Türkiye to launch domestic production of SMA drug by next year
ANKARA
The Turkish Health Ministry has unveiled plans to locally produce a vital drug for treating Spinal Muscular Atrophy (SMA), a rare genetic disorder causing motor neuron damage and muscle weakness.
According to authorities, the active substance nusinersen sodium, widely used worldwide under the brand name Spinraza, has already been successfully synthesized in Türkiye.
Clinical trials and regulatory approval procedures are ongoing, with mass production expected to begin in the first half of 2026.
According to the ministry, Türkiye currently has about 1,500 SMA patients, with around 150 new cases diagnosed each year.
Newly diagnosed patients receive six doses of the drug in their first year of treatment, followed by maintenance doses every four months.
The therapy is provided free of charge to patients under the national health insurance system through an agreement with the supplier.
At present, the imported version of the drug costs approximately 1.6 million Turkish Liras (about $38,400) per dose.
SMA most commonly appears in infancy and childhood, though it can also affect adults.
Symptoms include progressive muscle weakness, difficulty in movement and problems with breathing and swallowing.
Current treatment options include gene therapy, medication and physical therapy, with early diagnosis considered critical for slowing the progression.
Officials noted that developing a domestic alternative is not only aimed at SMA but also at expanding local production of active substances used in treatments for other rare genetic diseases, such as Duchenne Muscular Dystrophy (DMD).
In Türkiye, families of children with SMA or DMD have increasingly resorted to setting up donation stands in busy public spaces to raise money for costly overseas treatments.
While fundraising campaigns for treatment remain permitted under the law, the governor’s office in Istanbul banned stands in high-traffic locations last month, citing safety concerns and public order.
