Genzyme, a Sanofi company, recently announced that its investigational enzyme-replacement therapy was granted Breakthrough Therapy designation from US health regulators.
The company said that the agency granted the status to its olipudase alfa for the treatment of patients with nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B, as opposed to type A, which is characterized by neurological involvement.
Winning Breakthrough status provides the company with a speedy review process, as well as access to the FDA for guidance on the olipudase alfa development program.
ASMD is a rare, serious and life-threatening disorder caused by insufficient activity of the enzyme acid sphingomyelinase (ASM), which is required to metabolize a lipid called sphingomyelin. If ASM is absent or not functioning properly, sphingomyelin cannot be metabolized properly and is accumulated within the cell, eventually causing cell death and the malfunction of major organ systems.
Olipudase alfa is being developed to potentially address the fundamental defect underlying ASMD. According to Genzyme, supplementing the defective or deficient native enzyme with olipudase alfa allows for the breakdown of sphingomyelin. The company has started enrolling patients in the Phase I/II pediatric study and is preparing to enroll patients in a Phase II/III adult study in the second half of 2015.
“There is tremendous unmet need in the ASMD/Niemann-Pick disease type B community, and we are hopeful that olipudase alfa can be developed into a meaningful treatment for patients,” said Genzyme’s Global Head of Rare Diseases, Richard Peters, MD, PhD. “We appreciate FDA’s support for this important program giving us the opportunity to utilize an important expedited drug development pathway for olipudase alfa and providing hope for patients affected with a chronic and progressively debilitating disease.”
The Breakthrough designation was based on results from a Phase Ib study evaluating the drug. Findings in five adult patients with nonneuronophatic ASMD were presented in February at the Lysosomal Disease Network’s WORLD Symposium.
Last updated: 6/8/15; 11:00am EST