According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for Breakthrough Therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. A Breakthrough Therapy designation conveys all of the fast track program features, as well as more intensive FDA guidance on an efficient drug development program.
We are pleased that the FDA designated sebelipase alfa as Breakthrough Therapy for patients with early onset LAL Deficiency, or Wolman disease,” said Anthony Quinn, MBChB, PhD, FRCP, Senior Vice President and Chief Medical Officer of Synageva. “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.”