FDA Grants Breakthrough Designation to Genzyme’s Experimental ASMD Treatment

Published: Jun 08, 2015

FDA Grants Breakthrough Designation to Genzyme’s Experimental ASMD Treatment
June 4, 2015
By Alex Keown, BioSpace.com Breaking News Staff

CAMBRIDGE, Mass. – The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Genzyme Corporation’s olipudase alfa, an enzyme replacement therapy being investigated for the treatment of patients with nonneurological manifestations of acid sphingomyelinase deficiency (ASMD).

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, the company said.

ASMD, also known as Niemann-Pick disease type B, as opposed to type A which is characterized by neurological involvement, is a sometimes fatal 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. 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.

“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,” Richard Peters, Genzyme’s head of rare diseases, said in a statement.

ASMD, or Niemann-Pick disease type A and B, occur as a result of a deficiency of the enzyme acid sphingomyelinase, which is needed to break down sphingomyelin, a fatty substance found mostly in the brain and nervous system, according to WebMD.

ASMD results in excessive accumulation of sphingomyelin in various bodily organs, including the liver, spleen and brain. In total, at least five types of Niemann-Pick disease have been identified, types A, B, C, D and E. The disease typically hits children ranging from infancy to adolescence, depending on the disease type.

Investors reacted positively to the FDA’s action, sending stock prices for Sanofi, Genzyme’s parent company, to a morning high of $51.52 per share.

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 FDA’s designation came following a five-patient Phase Ib study of olipudase alfa. The study findings showed that the dose escalation regimen was well tolerated, with all patients, with no serious side effects.

“Though a small number of patients, the response we have observed to date is an early indication that this ASM enzyme replacement therapy is promising for this therapeutic area,” Peters said in a statement following the release of the study data.

The company has started enrollment of a Phase I/II pediatric study and is preparing for enrollment of a Phase II/III adult study in the second half of 2015, the company said.

This is the second FDA award in as many months for Genzyme. In April the FDA granted Fast Track designation for the development of GZ/SAR402671, a new investigational oral substrate reduction therapy for the treatment of Fabry disease, a rare lysosomal storage disorder that results in abnormal tissue deposits of a particular fatty substance called globotriaosylceramide, throughout the body.

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