Saudi Gazette report
RIYADH — The Saudi Food and Drug Authority (SFDA) has approved Qfitlia (Fitusiran) for the prevention or reduction of bleeding episodes in adults and adolescents aged 12 and above with hemophilia A or B, including patients with or without factor VIII or IX inhibitors.
In a statement issued on Thursday, the SFDA said the approval reflects its commitment to accelerating patient access to innovative and advanced therapies within the Kingdom, particularly for conditions with unmet medical needs.
Qfitlia had previously received designation under the SFDA’s Breakthrough Medicine Program, a regulatory pathway designed to fast-track the evaluation and availability of promising treatments that demonstrate significant clinical benefit.
Qfitlia is a small interfering RNA (siRNA) therapy that works by reducing plasma antithrombin levels, thereby enhancing thrombin generation and helping restore balance in the blood clotting process.
This mechanism addresses the underlying bleeding risk in hemophilia patients regardless of inhibitor status.
Clinical trials demonstrated that Qfitlia reduced annual bleeding rates by nearly 90% compared with on-demand treatment approaches, underscoring its potential to significantly improve disease control and quality of life for patients living with hemophilia.
The SFDA noted that the most commonly reported side effects in clinical studies included injection-site reactions and elevated liver enzyme levels.
As a precaution, patients are advised to undergo monthly liver function tests for at least six months after initiating treatment or following any dose adjustment.
The authority also highlighted that Qfitlia carries potential risks, including thrombotic events and gallbladder-related conditions, which require careful medical supervision and ongoing monitoring during treatment.
The approval marks another step in expanding access to advanced genetic and RNA-based therapies in Saudi Arabia, in line with national efforts to enhance healthcare outcomes and regulatory efficiency.
