KUALA LUMPUR - The Health Ministry (MOH) has approved the registration of Xenpozyme (Olipudase alfa 20mg), to treat patients suffering from Acid Sphingomyelinase Deficiency (ASMD), a rare genetic disease.
Health director-general Datuk Dr Muhammad Radzi Abu Hassan said that the decision to approve the product was made at the 389th Drug Control Authority (DCA) meeting, held on Oct 5.
He said that ASMD, which is listed in the Malaysian Rare Diseases List, is given the status of Orphan Medicine, and is eligible to obtain flexibility in registration requirements, in addition to the status which has also qualified the product to be given priority in the evaluation process by the National Pharmaceutical Regulatory Authority (NPRA) to enable immediate access to patients.
"At the DCA meeting this time, it has been agreed to approve the registration of a product given orphan medicine status, namely the product Xenpozyme 20mg powder for concentrate for solution for infusion (which contains the active ingredient olipudase alfa 20mg).
"The approved indication is enzyme replacement therapy for the treatment of non-Central Nervous System (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD), in paediatric and adult patients with type A/B or B,” he said in a statement today.
Xenpozyme products are manufactured by Genzyme Ireland Limited, Ireland, with Sanofi-Aventis (Malaysia) Sdn Bhd as the product registration holder.
Dr Muhammad Radzi said that Xenpozyme (Olipudase alfa 20mg) is an enzyme replacement therapy to replace the patient's enzyme. It is the first specific treatment for ASMD and is able to reduce the accumulation of fat in organ cells and relieve the symptoms of the disease.
"ASMD is a genetic disease in which the sufferer does not have enough of the enzyme needed to break down the sphingomyelin complex fat.
"This condition will result in fat accumulating in organs, including the liver, spleen, lungs and brain, which can result in symptoms such as swelling, pain, loss of appetite and premature death due to respiratory failure,” he said.
He added that NPRA has registered five products for the treatment of rare diseases, including Xenpozyme (Olipudase alfa 20mg) using the registration channel, since 2020.
"MOH, through NPRA, will continue to monitor and evaluate the latest data from time to time regarding the quality, safety and effectiveness of this Xenpozyme (Olipudase alfa 20mg).
"The ministry will also continue to commit to efforts to improve Malaysians’ access to quality, safe and effective medicines,” he said. - BERNAMA