Pfizer Inc. / Protalix BioTherapeutics, Inc.

Type 1 Gaucher Disease

Disease Overview

Gaucher disease is an autosomal-recessive lysosomal storage disorder caused by deficient glucocerebrosidase, leading to glucocerebroside accumulation in macrophages. Clinical features include hepatosplenomegaly, anemia, thrombocytopenia, and bone disease. Type 1 (non-neuronopathic) is most common and treatable with enzyme replacement therapy (ERT).

Competitive Landscape

At deal signing in November 2009, Genzyme's Cerezyme (imiglucerase) dominated the global Gaucher ERT market with multi-hundred-million-dollar annual sales, but a Genzyme manufacturing contamination crisis in mid-2009 caused acute Cerezyme shortages and opened the door for new entrants. Shire's VPRIV (velaglucerase alfa) and Protalix's taliglucerase alfa were both being rushed through FDA review under expanded-access pathways. Pfizer's licensing of taliglucerase alfa positioned it as a third ERT option with a differentiated plant-cell (carrot-cell) manufacturing platform that promised lower cost-of-goods than Genzyme's CHO-based or Shire's human-cell systems. The deal also gave Pfizer a foothold in lysosomal storage disorders and a hedge against Cerezyme supply risk for payers and patients. Oral substrate-reduction therapies (Actelion's Zavesca/miglustat, later Sanofi's Cerdelga/eliglustat) competed in the niche of patients unsuitable for or preferring to avoid IV ERT. Taliglucerase alfa (later branded ELELYSO) ultimately gained FDA approval in May 2012 but commercial uptake was modest as Genzyme restored Cerezyme supply, prompting Protalix to sell its 40% collaboration share back to Pfizer in 2015.