FDA approves Cerdelga for Type 1 Gaucher disease treatment

The aproval follows two clinical trials that evaluated the safety and effectiveness of Cerdelga in 199 participants with the disease that is estimated to affect nearly 6,000 people in US.

Gaucher disease is caused by deficiency of glucocerebrosidase, which leads to accumulation of fatty materials in the spleen, liver and bone marrow, causing liver and spleen enlargement, anemia, low blood platelet counts and bone problems.

Taken orally, Cerdelga is a hard gelatin capsule containing eliglustat and is claimed to have slowed down the production of the fatty materials in patients with Gaucher disease Type , by inhibiting the metabolic process that forms them.

FDA Drug Evaluation and Research Center Drug Evaluation III Office deputy director Amy Egan said: "Today’s approval offers another important treatment option for patients with Type 1 Gaucher disease.

"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."

The first one double-blind, placebo-controlled clinical trial involved 40 participants with Type 1 Gaucher’s disease who had not previously received enzyme replacement therapy and were given drug at a starting dose of 42 mg twice a day, with most receiving a dose of 84mg two times a day after four weeks for nine months.

Cerdelga treatment resulted in a greater reduction in spleen volume from baseline to the end of the study compared to placebo, whilst improving liver volume, blood platelet count, and hemoglobin level.

Undertaken with 159 participants previously treated and stabilized on enzyme replacement therapy, the second trial demonstrated that treatment with Cerdelga resulted in similar stabilization of hemoglobin level, platelet count and spleen and liver volume as imiglucerase.