BPOM grants marketing authorisation for first gene therapy for spinal muscular atrophy patients
Jakarta (ANTARA) - The Food and Drug Authority (BPOM) has granted marketing authorisation for the first gene therapy to treat Spinal Muscular Atrophy (SMA) in Indonesia, as part of efforts to support access to safe, efficacious and quality innovation in response to this rare genetic disease.
Head of BPOM Prof. dr. Taruna Ikrar said SMA, or spinal muscular atrophy, is a rare genetic disease that causes progressive muscle weakness due to damage to motor nerve cells.
“This rare disease is estimated to occur in about 1 in 10,000 live births worldwide and is one of the leading causes of infant death due to genetic factors,” he said in Jakarta on Thursday.
In Indonesia, Taruna said, although national epidemiological data remain limited, various studies show that SMA patients still face challenges including delayed diagnosis, limited access to genetic testing, and the need for more optimal services and therapy.
Based on PhRMA data, only 9 percent of new medicines worldwide are currently available in Indonesia, and only 2 percent are included in the national health insurance programme.
Head of the Indonesian Society of Human Genetics (InaSHG) Prof. dr. Gunadi, who is also Coordinator of the Rare Disease Hub at Dr Sardjito General Hospital under the Ministry of Health and Professor at Gadjah Mada University, emphasised the importance of understanding the genetic aspects of SMA.
“Parents can carry the genetic changes that cause SMA without showing symptoms. Therefore, understanding the genetic aspects of SMA is important, not only to raise awareness of the disease, but also to support proper diagnosis and genetic counselling,” he said.
Chair of the Integrated Rare Disease Service Centre at Dr Cipto Mangunkusumo National General Hospital Damayanti Rusli Sjarif highlighted that the impact of SMA is felt not only medically, but also psychosocially and economically by patients and families.
“The entry of therapeutic innovation into Indonesia must be balanced with strengthening an integrated medical referral system from primary care facilities to rare disease referral centres, as well as a national policy on the management and financing of rare diseases, including SMA,” said Damayanti.
Novartis Indonesia held a collaborative forum bringing together regulators, healthcare workers, academics, and patient communities to improve public understanding of SMA through a discussion titled “Driving New Hope for SMA Patients in Indonesia: From Disease Awareness to Advances in Therapeutic Innovation”.
On this occasion, a symbolic handover of the marketing authorisation for the first gene therapy for SMA treatment in Indonesia was also carried out by the Food and Drug Authority (BPOM), marking an important milestone in the handling of rare diseases in the country.
“We believe that no patient should be left behind simply because the disease they face is classified as rare. Collaboration is needed from all parties, including government, healthcare workers, communities, and industry, to ensure optimal availability and access to treatment for SMA patients,” said President Director of Novartis Indonesia Libby Hsu.