FDA Approves Drug Cerdelga to Treat Adults with Type 1 Form of Gaucher Disease

The FDA panel has approved a new drug Cerdelga to treat those with type 1 form of Gaucher disease.

In a latest announcement, the U.S. Food and Drug Administration approved cerdelga (eliglustat) for the long-term treatment of those with a rare genetic disorder - type 1 form of Gaucher disease. It is a condition that occurs in those who do not produce sufficient enzyme glucocerebrosidase.

Due to the deficiency of the enzyme glucocerebrosidase, the fatty materials accumulate in the spleen, liver and bone marrow. The potential signs of Gaucher include enlargement of the spleen and liver, anemia - low count of red blood cells, low blood platelet counts and bone problems.

The newly-approved drug Cerdelga, is a hard gelatin capsule that has eliglustat and is meant to be taken orally. In those with Gaucher disease type 1, the drug lowers the production of fatty materials by inhibiting the metabolic process that forms them. It is estimated that nearly 6,000 people in the U.S. are affected with type 1 Gaucher.

"Today's approval offers another important treatment option for patients with Type 1 Gaucher disease," said Amy G. Egan, M.D., M.P.H., deputy director of the Office of Drug Evaluation III in FDA's Center for Drug Evaluation and Research. "In addition, Cerdelga received orphan drug designation from the FDA, reflecting the agency's focus and commitment to the development of treatments for rare diseases."

They tested the safety and effectiveness of the new drug in two clinical trials with 199 participants with type 1 Gaucher disease.

In the randomized, double-blind, placebo-controlled clinical trial, the researchers tested the safety and effectiveness of the drug Cerdelga in 40 participants with type 1 Gaucher's disease who did not receive enzyme replacement therapy. As a starting dose, the participants received a dose of 42 mg twice a day and most of them received a dose of 84 mg two times a day after four weeks. The participants took the drug for nine months.

When compared to the placebo, those receiving Cerdelga noticed a drop in spleen volume from baseline to the end of the study. They also had an improvement in liver volume, blood platelet count and red blood cell level.

The safety and effectiveness of the drug was compared to the enzyme replacement therapy in 159 participants with type 1 Gaucher.

The side effects noticed were fatigue, nausea, diarrhea, back pain, pain in extremities and upper abdominal pain. Cerdelga is manufactured by Cambridge, Massachusetts-based Genzyme.