LEXINGTON, Mass., May 26, 2015 /PRNewswire/ -- Synageva
BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company
developing therapeutic products for rare disorders, today announced
the submission of a New Drug Application (NDA) to the Ministry of
Health, Labour and Welfare (MHLW) in Japan for Kanuma™ (sebelipase alfa) as a
treatment for patients with lysosomal acid lipase deficiency (LAL
Deficiency). Upon approval, Kanuma would be the first approved
therapy for these patients.
The NDA for Kanuma included previously reported data from the
global, randomized, double-blind, placebo controlled Phase 3 trial
in children and adults with LAL Deficiency, which included
enrollment of Japanese patients, and the Phase 2/3 trial of Kanuma
in infants with LAL Deficiency. The MHLW previously granted
Orphan Drug Designation (ODD) to sebelipase alfa for the treatment
of patients with LAL Deficiency. As a result, this NDA will receive
priority review for marketing authorization, and, if approved,
sebelipase alfa would have 10 years of market exclusivity for the
designated indication.
About LAL Deficiency
LAL Deficiency is a serious, life-threatening disease associated
with early mortality and significant morbidity. LAL Deficiency
is a chronic disease in which genetic mutations result in decreased
activity of the LAL enzyme; this leads to marked accumulation of
lipids in vital organs, blood vessels, and other tissues, resulting
in progressive and multi-systemic organ damage including fibrosis,
cirrhosis, liver failure, accelerated atherosclerosis,
cardiovascular disease, and other devastating consequences. LAL
Deficiency affects patients of all ages with sudden and
unpredictable clinical complications manifesting from infancy
through adulthood. The decreased LAL enzyme activity can be
diagnosed with a simple blood test.
About Kanuma
Kanuma is a recombinant form of the human LAL enzyme being
developed by Synageva as an enzyme replacement therapy for patients
with LAL Deficiency. Kanuma has been granted orphan
drug designation by the FDA, the European Medicines Agency
(EMA), and the Japanese Ministry of Health, Labour and
Welfare. Additionally, Kanuma received fast track designation
by the FDA, and Breakthrough Therapy designation by the FDA for LAL
Deficiency presenting in infants. The FDA accepted for review
the Biologics License Application (BLA) for Kanuma, granted the
company's request for Priority Review, and established a target
action date of September 8, 2015
under the Prescription Drug User Fee Act (PDUFA). The EMA validated
the Marketing Authorization Application (MAA) for Kanuma and
granted the company's request for accelerated
assessment.
About Synageva
Synageva is a biopharmaceutical company focused on the
discovery, development, and commercialization of therapeutic
products for patients with rare diseases. The company's pipeline
consists of protein therapeutic programs for rare diseases with
unmet medical need which are currently at various stages of
development. The company is planning for a global launch of
Kanuma for the treatment of LAL Deficiency and is dosing patients
in a Phase 1/2 trial with its second, first-mover program, SBC-103
for MPS IIIB. The company's third, first-mover program,
SBC-105, is an enzyme replacement therapy in preclinical
development for disorders of calcification. In addition to
these first-mover programs, the pipeline also consists of
opportunities that leverage the company's manufacturing platform
and other capabilities to create potentially bio-superior
treatments for patient populations where there is still unmet
medical need. The company has recently produced enzymes
targeting Hunter syndrome, Fabry disease and Pompe disease with
expression levels and activity that support further preclinical
development.
Synageva routinely posts information that may be important to
investors in the Investor Relations section of the company's
website at www.synageva.com. Synageva encourages investors
and potential investors to consult this website regularly for
important information about the company.
Medical information regarding Kanuma and LAL Deficiency is
available by email at medinfo@synageva.com.
Forward-Looking Statements
This news release contains "forward-looking statements".
Such statements generally can be identified by the use of words
such as "anticipate," "expect," "plan," "could," "intend,"
"believe," "may," "will," "estimate," "forecast," "project," or
words of similar meaning. These forward-looking statements address,
among other matters, potential approval and plans for the potential
launch of Kanuma, the ability for the company's manufacturing
platform to create potentially bio-superior treatments, and plans
to further support preclinical development for a potentially
bio-superior program. Many factors may cause actual results to
differ materially from forward-looking statements, including
inaccurate assumptions and a broad variety of risks and
uncertainties, some of which are known, such as, unanticipated
costs or delays in the company's research and development programs,
risk of delays in completing the company's preclinical and clinical
trials, risk that the outcomes of the preclinical or clinical
trials may not support registration or further development of the
company's product candidates due to safety, efficacy or other
reasons, the timing for initiating and completing potential
preclinical and clinical studies, the timing for reporting data
from potential preclinical and clinical studies, the content and
timing of decisions by the FDA, EMA and other regulatory
authorities, our ability to prepare for potential commercial launch
of Kanuma, the ability to continue to produce favorable product
attributes and characteristics expressed using the company's
proprietary platform, and the risks identified under the heading
"Risk Factors" in the company's annual report on Form 10-Q for the
fiscal quarter ending March 31, 2015
filed with the Securities and Exchange Commission (SEC) and other
filings Synageva periodically makes with the SEC, and others of
which are not known. Preclinical and clinical trial data are
subject to differing interpretations, and regulatory agencies, as
well as medical and scientific experts, may not share Synageva's
views regarding these data or its implications. Synageva may
encounter problems or delays in preclinical and clinical
development and the regulatory process. No forward-looking
statement is a guarantee of future results or events, and investors
should avoid placing undue reliance on such statements.
Synageva undertakes no obligation to update any forward-looking
statements, whether as a result of new information, future events
or otherwise. Our business is subject to substantial risks
and uncertainties, including those referenced above. Investors,
potential investors, and others should give careful consideration
to these risks and uncertainties.
"Synageva BioPharma™" and "Kanuma™" are trademarks, and
"Dedicated to Rare Diseases®" is a registered trademark, of
Synageva BioPharma Corp.
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/synageva-biopharma-submits-kanuma-sebelipase-alfa-application-for-lal-deficiency-in-japan-300088814.html
SOURCE Synageva BioPharma Corp.