Synageva BioPharma Completes Rolling Submission Of Biologics License Application To The FDA For Sebelipase Alfa And Submits Marketing Authorization Application To European Medicines Agency
LEXINGTON, Mass., Dec. 2, 2014 /PRNewswire/ -- Synageva BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today the completion of the rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for sebelipase alfa as a treatment for patients with lysosomal acid lipase deficiency (LAL Deficiency), a rare genetic disease with significant morbidity and early mortality. Included in the submission is a request for Priority Review which, if granted and the BLA is accepted for review, would shorten the FDA's anticipated regulatory review time from approximately 12 to 8 months.
The company also announced the submission of a Marketing Authorization Application (MAA) in the European Union for sebelipase alfa for LAL Deficiency, which is subject to the validation process by the European Medicines Agency (EMA). The EMA recently granted the company's request for accelerated assessment, which has the potential to shorten the EMA's regulatory review time.
The BLA and MAA include previously reported data from the global, randomized, double-blind, placebo controlled Phase 3 trial of sebelipase alfa in children and adults with LAL Deficiency, and the Phase 2/3 trial of sebelipase alfa in infants with LAL Deficiency. Patients in these trials, combined with patients in other ongoing clinical trials with sebelipase alfa, represent the largest patient population studied to date for this rare, devastating disease.
Positive results from the global, Phase 3, double-blind, placebo-controlled trial evaluating sebelipase alfa in children and adults with LAL Deficiency were presented during the American Association for the Study of Liver Diseases (AASLD) on November 10, 2014. The trial met the primary endpoint of ALT normalization (a marker of liver injury) and multiple secondary endpoints, and demonstrated significant improvements in multiple disease-related parameters of dyslipidemia and liver injury compared with placebo. Most adverse events during the double-blind treatment period were mild and unrelated to sebelipase alfa.
Positive results were also previously reported from the ongoing, open-label Phase 2/3 trial of sebelipase alfa in infants with LAL Deficiency. Six infants met the primary endpoint of survival at 12 months of age and five infants continue on treatment with sebelipase alfa. Three infants died shortly after the start of the study, unrelated to sebelipase alfa, and one infant who met the primary endpoint of survival at 12 months of age subsequently died at 15 months of age considered unlikely related to treatment with sebelipase alfa. Adverse events with sebelipase alfa were mostly mild to moderate.
Sebelipase Alfa for LAL Deficiency
LAL Deficiency is a serious, underdiagnosed disease with significant morbidity and early mortality. LAL Deficiency causes progressive and multisystemic organ damage including hepatic cirrhosis and accelerated atherosclerosis that can lead to sudden and unpredictable clinical complications. LAL Deficiency often manifests in childhood but can be diagnosed at all ages with a simple blood test. LAL Deficiency is caused by genetic mutations that result in decreased LAL enzyme activity in the lysosomes across multiple body tissues, leading to the buildup of fatty material in the liver, blood vessel walls and other tissues.
Sebelipase alfa is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency. Sebelipase alfa has been granted orphan designation by the FDA, the EMA, and the Japanese Ministry of Health, Labour and Welfare. Additionally, sebelipase alfa received fast track designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants.
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.
Further information regarding Synageva is available at www.synageva.com.
Medical information regarding sebelipase alfa and LAL Deficiency is available by email at [email protected].
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, the potential for the FDA to grant Priority Review, and the potential that the FDA's and EMA's regulatory review time periods may be shortened. 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, the content and timing of decisions by the FDA, EMA and other regulatory authorities, and the risks identified under the heading "Risk Factors" in Synageva's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on October 30, 2014 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 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.
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