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Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today announced sebelipase alfa and lysosomal acid lipase deficiency (LAL Deficiency) data from oral and poster presentations at the LDN WORLD Symposium being held February 11-13 in San Diego, California.
Initial results with sebelipase alfa from an ongoing Phase 2/3 trial in infants with LAL Deficiency
As part of an oral presentation, Vassili Valayannopoulos, MD, Clinical Lead of the Lysosomal Storage Disease Clinic at Necker-Enfants Malades Hospital, Paris, France, presented initial results from the ongoing Phase 2/3 trial with sebelipase alfa in infants with LAL Deficiency. Data from nine infants enrolled in the trial as of January 2014 were presented at the meeting. The median age of infants at first infusion with sebelipase alfa was 3.0 months. Infants continuing in the trial demonstrate improvements in weight gain and other disease-related abnormalities after starting treatment with weekly infusions of sebelipase alfa (1-3 mg/kg). Six infants continue to receive treatment with sebelipase alfa, including three infants who met the primary endpoint of survival at 12 months of age and three infants who continue on treatment beyond six months of age. Two infants died shortly after starting the study due to rapidly progressive disease not related to the study drug (sebelipase alfa). One infant died shortly after starting the study due to a non-study related procedure and also unrelated to sebelipase alfa.
Adverse events with sebelipase alfa were mostly mild to moderate. Serious adverse events were mainly related to central line infections or hospitalizations for treatment with antibiotics. One patient experienced three related serious adverse events that included fever, malaise, and tachycardia developed during the infusion of sebelipase alfa. Four infants developed anti-drug antibodies and all four continue on infusions with sebelipase alfa at 3 mg/kg.
"Sebelipase alfa represents a potentially dramatic breakthrough for infants with LAL Deficiency who often die without treatment, usually before six months of age,” said Dr. Valayannopoulos. “As an enzyme replacement therapy, sebelipase alfa addresses the underlying cause of this rapidly progressive and often fatal disease and we are excited to continue dosing these infants as part of the ongoing Phase 2/3 clinical trial.”