The Food and Drug Administration (FDA) has granted Genzyme a breakthrough therapy designation for a drug called olipudase alfa, an enzyme-replacement therapy intended for the treatment of Niemann-Pick Disease Type B (NPB) otherwise known as acid sphingomyelinase deficiency.
Genzyme touted the label in a statement,explaining that this life-threatening disease occurs when there is, “insufficient activity of the enzyme acid sphingomyelinase (ASM), which results in toxic accumulation of sphingomyelin.”
An inability to metabolize sphingomyelin, along with a buildup of the lipid within the cell,s can lead to cell death and malfunctioning of major organ systems, according to the company’s announcement.
Olipudase alfa is being designed to address the underlying cause of NPB and serve as a substitute for the missing enzyme so the body can breakdown the sphingomyelin. There are currently no approved treatments for this disorder.
FierceBiotech reports the FDA decided to give the tag to Genzyme after five patients demonstrated early signs of efficacy in a Phase 1b trial.
A breakthrough therapy designation will help Genzyme quickly move through the rest of the regulatory process.
Meanwhile, the drugmaker has begun enrollment for a Phase 1/2 pediatric study of olipudase alfa and is preparing to launch a Phase 2/3 adult study in the second half of 2015.
Filed Under: Drug Discovery