The Saudi Food and Drug Authority (SFDA) has granted breakthrough designation to Sanofi's medication, Fitusiran (Qfitlia), which is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and adolescent patients aged 12 years and older with hemophilia A or B, with or without factor VIII or IX inhibitors. This decision underscores the SFDA's commitment to facilitating access to innovative therapies, aligning with the Health Sector Transformation Program’s objectives under Saudi Vision 2030.

Reducing Antithrombin Levels to Improve Coagulation

Qfitlia is a double-stranded small interfering ribonucleic acid (siRNA) that causes degradation of antithrombin (AT) messenger RNA (mRNA) through RNA interference, leading to a reduction in plasma AT levels. This mechanism enhance thrombin generation, aiming to decrease the frequency of bleeding in individuals with hemophilia A or B, conditions characterized by deficiencies in clotting factors, ultimately helping to manage the disease and mitigate its complications.

Lack of Therapeutic Options for Hemophilia Patients

The submitted clinical data for Qfitlia showed a significant improvement in preventing the bleeding in patient with hemophilia A or B, with or without factor VIII or IX inhibitors. Hence, based on the limited existing therapeutic options for these patients, Qfitlia was granted breakthrough designation by SFDA.

Registration is Subjected to Full Scientific Evaluation

The SFDA emphasizes that this designation allows Qfitlia to be submitted and reviewed under the Breakthrough Track, subject to specific regulatory controls and mechanisms. This designation does not constitute a marketing authorization of the drug in Saudi Arabia. A final decision regarding registration will be announced following a full technical and scientific evaluation of the complete registration dossier.

The SFDA Breakthrough Medicine Program

The Program aims to expedite access to innovative and effective treatments for serious diseases where existing therapies may be inadequate. To qualify for this designation, a drug must have completed clinical trials demonstrating breakthrough efficacy and safety, offer a significant therapeutic advantage over current options, target serious or life-threatening conditions, and exhibit a positive benefit-risk balance. Notably, the product should not be registered with any other regulatory authority at the time of application.

For further information about the Breakthrough Medicines Program, please refer to the guide available on the SFDA website:

‏https://sfda.gov.sa/en/regulations/89310   or contact the SFDA via email at  Designation.Drug@sfda.gov.sa