Elelyso taliglucerase alfa regulatory update

Pfizer and partner Protalix said the European Commission refused to approve an MAA for Orphan Drug Elelyso taliglucerase alfa to treat Type I Gaucher's disease. In June, EMA's CHMP recommended against approval of the MAA. The committee said Elelyso has a positive benefit-risk profile but approval of the product would violate the existing EU Orphan drug exclusivity for a similar enzyme replacement therapy, Vpriv velaglucerase alfa from Shire plc (LSE:SHP; NASDAQ:SHPGY, Dublin, Ireland). Pfizer had been seeking an exemption to Vpriv's Orphan exclusivity on the basis of an "insufficient supply" of enzyme replacement therapy for Gaucher's disease and Elelyso's expected "major contribution to patient care" (see BioCentury, June 25).

Vpriv, a glucocerebrosidase enzyme replacement therapy, was approved in the EU in 2010 and has 10 years of exclusivity. CHMP had said Elelyso was not eligible for an exemption because there is no evidence that the product is clinically superior to Vpriv or that Vpriv is in short supply. ...