The FDA has announced its approval of the drug Cerdelga (eliglustat) for the long-term treatment of adult patients with the Type 1 form of Gaucher disease, a rare genetic disorder in which people do not produce enough of an enzyme called glucocerebrosidase. The enzyme deficiency causes fatty materials to collect in the spleen, liver and bone marrow. The major signs of Gaucher disease include liver and spleen enlargement, low red blood cell counts (anemia), low blood platelet counts and bone problems. It is estimated that 6,000 people in the US have Type 1 Gaucher disease.
Cerdelga, which comes as a hard gelatin capsule containing eliglustat works to slow down the production of the fatty materials by inhibiting the metabolic process that forms them.
“This approval offers another important treatment option for patients with this illness,” stated 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.”
The safety and effectiveness of Cerdelga were evaluated in two clinical trials with 199 participants with Type 1 Gaucher disease.Patients who received Cerdelga showed a greater reduction in spleen volume from baseline to the end of the study (by the 39th week), than those given placebos. Cerdelga also resulted in greater improvement in liver volume, blood platelet count, and red blood cell (hemoglobin) level. The most commonly observed side effects during the trials were fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.