Drugs Xagena
The FDA ( Food and Drug Administration ) has approved Cerdelga ( Eliglustat ) for the long-term treatment of adult patients with the type 1 form of Gaucher disease, a rare genetic disorder.
Gaucher disease occurs in people who 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.
Cerdelga is a hard gelatin capsule containing Eliglustat that is taken orally. In patients with Gaucher disease type 1, the drug slows down the production of the fatty materials by inhibiting the metabolic process that forms them.
Type 1 Gaucher disease is estimated to affect about 6,000 people in the United States.
The safety and effectiveness of Cerdelga were evaluated in two clinical trials with 199 participants with type 1 Gaucher disease.
In one randomized, double-blind, placebo-controlled, multicenter clinical trial the safety and effectiveness of Eliglustat were evaluated in 40 participants with type 1 Gaucher’s disease who had not previously received enzyme replacement therapy.
Subjects received the drug at a starting dose of 42 mg two times a day, with most receiving a dose of 84 mg two times a day after four weeks. Study participants continued the drug for nine months.
Compared to placebo, treatment with Eliglustat resulted in a greater reduction in spleen volume from baseline to the end of the study ( by the 39th week ), the trial’s primary endpoint.
Cerdelga also resulted in greater improvement in liver volume, blood platelet count, and hemoglobin level, compared to placebo.
The other trial sought to determine the safety and effectiveness of Eliglustat compared to enzyme replacement therapy in 159 participants with type 1 Gaucher disease previously treated and stabilized on enzyme replacement therapy. Subjects in the trial received either the enzyme replacement therapy drug Imiglucerase or Eliglustat. The trial has demonstrated that treatment with Eliglustat resulted in similar stabilization of hemoglobin level, platelet count and spleen and liver volume as Imiglucerase.
The most commonly observed side effects in the Eliglustat clinical trials were fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain. ( Xagena )
Source: FDA, 2014
XagenaMedicine_2014
