Synageva BioPharma Corp., a biopharmaceutical company developing therapeutic products for rare disorders, announced validation by the European Medicines Agency (EMA) of the Marketing Authorization Application (MAA) for sebelipase alfa for LAL Deficiency.  The EMA recently granted the company's request for accelerated assessment, which has the potential to shorten the EMA's regulatory review time.  

The MAA, and the Biologics License Application (BLA) submitted to the US Food and Drug Administration (US FDA), include previously reported data from the global, randomised, 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.

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.