LEXINGTON, MA, USA I February 23, 2015 I Synageva BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for Kanuma™ (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency (LAL Deficiency). The FDA granted the company’s request for Priority Review, which shortens the regulatory review period and is reserved for investigational therapies that treat serious conditions and, if approved, would provide a significant improvement in safety or effectiveness compared to available therapies. The FDA established a target action date of September 8, 2015 under the Prescription Drug User Fee Act (PDUFA).
The BLA for Kanuma included previously reported data from the global, randomized, double-blind, placebo controlled Phase 3 trial in children and adults with LAL Deficiency, and the Phase 2/3 trial of Kanuma in infants with LAL Deficiency. Patients in these trials, combined with patients in other ongoing clinical trials with Kanuma, represent the largest patient population studied to date for this rare, devastating disease.
Kanuma and LAL Deficiency
LAL Deficiency is a serious and life-threatening disease that can be diagnosed with a simple blood test. LAL Deficiency causes progressive and multisystemic organ damage including 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. 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.
Kanuma is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency. Kanuma has been granted orphan 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 BLA for Kanuma and granted the company’s request for Priority Review. 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 initiated a Phase 1/2 trial with its second, first-mover program, SBC-103, an enzyme replacement therapy for patients with mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome). The company’s third, first-mover program, SBC-105, is an enzyme replacement therapy in preclinical development for rare disorders of calcification, including generalized calcification in infants (GACI). 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.
SOURCE: Synageva BioPharma
Post Views: 189
LEXINGTON, MA, USA I February 23, 2015 I Synageva BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for Kanuma™ (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency (LAL Deficiency). The FDA granted the company’s request for Priority Review, which shortens the regulatory review period and is reserved for investigational therapies that treat serious conditions and, if approved, would provide a significant improvement in safety or effectiveness compared to available therapies. The FDA established a target action date of September 8, 2015 under the Prescription Drug User Fee Act (PDUFA).
The BLA for Kanuma included previously reported data from the global, randomized, double-blind, placebo controlled Phase 3 trial in children and adults with LAL Deficiency, and the Phase 2/3 trial of Kanuma in infants with LAL Deficiency. Patients in these trials, combined with patients in other ongoing clinical trials with Kanuma, represent the largest patient population studied to date for this rare, devastating disease.
Kanuma and LAL Deficiency
LAL Deficiency is a serious and life-threatening disease that can be diagnosed with a simple blood test. LAL Deficiency causes progressive and multisystemic organ damage including 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. 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.
Kanuma is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency. Kanuma has been granted orphan 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 BLA for Kanuma and granted the company’s request for Priority Review. 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 initiated a Phase 1/2 trial with its second, first-mover program, SBC-103, an enzyme replacement therapy for patients with mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome). The company’s third, first-mover program, SBC-105, is an enzyme replacement therapy in preclinical development for rare disorders of calcification, including generalized calcification in infants (GACI). 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.
SOURCE: Synageva BioPharma
Post Views: 189
