R&D PIPELINE THERAPY AREAS

R&D Pipeline Therapy Areas

The agents and uses mentioned below are investigational and have not been approved by the US Food and Drug Administration (FDA) for the uses under investigation. No conclusions regarding safety and efficacy should be drawn for such agents and uses.
Sanofi’s pipeline in rare blood disorders is investigating a new approach to hemophilia management in late-stage clinical trials. The rare blood disorders pipeline has a scope beyond hemophilia, including investigational therapies includes investigational therapies for warm autoimmune hemolytic anemia (wAIHA) and immune thrombocytopenia (ITP).

Educational Resources

General

Pre-approval Information Exchange: Overview of the Sanofi Specialty Care Pipeline
A presentation providing a brief general overview of Sanofi’s specialty care R&D pipeline, including investigational agents and proposed mechanistic targets in hemophilia A & B, wAIHA, and ITP. All information is updated quarterly.

Overview of the Sanofi Specialty Care Rare Blood Disorders Pipeline
A review of investigational products in disease states including immune thrombocytopenia, warm autoimmune hemolytic anemia, cold agglutinin disease, hemophilia, and immune thrombotic thrombocytopenic purpura. This piece covers mechanisms of action as well as additional details on the clinical development program of investigational agents.

Hemophilia

An Overview of Fitusiran for Hemophilia A and B
A presentation providing an overview of hemophilia A and B as well as the impact of inhibitors on treatment, while also discussing the mechanism of action of fitusiran and the phase 3 development program.

Immune Thrombo-cytopenia

An Overview of Rilzabrutinib for Immune Thrombocytopenia
A presentation providing an overview of immune thrombocytopenia disease burden, epidemiology, and guidelines, while also discussing the mechanism of action of rilzabrutinib and the LUNA clinical development program.

Immune Thrombocytopenia Disease Overview and Therapeutic Landscape
A presentation providing an overview of immune thrombocytopenia disease state, pathophysiology, guidelines, and pipeline therapies for ITP.

Rilzabrutinib in ITP Video
A brief review of Bruton’s Tyrosine Kinase’s role in immune mediated disorders, focused on immune thrombocytopenia, and introduction of BTK inhibition as a therapeutic target. Additionally, covering the specific characteristics of investigational agent, rilzabrutinib.