Zacks_Analysts' Blog : Pfizer/Protalix Seek EU Approval - Analyst Blog

Pfizer (PFE) and its partner Protalix BioTherapeutics Inc. (PLX) recently announced that they have submitted the Marketing Authorization Application (MAA) for their Gaucher disease candidate in the EU. The companies are seeking marketing approval for Uplyso (taliglucerase alfa) from the European Medicines Agency.

Uplyso is currently under regulatory review in the US with a response from the Food and Drug Administration (FDA) expected by Feb 25, 2011. Uplyso enjoys orphan drug status in both the EU and the US for the Gaucher disease indication.

The approval of Uplyso will be a major boost for Protalix which has a strong marketing partner in Pfizer. The companies entered into a commercialization agreement in late 2009. Per the terms of the agreement, Pfizer has exclusive worldwide rights for the commercialization of Uplyso in all territories excluding Israel.

Gaucher Market

Gaucher disease, a genetically transmitted disease, has an incidence of approximately 1 in 20,000 live births. Genzyme’s (GENZ) Cerezyme holds a leading position in the treatment of Gaucher disease.

Another important player in the market is Shire Plc’s (SHPGY) Vpriv (velaglucerase alfa), which gained approval in the US as well as the EU in 2010. The potential approval of Protalix's Uplyso, expected early next year, should intensify competition in the Gaucher disease market, where the patient population is not very large. We note that both Vpriv and Uplyso were made available to patients under the FDA’s expanded access program due to a shortage in Cerezyme supplies.

Neutral on Protalix

We currently have a Neutral recommendation on Protalix which is supported by a Zacks #3 Rank (short-term “Hold” rating). We expect investor focus to remain on the approvability status of Ulypso.