Sanofi's Rilzabrutinib Granted FDA Fast Track Designation for Immune Thrombocytopenia (ITP) as Potential First-in-Class BTK Inhibitor

November 19, 2020 News /BioValleyBIOON/ -- Sanofi recently announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to rilzabrutinib for the treatment of immune thrombocytopenia (ITP). Rilzabrutinib is an investigational oral Bruton's tyrosine kinase (BTK) inhibitor with the potential to become the first BTK inhibitor for the treatment of ITP. In October 2018,FDARilzabrutinib has been granted orphan drug designation (ODD) for the treatment of ITP. Following positive results from a Phase 1/2 study, Sanofi has initiated a Phase 3 clinical trial to evaluate rilzabrutinib for the treatment of ITP.

Orphan Drug refers to drugs used for the prevention, treatment,DiagnosisDrugs for rare diseases, which are a group of conditions with extremely low incidence rates, also known as "orphan diseases." In the United States, rare diseases refer to those affecting fewer than 200,000 people. Incentives for the research and development of orphan drugs include various clinical development incentives, such as tax credits related to clinical trial costs,FDAUser Fee Waiver,Clinical TrialsUnder DesignFDAassistance, as well as a 7-year market exclusivity period for the approved indications after the drug is marketed.

Fast Track Designation (FTD) aims to accelerate drug development and expedited review for serious diseases, addressing critical areas of unmet medical needs. When an investigational drug receives Fast Track designation, it means that pharmaceutical companies can engage withFDAEngage in more frequent interactions; if the relevant criteria are met after submission of the marketing application, it will be eligible for accelerated approval and priority review, as well as rolling review.

Rilzabrutinib Chemical Structure (Image Source: PubChem)

Immune Thrombocytopenia (ITP) is a rare, severeAutoimmunityImmune thrombocytopenia (ITP) is characterized by immune-mediated platelet destruction and impaired platelet production, leading to low platelet counts in the blood (thrombocytopenia), increased susceptibility to bleeding, and adverse effects on patients' quality of life. For ITP patients who experience relapse or resistance to corticosteroid therapy, there remains a significant unmet medical need in achieving rapid and durable remission.

Rilzabrutinib is an oral, reversible, covalent Bruton’s tyrosine kinase (BTK) inhibitor currently in clinical development for the treatment of immune-mediated diseases. BTK plays a role in innate and adaptive immune responses and serves as a signaling molecule in immune-mediated diseases. Research data indicate that rilzabrutinib can block inflammatory immune cells, eliminate destructive signaling from autoantibodies, and prevent the production of new autoantibodies without depleting B cells. Rilzabrutinib has the potential to target the pathogenesis of the disease and has not been shown to alter platelet aggregation. The clinical significance of these mechanisms is currently under investigation, and its safety and efficacy have not yet been reviewed by any regulatory authority.

In addition to ITP, rilzabrutinib is currently in Phase 3 clinical trials for the treatment of pemphigus, an immune-mediated disease characterized by blisters on the mucous membranes and skin. Furthermore, rilzabrutinib treatmentAutoimmunityIgG4-related disease has also initiated Phase II clinical trials. (Bioon.com)

Original Source: Sanofi's Rilzabrutinib Granted Fast Track Designation in the U.S. for Low Platelet Count