to working closely with the Agency
over the coming months," said Hank Fuchs, M.D., Chief Medical
Officer of BioMarin. "We thank the community for its continued
support, as well as the patients and families who dedicated their
time to the clinical development of cerliponase alfa.”
"This is an historic milestone for the Batten disease community.
For the first time since it was originally described more than a
century ago, there is a potential treatment for our children with
CLN2 disease,” said Margie Frazier, PhD, Executive Director of the
Batten Disease Support and Research Association. “We appreciate
BioMarin’s continued commitment to pursue a therapy for this
devastating disease and to advancing it quickly through the
development process."
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 8 and 12 years of age.
Early Access Program
BioMarin is planning to implement an early access program to
provide access to treatment for additional CLN2 patients prior to
obtaining marketing approval. The program will be limited in
scope and number of participants and will be conducted under a
protocol. BioMarin expects that the program will be conducted
initially 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 timing of the start of the program is on track, and the
initial site is expected to be open during Q3 2016. The exact
timing will vary by country of the sites participating. The overall
scope and details of this program are still being determined.
BioMarin must adhere to specific legal procedures for each country
and has begun these preparations with the goal of being ready to
dose patients in Q3 2016.
About Cerliponase Alfa
Cerliponase alfa 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) by
intracerebroventricular (ICV) infusion, an established technique
for delivering drugs to the brain.
For additional information regarding the investigational product
cerliponase alfa, 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. 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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