FDA Grants Breakthrough Therapy Designation for Genzyme's Olipudase Alfa
Investigational Enzyme Replacement Therapy to Treat Non-Neurological Manifestations which characterize Niemann-Pick Type B
Breakthrough Therapy designation is intended to expedite the development and review of investigational new drugs that target serious or life-threatening conditions. The criteria for granting Breakthrough Therapy designation are preliminary clinical evidence of substantial improvement on a clinically significant endpoint over available therapies. The Breakthrough Therapy designation is distinct from the FDA's other mechanisms to expedite drug development and review, and will allow for a close collaboration between Genzyme and the FDA on the olipudase alfa development program.
Olipudase alfa is being developed by Genzyme to potentially address the fundamental defect underlying the disease. Supplementing the defective or deficient native enzyme with olipudase alfa allows for the breakdown of sphingomyelin, whose accumulation is responsible for the clinical manifestation of ASMD.
The Breakthrough Therapy designation is supported by data from a completed Phase 1b study of olipudase alfa. Findings in five adult patients with nonneuronopathic ASMD were presented at the Lysosomal Disease Network's WORLD Symposium in February 2015. The data presented on the repeat-dose safety, pharmacodynamics, and exploratory efficacy of olipudase alfa support its continued development for the investigational use in nonneurological manifestations of ASMD.
The company has started enrollment of a Phase 1/2 pediatric study and is preparing for enrollment of a Phase 2/3 adult study in the second half of 2015.
Most read news
Organizations
Other news from the department research and development
Get the life science industry in your inbox
By submitting this form you agree that LUMITOS AG will send you the newsletter(s) selected above by email. Your data will not be passed on to third parties. Your data will be stored and processed in accordance with our data protection regulations. LUMITOS may contact you by email for the purpose of advertising or market and opinion surveys. You can revoke your consent at any time without giving reasons to LUMITOS AG, Ernst-Augustin-Str. 2, 12489 Berlin, Germany or by e-mail at revoke@lumitos.com with effect for the future. In addition, each email contains a link to unsubscribe from the corresponding newsletter.