KUALA LUMPUR, Nov 20 – Life-saving treatments for spinal muscular atrophy (SMA) are not available in Malaysia because the drug makers have yet to register them here, the Ministry of Health (MOH) said.
Deputy Health Minister Dr Lee Boon Chye was answering Batu Gajah MP Sivakumar Varatharaju Naidu, who asked if SMA patients in Malaysia were treated with Spinraza by Biogen or Zolgensma by Novartis.
Both gene therapy drugs have been approved by the Food and Drug Administration (FDA) in the United States to treat the rare disease, SMA.
“All medicines used in Malaysia must be approved and registered by the National Pharmaceutical Regulatory Division,” said Dr Lee in Parliament today.
“To date, the responsible pharmaceutical company has not yet submitted an application to register or market this drug in Malaysia.
“If the application is submitted, the MOH will give due consideration. Therefore, there are currently no SMA patients in Malaysia treated with Spinraza or Zolgensma.”
He further added that government’s data on SMA is not comprehensive.
“The estimated incidence rate in Malaysia for Spinal Muscular Atrophy is 4.9 per 100,000 live births or 1 in 20,000 population. Data from the Institute of Medical Research shows that the number of cases from 2013 to 2017 is 20 to 30 cases per year.
“However, this data is not the overall data of Spinal Muscular Atrophy patients as there is no database or registry for Spinal Muscular Atrophy,” he stated.
In July, Edmund Lim, a father of a nine-year-old boy who has been growing up with SMA, appealed to have these drugs provided in Malaysia.
“I am conflicted – so happy to see so many patients with SMA from so many countries getting new drug therapies that completely change their lives by stopping and stabilising this dreaded disease – yet intensely frustrated with why and how it’s so unbelievably hard here. Spinraza, Zolgensma, and soon Risdiplam – revolutionary drug therapies that aren’t cheap but certainly not as expensive as prices quoted in the USA,” he said in an article on CodeBlue.
“Malaysia does not lack the ability or resources to provide these life-saving and life-changing treatments. But the way things work (or don’t) for kids like Branden, forces them to suffer deteriorating health, then early death. Buried under a system that could and should be more equitable and fairer.
“We pray that action will be taken to provide these drug therapies to Malaysians with SMA, and give them the chance in life that they deserve. We look forward to a future that is equitable and fairer in access to such medicines,” he added.