WANA (Dec 06) – In a pharmaceutical achievement, an Iranian knowledge-based company has successfully reverse-engineered and locally manufactured the highly complex drug Ivacaftor, used to treat the genetic disorder cystic fibrosis (CF).

 

This breakthrough has reduced the annual treatment cost per patient from roughly USD 360,000 to about USD 3,000, offering renewed hope to more than 3,100 identified CF patients in the country.

 

According to official sources, the company Alborz Pharmed has mastered the full production technology for Ivacaftor, a drug approved by the U.S. FDA in 2012 and considered one of the most effective therapies for CF due to its critical role in improving lung function. The project includes complete domestic synthesis of the active pharmaceutical ingredient, eliminating reliance on imported raw materials.

 

Project managers report that research teams, through redesigned chemical synthesis steps and process optimizations, managed to reproduce the drug’s original molecular formulation—one of the most technically challenging tasks in pharmaceutical reverse-engineering—thereby enabling fully local production.

 

Cystic fibrosis is a genetic disorder caused by defects in the chloride-transport protein within cell membranes, leading to thick secretions in the lungs and digestive system. Ivacaftor improves this protein’s function, significantly enhancing respiratory capacity and reducing hospitalizations and mortality rates.

 

According to the Iranian CF Foundation, 3,100 patients have been identified nationwide, and only about 20 percent reach adulthood—highlighting the urgent need for accessible and effective treatment.

 

The original international version of this drug is extremely expensive, but the domestically produced version reduces the cost to one percent of the global price. Estimates suggest that treating just 200 patients with the Iranian formulation would save around USD 60 million annually in foreign currency and effectively break the existing international monopoly.

 

The manufacturer states that the drug already holds GMP, ISO, and IRC certifications, and its dossier is in the final stages of approval by Iran’s Food and Drug Administration, with a production license expected by year’s end.

 

This project showcases the growing technological capability of Iran’s pharmaceutical sector in advanced drug development and offers CF patients a long-awaited opportunity for sustainable and affordable treatment.