Protalix will continue to manage, administer, and sponsor current, ongoing adult and pediatric extension studies of clinical trials relating to its Gaucher disease drug Elelyso™ (taliglucerase alfa) while development partner Pfizer agreed to oversee new clinical trials, as both companies continue pursuing approvals beyond the U.S. and Israel.
In return, under the companies’ clinical development, Protalix will be eligible to receive from Pfizer a milestone payment of $8.3 million tied to achieving undisclosed near-term clinical development milestones.
Elelyso is an enzyme replacement therapy approved in May for injection in the U.S. in adults with a confirmed diagnosis of type 1 Gaucher disease. Israel’s Ministry of Health approved Elelyso in September.
Protalix in a statement hailed the agreement as one that “helps maintain the continuity of the ongoing clinical trials for Gaucher patients and physicians and reinforces the companies’ mutual commitment to the Gaucher community.”
The two companies partnered to develop and commercialize Elelyso in 2009, with Israeli-owned Protalix holding development and commercialization rights in Israel and Pfizer, the rest of the world.
In Europe, however, the European Commission on November 1 rejected the marketing authorization application for Elelyso. The EC sided with the European Medicines Agency’s advisory Committee for Medicinal Products for Human Use, which noted that despite a positive risk-benefit assessment of the drug, it could not recommend ending Shire’s 10-year orphan market exclusivity, starting with the EC’s approval in August 2010 of another drug, velaglucerase alfa, indicated for the same disease. Pfizer sought to upend Shire’s orphan market exclusivity but was denied.