Türkiye to begin domestic production of key SMA drug

Türkiye has announced that it will begin producing the active ingredient of a major spinal muscular atrophy (SMA) treatment and the production of the drug domestically, marking a significant step toward reducing dependence on costly imported therapies for the rare genetic disease.

Under a cooperation protocol signed at the 11th Turkish Medical World Congress in Ankara, the country will synthesize and manufacture Nusinersen, one of the primary drugs used in SMA treatment, through a partnership led by a Turkish pharmaceutical company and the Health Institutes of Türkiye (TÜSEB).

Officials said clinical studies for the locally developed drug and its raw material have already begun.

TÜSEB President Ümit Kervan said the start of clinical trials means SMA patients will be able to access the new treatment once it proves effective, adding that the molecule was developed entirely by a domestic company.

The collaboration will also support research to produce active ingredients for other rare and genetic diseases.

SMA is a severe neuromuscular disorder that causes irreversible loss of motor neurons and progressive muscle weakness.

While Türkiye lacks precise national prevalence data, experts estimate 130-180 new SMA cases occur each year, with around 3,000 patients currently monitored. Since 2021, a nationwide premarital carrier-screening program has aimed to identify at-risk couples and provide genetic counseling.

As treatment costs remain high, families of children with SMA have increasingly resorted to public fundraising campaigns.

Donation stands have become a common sight in busy urban areas as parents seek to finance expensive overseas gene therapies. Erdoğan receives WHO award At the same congress in Ankara, President Recep Tayyip Erdoğan received the WHO Europe Award for Türkiye’s humanitarian support for Palestinians and its role in coordinating medical evacuations from Gaza.

In his remarks, Erdoğan criticized the international community for remaining silent amid the destruction of hospitals in Gaza and the deaths of patients, health workers and infants. He added that the world would become “a paradise” the day humanity shares the collective joy of saving a life and celebrates every newborn’s safe arrival as its own happiness.

Erdoğan highlighted extensive reforms to Türkiye’s health system over the past two decades, noting that it now provides affordable care to 86 million people through more than 1.47 million health personnel. He said Türkiye aims to further advance in scientific research, pharmaceuticals and medical device production.

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