Synageva Biotech Corp., a Lexington biopharmaceutical company developing therapies for rare diseases, said Monday that the Food and Drug Administration has granted breakthrough therapy designation to its drug candidate for the treatment of the early onset form of LAL deficiency.
The drug candidate is called sebelipase alfa.
Lysosomal acid lipase deficiency (or LAL deficiency) is a rare disorder caused by a marked decrease in LAL enzyme activity. Early onset LAL deficiency, sometimes called Wolman disease, is the most rapidly progressive form of LAL deficiency and is usually fatal within the first six months of life.
According to the FDA, breakthrough therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions.
In a statement, Synageva chief medical officer Anthony Quinn: “We are deeply aware of the devastating impact this disease has on infants who often die within the first six months of life because of this disease. Our ongoing Phase 2/3 trial delivers hope for these infants and their families. We continue to progress site activation and patient enrollment in both this trial and the global Phase 3 ARISE trial in children and adults, and look forward to working closely with the FDA to support approval of the drug in an efficient manner.”