Malaysia Registers 26 Orphan Medicines Since 2020 as Rare Disease List Reaches 529 Conditions
Key Insights
Malaysia has registered 26 orphan medicines for rare diseases as of April 2026, up from zero before 2020, reflecting accelerated regulatory efforts.
The Malaysian Rare Disease List now includes 529 conditions, with approximately 80% linked to genetic causes, including Lysosomal Storage Disease (search) and Spinal Muscular Atrophy (search).
The government increased annual funding for rare disease diagnosis and treatment to RM42 million in 2026, up from RM25 million, with per-patient costs ranging from RM100,000 to RM1 million.
Malaysia has registered 26 orphan medicines for rare diseases as of April this year, a milestone that marks significant progress from having no such registered products before 2020, Deputy Health Minister Datuk Hanifah Hajar Taib told the Dewan Negara today.
The achievement reflects the Ministry of Health's (MOH) sustained efforts to improve access to medicines and treatment for patients living with rare diseases across the country. "The ministry has introduced the Malaysian Orphan Medicines Guideline 2020, which sets out the procedures and criteria for evaluating new medicines. Compliance with these guidelines has helped expedite the approval of safe and effective medicines," Hanifah said during a question-and-answer session, responding to a query from Senator J. Isaiah.
Malaysia's Rare Disease Landscape
The Malaysian Rare Disease List now encompasses 529 identified rare diseases, with approximately 80 per cent attributed to genetic factors. Among the conditions included are Lysosomal Storage Disease (search), a group of disorders that impair the body's ability to break down certain substances due to enzyme deficiencies; Spinal Muscular Atrophy (search) (SMA), which affects nerve cells and muscle function; and several rare skin disorders.
The Deputy Minister noted that treatment costs for a single patient can range from RM100,000 to RM1 million, depending on the condition and medication required. In recognition of this financial burden, the government has increased its annual allocation for the diagnosis and treatment of rare diseases to RM42 million this year, up from RM25 million since 2024.
Policy Framework and Regional Leadership
The National Policy on Rare Diseases in Malaysia was introduced in 2025, providing a formal framework to guide the country's approach to rare disease management. A national action plan is currently being finalised through collaboration among various ministries, Hanifah said.
At the regional level, Malaysia has taken a leadership role by hosting and chairing the Southeast Asia Rare Disease Policy Forum 2025. The country is also working towards developing an ASEAN declaration on rare diseases, aimed at strengthening regional cooperation in addressing the challenges posed by rare diseases across Southeast Asia.