FDA grants Breakthrough Therapy designation to Genzyme’s olipudase alfa

Posted: 4 June 2015 |

The FDA has granted Breakthrough Therapy designation to Genzyme’s olipudase alfa for the treatment of Niemann-Pick disease type B…

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Genzyme’s olipudase alfa.


This enzyme replacement therapy is being investigated for the treatment of patients with non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B, as opposed to type A which is characterised by neurological involvement. ASMD is a serious and life-threatening disorder caused by insufficient activity of the enzyme acid sphingomyelinase (ASM), which results in toxic accumulation of sphingomyelin. There are currently no approved treatment options for patients with Niemann-Pick disease type B.

programmeOlipudase 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.

Breakthrough designation supported by data from a Phase1b study of olipudase alfa

The Breakthrough Therapy designation is supported by data from a completed Phase 1b study of olipudase alfa. Findings in five adult patients with non-neuronopathic 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 non-neurological manifestations of ASMD.

The company has started enrollment of a Phase 1/2 paediatric study and is preparing for enrollment of a Phase 2/3 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, Ph.D. “We appreciate FDA’s support for this important programme 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.”

Related organisations

Comments are closed.

Send this to a friend