Good news for Protalix Biotherapeutics (PLX) once again! The company completed the submission of its New Drug Application (NDA) with the US Food and Drug Administration (FDA) for taliglucerase alfa, which is meant for the treatment of Gaucher disease. It has been granted orphan-drug designation and fast-track status by the FDA to facilitate its development.
Taliglucerase alfa is currently being given to patients suffering from Gaucher disease in the US and the European Union under the Expanded Access Program and compassionate use protocol, respectively.
Earlier this month, the company entered into an agreement with Pfizer (PFE) related to the development and commercialization of this drug. As per the deal, Pfizer will have exclusive worldwide licensing rights to commercialize taliglucerase alfa while Protalix has retained its commercialization rights in Israel. Under the agreement, Pfizer will also retain 60% ownership of the drug’s revenue and expenses. Accordingly, the company will pay $60 million upfront to Protalix and up to $55 million in additional milestone payments.
Protalix is going ahead with its plan for the drug’s use in pediatric patients, as well. The company filed its proposed pediatric investigation plan to the pediatric committee of the European Medicines Agency (EMEA) for a clinical study of the drug in patients of 2 to 18 years of age. The filing of the plan triggers a milestone payment of $5 million from Pfizer as per the deal.
Gaucher disease is a rare inherited condition, caused by the deficiency of a particular enzyme. It damages the liver and bone marrow causing anemia, excessive bleeding and bruising, among other symptoms.
Competition in the Gaucher disease market is quite strong with the presence of drugs such as Cerezyme from Genzyme (GENZ) and Shire plc’s (SHPGY) velaglucerase alfa, which is currently available under the FDA’s expanded access program. However, we believe Protalix’s taliglucerase alfa to be a formidable competitor in the market, backed by Pfizer’s marketing muscle.
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