Pfizer and Protalix have entered into an agreement to develop and commercialize taliglucerase alfa, a plant-cell expressed form of glucocerebrosidase (GCD) in development for the potential treatment of Gaucher’s disease. Under the terms of the agreement, Pfizer will receive exclusive worldwide licensing rights for the commercialization of taliglucerase alfa, while Protalix will retain the exclusive commercialization rights in Israel. Taliglucerase alfa is the first enzyme replacement therapy derived from a proprietary plant cell-based expression platform using genetically engineered carrot cells.
With the successful completion of phase-III clinical studies, Protalix is preparing to complete a rolling New Drug Application (NDA) with the US Food and Drug Administration (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 Authorization. The FDA has also requested, and subsequently approved, an Expanded Access Programme (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 European Union under a compassionate use protocol.
“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,” commented Dr David Aviezer, president and CEO of Protalix. “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 US$ 60 million to Protalix. In addition, Protalix is eligible to receive additional regulatory milestone payments of up to US$ 55 million. Pfizer and Protalix will share future revenues and expenses for the development and commercialization of taliglucerase alfa on a 60 per cent/40 per cent basis respectively.
“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,” said David Simmons, president and general manager of Pfizer’s Established Products Business Unit. “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.”
Peter L Saltonstall, president and CEO, National Organization for Rare Disorders (NORD) stated, "NORD is always pleased when treatment options are expanded for people with rare diseases. We welcome Pfizer’s commitment to the rare disease arena, and look forward to working with both Pfizer and Protalix in support of increased options for patients and families affected by rare diseases."
Protalix is a biopharmaceutical company focused on the development and commercialization of proprietary recombinant therapeutic proteins expressed through its proprietary plant cell based expression system.