Pfizer and Protalix BioTherapeutics announce FDA approval Of ELELYSO™ (taliglucerase alfa)

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).

Please see Safety Information for ELELYSO below as well as the full Prescribing Information here >>

ELELYSO™ Clinical Data

The approval of ELELYSO™ (taliglucerase alfa) is based on the review of Protalix’s clinical development program.

In a study of 31 adult patients with Type 1 Gaucher disease naïve to enzyme replacement therapy, the safety and efficacy of ELELYSO was assessed. The trial was a nine-month, multi-center, double blind, randomized study in patients with Gaucher disease-related enlarged spleens and thrombocytopenia. Patients were randomized to receive ELELYSO at a dose of either 30 Units/kg (n=15) or 60 Units/kg (n=16). Data showed the pivotal phase III clinical trial achieved its primary endpoint as ELELYSO significantly reduced spleen volume after nine months compared to baseline in both treatment groups. Secondary endpoints of liver volume, hemoglobin and platelet counts also showed improvement.

Twenty-six patients continued to be treated with ELELYSO in an extension of this study in a blinded manner for a total treatment duration of 24 months. The data demonstrated continued improvement in the clinical parameters.

In a study of 25 patients with Type 1 Gaucher disease who were switched from imiglucerase to ELELYSO, the safety and efficacy of ELELYSO was assessed. The trial was a nine-month, multi-center, open-label, single arm study in patients who had been receiving treatment with imiglucerase at doses ranging from 11 Units/kg to 60 Units/kg for a minimum of 2 years. Imiglucerase therapy was stopped, and treatment with ELELYSO was administered every other week at the same number of units as each patient’s previous imiglucerase dose. Organ volumes and hematologic values remained stable on average through nine months of ELELYSO treatment.

The most common adverse reactions during clinical studies were infusion reactions. Other commonly observed adverse reactions in less than ten percent of patients were URTI/nasopharyngitis, pharyngitis/throat infection, headache, arthralgia, influenza/flu, UTI/pyelonephritis, back pain and extremity pain.

“The clinical trials for ELELYSO™ demonstrate that it is an effective enzyme replacement therapy for Gaucher disease. Patients and physicians now have an additional treatment option, coupled with a patient assistance program focused on auxiliary services,” said Gregory M. Pastores, MD, associate professor of Neurology and Pediatrics at the New York University School of Medicine.

Safety Information for ELELYSO™

As with any intravenous protein product, allergic reactions, some severe, were reported in the taliglucerase alfa clinical trials. A definition of anaphylaxis (as defined by Sampson et al 2006) was retrospectively applied to some of these reports. In patients who have experienced anaphylaxis during infusion with ELELYSO or with other ERT, caution should be exercised upon retreatment; appropriate medical support should be readily available.

Infusion reactions (including allergic reactions), defined as a reaction occurring within 24 hours of the infusion, were the most commonly observed reactions in patients treated with ELELYSO in clinical studies. The most commonly observed symptoms of infusion reactions were headache, chest pain or discomfort, asthenia, fatigue, urticaria, erythema, increased blood pressure, back pain and arthralgia, and flushing. Most of these reactions were mild and did not require treatment intervention.

About Gaucher Disease

Gaucher disease is an inherited lysosomal storage disorder in humans that affects an estimated 10,000 people worldwide and can cause severe and debilitating symptoms, including: enlargement of the liver and spleen, various forms of bone disease, easy bruising, and anemia (a low number of red blood cells). Gaucher disease consists of varying degrees of severity; it has been sub-divided into three subtypes – Types 1, 2, and 3 – according to the presence or absence of neurological involvement. Type 1, the most common, is found at a higher frequency among individuals who are of Ashkenazi Jewish ancestry.

About ELELYSO™ (taliglucerase alfa)

ELELYSO™ (taliglucerase alfa) for injection is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for adults with a confirmed diagnosis of Type 1 Gaucher disease.

Pfizer and Protalix BioTherapeutics Collaboration

On November 30, 2009, Pfizer and Protalix BioTherapeutics, Inc. entered into an agreement to develop and commercialize taliglucerase alfa. Under the terms of the agreement, Protalix has retained exclusive commercialization rights in Israel, while Pfizer received exclusive licensing rights for the commercialization of ELELYSO™ in all other markets. As part of this agreement, and at the conclusion of the FDA approval process, Protalix will transfer the ELELYSO™ NDA and IND to Pfizer.