Sanofi Genzyme Korea, a subsidiary of Sanofi, said Thursday that the Ministry of Health and Welfare has decided to expand insurance benefit for Cerezyme (Ingredient: imiglucerase), its oral treatment for Gaucher disease.
|Sanofi Genzyme's Gaucher disease treatment Cerezyme|
Under the new reimbursement plan, patients with type 3 Gaucher disease will get insurance benefits in addition to the previous benefits for type 1 Gaucher disease.
Gaucher disease is a rare genetic disorder, classified into three clinical subtypes, wherein an enzyme called glucocerebrosidase does not work correctly and cannot break down a lipid called glucocerebroside. Glucocerebroside then accumulates in the body and affects various parts, including the bone marrow, liver, bone, lung, and spleen, among others. Type 1 is the most common and less severe, while type 2 and 3 are more severe because they affect the brain and spinal cord.
In Asian countries, including Korea, more than 50 percent of patients suffer from type 2 and 3 Gaucher disease. In particular, the prevalence of type 3 Gaucher disease is 38.9 percent in Korea, which is significantly higher than the global prevalence of 5 percent. However, there has been no nationally approved type 3 Gaucher disease treatment in Korea until now.
Cerezyme is an enzyme replacement therapy (ERT) that enhances enzyme activity by intravenous administration of Glucocerebrosidase enzyme deficient in Gaucher disease patient.
The ministry approved the additional indication after a postmarketing clinical trial in Japan.
The company administered Cerezyme to patients with nerve-type Gaucher disease in Japan, and as a result, after the end of ERT, significant symptoms of hematology, organs, and skeletal system reached treatment goals. The company also confirmed the safety of the drug.
The International Collaborative Gaucher Group (ICGG) Gaucher Registry further demonstrated that significant non-neurological symptoms, such as hemoglobin levels, platelet counts, liver and spleen volumes, significantly improved in type 3 Gaucher disease patients treated with Cerezyme.
“The company is very pleased to be able to offer a new treatment option to patients with type 3 Gaucher disease who have had a high prevalence rate but have no treatment,” Sanofi Genzyme Korea General Manager Christine Park said. “Sanofi Genzyme will continue to make efforts to improve the quality of life of patients and their families through various treatments for patients with Gaucher disease.”