The U.S. Food and Drug Administration (FDA) on Thursday granted orphan drug designation to Sanofi SA ( SNY )‘s rilzabrutinib.

The treatment is an investigational, novel, advanced, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor, for two rare diseases, warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD).

The FDA grants orphan drug designation to investigational therapies that address rare medical diseases or conditions that affect fewer than 200,000 people in the US.

Rilzabrutinib is currently under regulatory review in the US, the EU, and China for its potential use in immune thrombocytopenia (ITP).

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The target action date for the FDA regulatory decision on ITP, which was granted fast-track designation, is Aug. 29.

Rilzabrutinib also received orphan drug designation for ITP in the US, EU, and Japan.

Results from a phase 2b study on wAIHA presented at ASH 2024 demonstrated that treatment with rilzabrutinib showed clinically meaningful outcomes on response rate and disease markers.

In IgG4-RD patients, results from a phase 2a study showed treatment with rilzabrutinib for 52 weeks led to reduction in disease flare, other disease markers, and glucocorticoid sparing.

The safety profile of rilzabrutinib in both studies was consistent with previous studies.

Last week, the FDA approved Sanofi’s Qfitlia (fitusiran), the first antithrombin-lowering (AT) therapy for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B with or without factor VIII or IX inhibitors.

The approval is based on data from the ATLAS phase 3 studies that demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates.

Price Action: Sanofi’s stock is up 3.54% at $55.86 during the premarket session at the last check Thursday.

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