New Orphan Drug for Gaucher Disease Made with Plant Cells

The FDA approved Elelyso (taliglucerase alfa), the first drug ever produced in a genetically engineered plant cell.

The FDA approved Elelyso (taliglucerase alfa) to treat patients with type 1 Gaucher disease, a lysosomal storage disease characterized by bruising, fatigue, anemia, low blood platelets, and enlargement of the liver and spleen. It is the first drug of its kind to be approved by the FDA, and the first drug that is derived from ProCellEx, Protalix’s proprietary manufacturing system.

The drug, developed by researchers at the Israeli biotech firm Protalix Biotherapeutics and licensed to Pfizer Inc, is a form of the human lysosomal enzyme, glucocerebrosidase. Scientists inserted a gene that encodes for the protein into carrot cells to make the replacement enzyme taliglucerase alfa.

The existing therapeutic enzyme replacement therapies for Gaucher’s disease, imiglucerase and velaglucerase, are produced within hamster cells. Elelyso is cheaper to produce because it is not an animal cell culture. It will be available to patients at a cost 25% below that of imiglucerase.

Gaucher disease is a hereditary condition caused by a recessive mutation in a gene located on chromosome 1. Approximately 1 in 100 people in the United States are carriers of the most common type of Gaucher disease (type 1). The carrier rate among Ashkenazi Jews is 8.9%.

“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, PhD, MBA, president and chief executive officer of Protalix Bio Therapeutics, in a press release.

Drug shortages in 2009 of enzyme replacement therapies to treat Gaucher disease spurred the creation of Elelyso, and Pfizer is launching the “Supply Continuity Program” in conjunction with the launch of the drug to ensure that at least 24 months of supply is available to patients with a prescription. They are also introducing Gaucher Personal Support, a program that provides patient assistance programs, patient support, and specialty pharmacy services for people living with Gaucher disease.

“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, in a statement.

The approval “demonstrates FDA’s commitment to developing treatments for rare diseases,” Julie Beitz, director of the FDA’s Office of Drug Evaluation III, said in a statement. The approval also has important implications for further development of drug treatments using plant cell platforms.