Sanofi Genzyme emphasized the convenience of Cerdelga (Ingredient: eligustat), an oral treatment for Gaucher disease type 1, which has recently gained insurance coverage, in a news conference at the Novotel Ambassador Seoul Gangnam Hotel Friday.
Cerdelga, which halts the accumulation of the fat molecule glucosylceramide and Gaucher cells by partially inhibiting the glucosylceramide synthase enzyme, received health insurance coverage as a treatment for adult patients with Gaucher disease from the Ministry of Health and Welfare in November. The treatment received sales approval in 2015.
Patients insured by the treatment include adult patients with Gaucher disease type 1 who have poor, intermediate, or extensive CYP2D6-metabolizer phenotypes.
Gaucher disease is a rare hereditary disease caused by glucocerebrosidase enzyme deficiency. There are two treatment options -- enzyme replacement therapy (ERT) and substrate reduction therapy (SRT) -- used to treat the disease. EMT injects a therapeutic agent that can supplement the glucocerebrosidase enzyme deficiency, while SRT is a treatment that reduces the substrate production that breaks down glucocerebrosidase.
Cerdelga is an SRT, while Cerezyme, which was introduced earlier by Sanofi Genzyme, is an ERT.
According to the company, Cerdelga was found to be highly potent in both retention, and early treatment for patients initially treated with Cerezyme.
It has also proved effective in clinical trials (ENGAGE) for first-time patients, while also showing similar results in clinical trials (ENCORE) for patients that previously received ERT.
“Cerdelga showed significant clinical and statistical data not only for patients who received ERT but also for patients who did not have previous treatment,” said Pramod K. Mistry, professor of gastroenterology at Yale School of Medicine. “Besides proving equivalence to ERT, 98 percent of the participating patients responded that they preferred oral medicines rather than injections because of its convenience.”
The treatment is an effective SRT that can relieve symptoms by dismantling symptoms in about 90 percent of patients with Gaucher disease type 1, Mistry added.
The company conducted clinical trials for Cerdelga to 395 patients in 29 countries for 14 years. Therefore, the company expects that it has verified the medication’s efficacy and adverse effects that have been pointed out as limitations of SRT through its long-term clinical trials.