The first BTK inhibitor to treat immunoplate plateroid reduction (ITP)! FDA grants Sanofi rilzabrutinib fast track eligibility!

November 19, 2020 // -- Sanofi recently announced that the U.S. Food and Drug Administration (FDA) has granted rilzabrutinib fast-track eligibility (FTD) for immunodeficirative plateroid reduction (ITP).
rilzabrutinib is an oral Bruton tyrosine kinase (BTK) inhibitor in the study that has the potential to become the first BTK inhibitor to treat ITP.
October 2018, the FDA granted rilzabrutinib the status of orphan drug (ODD) for the treatment of ITP.
after testing positive in the 1/2 study, Sanofi has launched a Phase 3 clinical study evaluating the treatment of ITP by rilzabrutinib.
is a drug used to prevent, treat and diagnose rare diseases, which are commonly referred to as "orphan diseases" in the form of very low-incidence diseases.
In the U.S., rare diseases are disease types with fewer than 200,000 people, and incentives for the development of rare disease drugs include incentives for clinical development, such as tax credits related to clinical trial costs, FDA user fee waivers, FDA assistance in clinical trial design, and a seven-year market exclusive period for approved adaptations after the drug is marketed.
Fast Track Qualification (FTD) is designed to accelerate drug development and rapid review for serious diseases to address critically unsolved medical needs in key areas.
's fast-track eligibility for experimental drugs means that pharmaceutical companies can interact more frequently with the FDA during the development phase, be eligible for accelerated approval and priority review if the relevant criteria are met after filing a listing application, and also be eligible for rolling review.
rilzabrutinib chemical structure (Photo: pubchem) immunoplate plateroid reduction (ITP) is a rare and serious autoimmune disease characterized by immunomediation plateplate damage and damage to plate plate plate production, resulting in low plate plate count in the blood (plate plate reduction), prone to bleeding, and adverse effects on the quality of life of patients.
for ITP patients who have relapsed or are resistant to corticosteroid treatment, there are still significant unsolved medical needs for rapid and long-lasting remission.
is an oral, reversible, co-priced, BTK inhibitor that is being clinically developed for the treatment of immuno-mediated diseases.
BTK is involved in congenital and adaptive immune responses and is a signaling molecule for immuno-mediated diseases.
study data show that rilzabrutinib blocks inflammatory immune cells, eliminates damaging signals of autoantibodies, and blocks the production of new autoantibodies without consuming B cells.
has the potential to target the pathogenesis of disease and has not been shown to alter plate platetation.
clinical significance of these mechanisms is currently under investigation and their safety and effectiveness have not been reviewed by any regulatory body.
addition to ITP, rilzabrutinib is currently in Phase 3 clinical treatment for herpes, an immunometric-mediated disease characterized by blisters in the mucous membranes and skin.
addition, the treatment of the autoimmune disease IgG4 disease by rilzabrutinib has also initiated Phase 2 clinical studies.
() Original origin: Sanofi's rilzabrutinib Fast Track'd in U.S. for low platelet count