Sanofi Rilzabrutinib is eligible for the FDA Fast Track

Sanofi recently announced that Rilzabrutinib, an oral drug for immunodeficirative plateroid reduction (ITP), has been awarded fast-track status by the U.S. Food and Drug Administration (FDA) and is on track to become the first Bruton tyrosine kinase (BTK) inhibitor to treat ITP.ITP is characterized by immunometric plateplate damage and plate production damage, resulting in reduced downstream plateplates, susceptible bleeding, and affecting the quality of life of patients. The current ITP treatment methods are divided into first- and second-line treatment. First-line therapy usually uses glucoglobulin, intravenous cglobulin, and anti-D-immunoglobulin, although effective in the short term, but with many side effects. Second-line treatment options are not perfect and there are risks. As a result, there are still unsolved clinical needs in ITP to provide rapid, lasting relief to patients who relapse or resist after treatment with corticosteroids.BTK belongs to the non-subject tyrosine kinase Tec family and plays an important regulatory role in the differentiation and development of B cells as a key kinase in the signaling path of B-cell-like (BCR). The research shows that the large amount of expression of BTK can make BCR signaling pathline abnormal activation, make B cell dysfunction, immune tolerance state change, and convert to self-reactive B cells, secrete a large number of autoantibodies induced autoimmune diseases.Rilzabrutinib is an oral, reversible, co-priced BTK inhibitor in clinical studies used to treat immuno-mediated diseases. The data showed that Rilzabrutinib blocks inflammatory immune cells, eliminates damaging signals of autoantibodies, and prevents the production of new autoantibodies without consuming B cells. In addition, Rilzabrutinib has the potential to target the pathogenesis of potential diseases and has not yet been shown to alter plateplate aggregation. The clinical significance of these mechanisms is currently under study and their safety and importance have not yet been reviewed by any regulatory body.In terms of ITP therapeutic drugs, Nplate (romiplostim) is the first FDA-approved drug to treat ITP, a platealine-producing analog peptide that opens up new avenues for long-term treatment of this chronic disease by increasing and maintaining plate concentrations. In addition to being approved in the United States, Nplate has also been approved in the European Union for ITP adult patients, as well as for ITP for at least 6 months and for pediatric patients 1 year and older who have not responded enough to corticosteroids, immunoglobulin or spleen excision. To date, Nplate has been ratified by more than 100 countries worldwide.Another FDA-approved drug for the treatment of ITP is GlaxoSmithKline (GSK), a non-peptide oral platelet-producing receptor agonist (TPO-RA) that interacts selectively with the cross-membrane domain of TPO receptors, increasing platelet production and rapidly raising platelet levels in ITP patients to safe levels. It is used to treat plate plate plate reduction in patients with glucodermal hormone drugs, ineffective immunoglobulin therapy, or chronic primary immunoploid reduction after spleen excision. The drug will be available in China in 2018.With the increasing number of ITP treatment drugs, it is bound to provide ITP patients with more treatment options and better treatment experience for patients.
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