KUALA LUMPUR, Feb 13 – Malaysia’s Drug Control Authority (DCA) has approved Zolgensma, the first gene therapy authorised in the country since controls over cell and gene therapy products began in 2021.
Zolgensma is an adeno-associated virus vector-based gene therapy that treats spinal muscular atrophy (SMA), a rare disease, in children aged below two years.
SMA is an inherited neuromuscular disease that causes physical weakness and can also cause paralysis.
“SMA is listed in the Malaysian Rare Disease List and the Zolgensma product used to treat the disease has been given Orphan Medicine status that enables the product to get priority review by the National Pharmaceutical Regulatory Agency (NPRA) for quick access to needy patients,” Health director-general Dr Muhammad Radzi Abu Hassan said in a statement last Friday.
His statement was issued after the 393rd DCA meeting on February 8 approved product registration for Zolgensma.
Zolgensma is manufactured by Novartis Gene Therapies Inc., Durham, in the United States, while Novartis Corporation (Malaysia) Sdn Bhd is the product registration holder.
