FDA approves Protalix, Pfizer’s ELELYSO for treatment of type 1 Gaucher disease

Pfizer Inc.(NYSE:PFE) and Protalix BioTherapeutics, Inc. (NYSE-AMEX:PLX, TASE:PLX) announced today that the United States (U.S.) Food and Drug Administration (FDA) approved ELELYSO™ (taliglucerase alfa) for injection, an enzyme replacement therapy (ERT) for the long-term treatment of adults with a confirmed diagnosis of type 1 Gaucher disease.    

ELELYSO is the first FDA-approved plant cell-based ERT for Gaucher disease. It is also the first approved plant cell-expressed drug that is derived from ProCellEx^®, Protalix's proprietary manufacturing system, using genetically engineered carrot cells. ELELYSO™ is a form of the human lysosomal enzyme, glucocerebrosidase, used to treat Gaucher disease.

"The approval of ELELYSO is important for patients who depend on available ERT to manage their Gaucher disease. At Protalix, our passion to develop ELELYSO was strongly driven by our personal experience with family members and friends who have to live with this disease every day," said David Aviezer, Ph.D., MBA, president and chief executive officer of Protalix BioTherapeutics. "We also believe that this great news is a recognition of our technology, which is a plant cell manufacturing system from Protalix. This technology is the production process behind ELELYSO and other Protalix product candidates."

Supply disruptions of approved ERTs have been affecting those living with Gaucher disease since 2009 in multiple countries including the U.S. To help minimize the possibility of supply disruptions, Pfizer is launching the "Supply Continuity Program," which will endeavor to maintain a continuously restocked 24 months of supply at various stages of production for U.S. patients prescribed ELELYSO.

With the approval of ELELYSO, Pfizer is also introducing Gaucher Personal Support (GPS), a specialized support program for people living with Gaucher disease. Through the GPS program, Pfizer will provide an assistance program covering 100 percent of prescription co-pay expenses for eligible patients on ELELYSO who have commercial health insurance. Additionally, Pfizer will provide financial assistance for eligible patients who are uninsured or under-insured where allowed by law.

GPS is a one-stop resource for patient support and specialty pharmacy services for those taking ELELYSO. GPS staffs a dedicated team of health care specialists who are available 24/7 to help Gaucher disease patients and their families with reimbursement assistance, facilitate locating infusion services, and provide ongoing pharmacy support. Patients can call 1-855-ELELYSO (1-855-353-5976) for a free patient information kit about ELELYSO and the GPS program.

"With ELELYSO, Pfizer is proud to be making a difference in the lives of people living with Gaucher disease," said Diem Nguyen, Ph.D., MBA, general manager, Pfizer Biosimilars. "ELELYSO is an important addition to Pfizer's rare disease portfolio."

There are currently two other ERTs approved for Gaucher disease in the U.S., imiglucerase and velaglucerase. Taliglucerase alfa for injection (ELELYSO) will be available to U.S. patients at a cost that will be priced at 25 percent below the cost of imiglucerase.

"I am delighted that the Gaucher community will have another treatment option. It is especially important for this group of patients who have suffered from supply shortages in recent years," said Rhonda Buyers, CEO and executive director of the National Gaucher Foundation (NGF).