Sanofi company Genzyme has received breakthrough therapy designation from the US Food and Drug Administration (FDA) for its enzyme replacement therapy, olipudase alfa.
Olipudase alfa is being developed to treat patients with nonneurological manifestations of acid sphingomyelinase deficiency (ASMD) or Niemann-Pick disease type B. It is opposed to type A that is characterised by neurological involvement.
ASMD is a serious and life-threatening disorder that is induced by insufficient activity of the enzyme acid sphingomyelinase (ASM), which results in toxic accumulation of sphingomyelin.
Genzyme rare diseases global head Dr Richard Peters said: “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.
“We appreciate FDA’s support for this important program giving us the opportunity to utilise an important expedited drug development pathway for olipudase alfa and providing hope for patients affected with a chronic and progressively debilitating disease.”
The FDA granted the breakthrough therapy status based on the data from a completed Phase Ib study of olipudase alfa.
The trial included five adult patients with nonneuronopathic ASMD and data presented on the repeat-dose safety, pharmacodynamics, and exploratory efficacy of olipudase alfa supported the firm’s continued development for the investigational use in nonneurological manifestations of ASMD.
Genzyme already commenced enrolment of patients in the Phase I/II paediatric study and is planning to start Phase II/III adult study in the second half of this year.
Image: The entrance to the Genzyme building at 500 Kendall Square in Cambridge, Massachusetts. Photo: courtesy of Tim Pierce.