Pfizer and partner Protalix BioTherapeutics have filed taliglucerase alfa, with regulators in Europe for the treatment of Gaucher disease.
They have submitted an application to the European Medicines Agency for the drug, a plant-cell expressed form of glucocerebrosidase, which Pfizer licensed from the Israeli firm in November last year for the treatment of Gaucher disease. Taliglucerase alfa was granted orphan designation by the European Commission in March.
The drug is currently being provided to Gaucher’s patients in the USA and in the European Union under a compassionate use protocol, which was triggered by the global supply shortage caused by Genzyme Corp temporarily shutting down its manufacturing facility in Boston last year, thus affecting production of Cerezyme (imiglucerase), the standard of care. Taliglucerase alfa has a Prescription Drug User Fee Act action date of February 25 next year in the USA.
Taliglucerase alfa, if approved, will be competing with Cerezyme and Shire's Vpriv (velaglucerasealfa) which was approved in Europe in August and given the green light across the Atlantic in February. Although Gaucher is a rare disease, which can lead to the build-up of fatty substances in organs including the spleen, liver, kidney and brain, resulting in a whole host of complications, the market is a lucrative one. Third-quarter sales of Vpriv reached $50 million, while Cerezyme sales for that period nearly doubled to $179.8 million.