Pfizer, Protalix Enter Into Agreement To Develop Gaucher’s Disease Treatment

Pfizer and Protalix have entered into an agreement to develop and commercialise Taliglucerase Alfa, a plant-cell expressed form of glucocerebrosidase (GCD) in development for the potential treatment of Gaucher’s disease. Taliglucerase Alfa is the first enzyme replacement therapy derived from a proprietary plant cell-based expression platform using genetically engineered carrot cells.

Under the terms of the agreement, Pfizer will receive exclusive worldwide licensing rights for the commercialisation of Taliglucerase Alfa, while Protalix will retain the exclusive commercialisation rights in Israel.

With the successful completion of Phase III clinical studies, Protalix is preparing to complete a rolling New Drug Application (NDA) with the FDA. The FDA has granted Orphan Drug designation and Fast Track status, facilitating the development and expediting the review of drugs to treat rare conditions or diseases, as well as an emergency use authorisation.

The FDA has also requested, and subsequently approved, an Expanded Access Program (EAP) treatment protocol. Taliglucerase Alfa is currently being provided to Gaucher’s patients in the US under the EAP protocol, as well as to patients in the EU under a compassionate use protocol.

David Aviezer, president and CEO of Protalix, said: “We are excited about this collaboration, which represents a significant step towards bringing, for the first time, a plant-based enzyme replacement treatment option to patients affected by Gaucher’s disease.

“By joining our advances in biologics manufacturing and protein development with Pfizer’s global strengths in patient services and reimbursement we expect to help make Taliglucerase Alfa an important and cost-effective treatment choice for Gaucher’s patients throughout the world.”

Under the agreement, Pfizer will make an upfront payment of $60m to Protalix. In addition, Protalix is eligible to receive additional regulatory milestone payments of up to $55m. Pfizer and Protalix will share future revenues and expenses for the development and commercialisation of Taliglucerase Alfa on a 60% / 40% basis respectively.

David Simmons, president and general manager of established products business unit at Pfizer, said: “By combining our respective strengths to advance this innovative therapy, Pfizer and Protalix expect to quickly deliver an alternative treatment for people suffering from Gaucher’s disease. This agreement supports our goal to meet the needs of many patient populations, including those affected by rare diseases, and brings the best minds together to challenge the most feared diseases of our time.”