Sanofi Scores Two Major China Approvals For Rare Blood Disorder Drugs

The National Medical Products Administration (NMPA) in China has approved Sanofi SA’s (NASDAQ:SNY) two medicines for rare hematologic diseases: Qfitlia (fitusiran) for hemophilia and Cablivi (caplacizumab) for acquired thrombotic thrombocytopenic purpura.

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With Qfitlia and Cablivi, Sanofi reaches its fourth and fifth approvals in China this year, following Tzield for stage 2 type 1 diabetes and Sarclisa for two indications in relapsed and newly diagnosed multiple myeloma.

Qfitlia is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in pediatric patients 12 years of age and older, and adults with severe hemophilia A.

The approval is based on data from the ATLAS phase 3 studies that demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates (ABR) across hemophilia patients with or without inhibitors.

By lowering AT, a protein that inhibits blood clotting, Qfitlia helps increase thrombin generation to restore hemostasis in people with hemophilia. Hemophilia affects more than 40,000 people in China.

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Cablivi is the first Nanobody targeted therapy designed to treat acquired/immune-mediated thrombotic thrombocytopenic purpura (aTTP/iTTP) in adults and adolescents aged 12 or older weighing at least 40 kg.

Approximately 2700 patients are diagnosed annually in China.

Cablivi targets von Willebrand factor (vWF), a protein in the blood involved in hemostasis, and is designed to inhibit the interaction between vWF and platelets.

Used in conjunction with plasma exchange and immunosuppressive therapy, it helps by inhibiting the formation of microthrombi, which contribute to organ damage during the course of the disease.

These two approvals expand Sanofi’s rare hematology portfolio in China.

In the ATLAS clinical development program, Qfitlia demonstrated low bleed rates across subgroups with as few as six injections a year. Key results include:

• Significant bleed reduction by 71% in ABR for patients without inhibitors treated with Qfitlia prophylaxis compared to clotting factor concentrate on-demand and by 73% in ABR compared to bypassing agent on-demand for patients with inhibitors.
• Median observed ABR during the open-label extension study was 3.8 in patients without inhibitors and 1.9 in patients with inhibitors.
• Nearly half of patients in the open-label extension study experienced one or fewer bleeds (47% 0-1 bleeds and 31% zero bleeds).
• Nearly 80% of participants were on a regimen of six injections per year by the conclusion of the open-label extension study, and 94% achieved target AT levels with 0-1 dose adjustments.

SNY Price Action: Sanofi stock is up 1.31% at $48.89 at publication on Thursday.

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