Govt approves enzyme therapy for rare genetic disorder

 

The director-general of health, Dr Radzi Abu Hassan, said the product had been given priority to enable patients to gain immediate access. (Bernama pic)

PETALING JAYA: The health ministry has approved the registration of an enzyme replacement therapy product to treat patients suffering from a rare genetic disorder known as acid sphingomyelinase deficiency.

The director-general of health, Dr Radzi Abu Hassan, said approval was granted at a meeting of the Drug Control Authority on Thursday.

The product Xenpozyme (Olipudase alfa 20mg) had been given priority for evaluation by the National Pharmaceutical Regulatory Authority to enable patients to gain immediate access.

Radzi said Xenposymet is the first specific treatment for ASMD which can reduce the accumulation of fat in organ cells and relieve the symptoms of the disease.

He said patients with ASMD lack an enzyme needed to break down the sphingomyelin complex fat, resulting in accumulation of the fat in organs such as the liver, spleen, lungs and brain, and causing premature death from respiratory failure.

He said NPRA has registered five products for the treatment of rare diseases since 2020. - FMT