WANA (Nov 17) – Iranian scientists have successfully produced an oral treatment for patients with spinal muscular atrophy (SMA), making the drug available at only 2% of the cost of its foreign counterpart, which is priced at £7,900 per bottle—one of the most expensive medicines worldwide. Researchers say domestic production not only meets the country’s needs but also saves approximately $55 million in foreign currency annually.

 

Zahra Ghasemi, a research and development expert at an Iranian pharmaceutical company, announced in an interview that the company had developed the first SMA treatment in Iran. “Since 2024, we have successfully developed a specialized medicine for SMA patients, transforming our company into a knowledge-based enterprise,” she said. SMA is a genetic disorder that causes progressive degeneration of nerves and muscles.

 

Ghasemi added that the company is only the second in the world, after a Swiss pharmaceutical firm, to produce this drug. “This achievement is not only scientifically significant but also economically impactful. The price difference with the foreign version is substantial. The Swiss drug costs around £7,900 per bottle, and patients typically require 2.5 to 3 bottles per month, depending on age and dosage. Our domestically produced version costs roughly 20 million Iranian tomans, with a large portion covered by insurance.”

 

Explaining the disease mechanism, Ghasemi said, “In SMA patients, muscles and nerves gradually deteriorate, potentially leading to severe motor weakness or paralysis. It is a genetic condition that can manifest from the fetal stage to adulthood.”

 

She highlighted the drug’s therapeutic effects, stating, “These patients lack a critical protein in their bodies, which leads to muscle degeneration. Our medication helps restore the production of this protein, significantly improving nerve and muscle function. While the Swiss manufacturer designed the drug primarily to halt disease progression, Iranian patients using the domestic product have also shown noticeable motor improvements.”

 

Ghasemi shared a case example: “A child who was completely paralyzed before treatment could sit up to a significant extent after using the drug. This demonstrates that, besides slowing disease progression, the medication can also partially restore muscle function.”

 

She emphasized early treatment: “If SMA is diagnosed at birth and the newborn receives the drug promptly, no symptoms may appear. The earlier the treatment starts, the more effective it is, and the better the patient’s quality of life.”

 

Regarding diagnosis, she noted that SMA is usually easy to identify once confirmed by a physician. “Symptoms in infants and children, such as inability to move, lack of response to sounds, or absent reflexes, make diagnosis straightforward.”

 

Ghasemi explained that the drug entered production and commercialization in 2024. Currently, around 1,500 SMA patients have been identified in Iran, and their monthly medicine requirements are fully met by domestic production. Each patient consumes 2.5 to 3 bottles per month, depending on age and physical condition.

 

The oral formulation requires patients to take specific doses as prescribed. “The main advantage of the domestic product is full patient access,” Ghasemi said. “Given that a foreign bottle costs over 1 billion tomans (At the current exchange rate in Iran’s open market, this amount is approximately $9,091), few patients could afford it. The local version costs only about 2% of that and is largely covered by insurance.”

 

She added that the drug has undergone efficacy evaluations under Iran’s Food and Drug Administration standards, and analyses show its performance is equivalent to the international version, with all necessary approvals obtained.

 

Regarding export potential, Ghasemi said, “Turkey purchased 150 million euros worth of this medicine from our company last year. Entering international markets could bring significant foreign revenue. Domestic production is estimated to save the country about $55 million annually.”

 

On the drug’s mechanism, she explained, “SMA patients lack a vital protein essential for normal nerve and muscle function. Our medication stimulates the body to produce this protein, preventing muscle degeneration and improving motor abilities.”