BioMarin Receives European Orphan Drug Designation for BMN 270, First Investigational AAV-Factor VIII Gene Therapy for Patien...
March 24 2016 - 8:30AM
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that BMN
270, an investigational gene therapy for the treatment of
hemophilia A, has been granted orphan drug designation by the
European Commission. In the European Union, orphan drug
designation is given to treatments that are intended for
life-threatening or chronically-debilitating conditions with a
prevalence of not more than 5 in 10,000 people. Earlier this month,
BioMarin announced BMN 270 had also received orphan drug
designation from the U.S. Food and Drug Administration.
BMN 270 is an AAV 5 factor VIII vector designed to restore
factor VIII plasma concentrations, essential for blood clotting in
patients with hemophilia A. BioMarin is currently conducting a
Phase 1/2 study to evaluate the safety and efficacy of BMN 270 gene
therapy in patients with severe hemophilia A.
Study Design
The Phase 1/2 study will evaluate the safety and efficacy of BMN
270 gene therapy in up to 12 patients with severe hemophilia A. The
primary endpoints are to assess the safety of a single intravenous
administration of a recombinant AAV, human-coagulation factor VIII
vector and to determine the change from baseline of factor VIII
expression level at 16 weeks after infusion. The kinetics, duration
and magnitude of AAV-mediated factor VIII activity in individuals
with hemophilia A will be determined and correlated to an
appropriate BMN 270 dose.
This is a dose escalation study with the goal of observing an
increase in factor VIII levels. Secondary endpoints include
assessing the impact of BMN 270 on the frequency of factor VIII
replacement therapy, the number of bleeding episodes requiring
treatment and any potential immune responses. Patients will be
monitored for safety and durability of effect for five years.
About Hemophilia A
Hemophilia A, also called factor VIII (FVIII) deficiency or
classic hemophilia, is a genetic disorder caused by missing or
defective factor VIII, a clotting protein. Although it is passed
down from parents to children, about 1/3 of cases are caused by a
spontaneous mutation, a new mutation that was not
inherited.1 Approximately 1 in 10,000 people is born with
hemophilia A.2 People living with the disease are not able to
form blood clots efficiently and are at risk for excessive bleeding
from modest injuries, potentially endangering their life. People
with severe hemophilia often bleed spontaneously into their muscles
or joints. The standard of care for the 43 percent of hemophilia A
patients, who are severely affected, is a prophylactic regimen of
factor VIII infusions three times per week.3 Even with
prophylactic regimens, many patients still experience spontaneous
bleeding events that result in progressive and debilitating joint
damage.
About Gene Therapy
Gene therapy is a form of treatment designed to fix a genetic
problem by adding a corrected copy of the defective gene. The
functional gene is inserted into a vector – containing a small DNA
sequence – that acts as a delivery mechanism, providing the ability
to deliver the functional gene to cells. The cells can then use the
information to build the functional proteins that the body needs,
potentially reducing or eliminating the cause of the disease.
Currently, gene therapy for the treatment of hemophilia A is
available only as part of a clinical trial.
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.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 the
development of BioMarin's BMN 270 program generally and the timing
and results of the clinical trial of BMN 270. 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 preclinical studies and clinical
trials of BMN 270; the content and timing of decisions by the U.S.
Food and Drug Administration, the European Commission and other
regulatory authorities; our ability to successfully manufacture the
product candidate for the preclinical and clinical trials; 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, and the factors contained in BioMarin's
reports on Form 10-Q. 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.
1 Source: National Hemophilia
Foundationhttp://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A
2 Source: World Federation of
Hemophiliahttp://www.wfh.org/en/page.aspx?pid=637#How_common
3 Source: World Federation of
Hemophiliahttp://www.wfh.org/en/resources/annual-global-surveyhttp://www.wfh.org/en/abd/prophylaxis/prophylaxis-administration-and-dosing-schedules
Contact:
Investors:
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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