Fitusiran for hemophilia receives orphan drug designation

Indicated for all types of hemophilia

CPAC meets unmet needs... Continuous post-marketing surveillance is required

Sanofi

A long-acting treatment for all types of hemophilia, administered every 2 months, has been designated as an orphan drug in South Korea.

 

According to the “Regulations on the Designation of Orphan Drugs” released by the Ministry of Food and Drug Safety on the 7th, Sanofi's Fitusiran was designated as the 386th orphan drug in Korea.

 

Fitusiran was released under the brand name Qfitlia in the U.S..

 

In March, the U.S.

 

FDA approved Fitusiran as the first antithrombin inhibitor-based therapy with the convenience of a twice-monthly subcutaneous injection, for the routine prophylaxis to prevent bleeding in patients aged 12 years and older with hemophilia A or B.

 

Fitusiran is an siRNA (small interfering RNA) therapy designed to target antithrombin, a protein involved in blood clotting, for the treatment of patients with hemophilia A and B.

 

It works by lowering antithrombin levels to promote thrombin production, thereby improving hemostasis and preventing bleeding in hemophilia patients.

 

Currently, the hemophilia treatment market is dominated by Hemlibra for hemophilia A, while concizumab by Sanofi and Novo Nordisk is under development as a treatment for both hemophilia A and B.

 

Regarding its designation as an orphan drug, according to the minutes of the Central Pharmaceutical Affairs Council meeting released by the MFDS on the 7th, there was an opinion that Fitusiran is a drug that can meet unmet needs and requires post-marketing surveillance.

 

The committee members believe that Fitusiran is expected to provide patients with a better treatment option by addressing the limitations of current treatments in key areas such as ABR, joint bleeding, patient compliance, and the impact on quality of life.

 

Additionally, while existing medications are currently available, if Fitusiran demonstrates superiority over existing products in terms of ensuring a smooth supply of medications for patients, reducing treatment costs, decreasing administration frequency, alleviating pain, and reducing bleeding, it was deemed to meet the criteria for having an unmet need.

 

In particular, existing treatments have low convenience due to the burden of intravenous injections and frequent administration (2–3 times per week).

 

However, Fitusiran, which is an intravenous injection usable for both hemophilia A and hemophilia B, has the advantage of subcutaneous injection every 4–8 weeks, offering improved convenience.

 

However, while it appears to have improved safety and efficacy compared to alternatives, given its new pharmacological mechanism, careful review is necessary regarding data submission exemptions..

 

Although the structure of N-acetylgalactosamine and its pharmacologic mechanism when used as a target ligand in oligonucleotide and siRNA therapy are already well known, a detailed review of domestic clinical results would be necessary due to safety concerns identified in previous clinical trials.

 

Therefore, regarding safety, the CPAC decided that the risk level could be judged as acceptable for approval by referencing overseas approval criteria, but appropriate patient monitoring would be necessary.

 

The CPAC members stated that “Hemophilia is a disease with a clear pathophysiology and no significant differences in response between races.

 

The submitted Phase III clinical trial results showed that Asian patients accounted for a relatively high proportion of participants, and submitted the opinion that domestic approval is reasonable.

 

Meanwhile, it is reported that the annual cost of Fitusiran in the United States amounts to approximately USD 642,000 (approximately KRW 944 million).