Rilzabrutinib

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Rilzabrutinib
Rilzabrutinib.svg
Clinical data
Trade names Wayrilz
Other namesPRN-1008
AHFS/Drugs.com Wayrilz
License data
Routes of
administration 		By mouth
Drug class Antineoplastic
ATC code
  • None
Legal status
Legal status
Identifiers
  • (E)-2-[(3R)-3-[4-amino-3-(2-fluoro-4-phenoxyphenyl)pyrazolo[3,4-d]pyrimidin-1-yl]piperidine-1-carbonyl]-4-methyl-4-[4-(oxetan-3-yl)piperazin-1-yl]pent-2-enenitrile
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
Chemical and physical data
Formula C36H40FN9O3
Molar mass 665.774 g·mol−1
3D model (JSmol)
  • CC(C)(/C=C(\C#N)/C(=O)N1CCC[C@H](C1)N2C3=NC=NC(=C3C(=N2)C4=C(C=C(C=C4)OC5=CC=CC=C5)F)N)N6CCN(CC6)C7COC7
  • InChI=1S/C36H40FN9O3/c1-36(2,45-15-13-43(14-16-45)26-21-48-22-26)18-24(19-38)35(47)44-12-6-7-25(20-44)46-34-31(33(39)40-23-41-34)32(42-46)29-11-10-28(17-30(29)37)49-27-8-4-3-5-9-27/h3-5,8-11,17-18,23,25-26H,6-7,12-16,20-22H2,1-2H3,(H2,39,40,41)/b24-18+/t25-/m1/s1
  • Key:LCFFREMLXLZNHE-GBOLQPHISA-N

Rilzabrutinib, sold under the brand name Wayrilz, is an anti-cancer medication used for the treatment of immune thrombocytopenia. [1] Rilzabrutinib is a tyrosine kinase inhibitor. [1] It is taken by mouth. [1]

Contents

Rilzabrutinib may increase the risk of serious infections (including bacterial, viral, or fungal). [2] The most common side effects include diarrhea, nausea, headache, abdominal pain, and COVID-19. [2]

Rilzabrutinib was approved for medical use in the United States in August 2025. [2]

Medical uses

Rilzabrutinib is indicated for the treatment of adults with persistent or chronic immune thrombocytopenia who have had an insufficient response to a previous treatment. [1] [2]

History

The safety and efficacy of rilzabrutinib was evaluated in a 24-week, double-blind, parallel-group study looking at durable platelet response (i.e., an increased number of platelets from baseline lasting the majority of the last twelve weeks of the treatment period). [2] In this study, 202 participants were randomly assigned to either the rilzabrutinib group (133) or placebo group (69). [2] During the 24-week treatment period, 31 (23%) participants in the rilzabrutinib group and no participants in placebo group achieved a sufficient and durable platelet count response, as determined by the study. [2]

Society and culture

Rilzabrutinib was approved for medical use in the United States in August 2025. [3]

Names

Rilzabrutinib is the international nonproprietary name. [4]

Rilzabrutinib is sold under the brand name Wayrilz. [2]

Research

Rilzabrutinib is an oral, reversible covalent inhibitor of Bruton's tyrosine kinase, that may increase platelet counts in people with immune thrombocytopenia by means of dual mechanisms of action: decreased macrophage (Fcγ receptor)–mediated platelet destruction and reduced production of pathogenic autoantibodies. [5]

References

  1. 1 2 3 4 5 https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/219685s000lbl.pdf
  2. 1 2 3 4 5 6 7 8 "FDA Approves Drug to Treat Adults with Persistent or Chronic Immune Thrombocytopenia". U.S. Food and Drug Administration. 2 September 2025. Retrieved 5 September 2025.PD-icon.svg This article incorporates text from this source, which is in the public domain .
  3. ↑ "Press Release: Sanofi's Wayrilz approved in US as first BTK inhibitor for immune thrombocytopenia" (Press release). Sanofi. 29 August 2025. Retrieved 5 September 2025– via GlobeNewswire.
  4. ↑ World Health Organization (2020). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 83". WHO Drug Information. 34 (1). hdl: 10665/339768 .
  5. ↑ Kuter DJ, Efraim M, Mayer J, Trněný M, McDonald V, Bird R, et al. (April 2022). "Rilzabrutinib, an Oral BTK Inhibitor, in Immune Thrombocytopenia". The New England Journal of Medicine. 386 (15): 1421–1431. doi:10.1056/NEJMoa2110297. PMID   35417637.
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