Pexidartinib

Pexidartinib
Names
Trade namesTuralio
Other namesPLX-3397
IUPAC name
  • 5-[(5-Chloro-1H-pyrrolo[2,3-b]pyridin-3-yl)methyl]-N-{[6-(trifluoromethyl)-3-pyridinyl]methyl}-2-pyridinamine
Clinical data
Drug classKinase inhibitor[1]
Main usesTenosynovial giant cell tumor (TGCT)[1]
Side effectsLiver problems, loss of hair color, tiredness, low neutrophils, increased cholesterol, eye swelling, rash[2]
Routes of
use		By mouth
External links
AHFS/Drugs.comMonograph
US NLMPexidartinib
MedlinePlusa619050
Legal
License data
Legal status
Chemical and physical data
FormulaC20H15ClF3N5
Molar mass417.82 g·mol−1
3D model (JSmol)
SMILES
  • ClC=1C=C2C(=NC1)NC=C2CC=2C=CC(=NC2)NCC=2C=NC(=CC2)C(F)(F)F
InChI
  • InChI=1S/C20H15ClF3N5/c21-15-6-16-14(10-28-19(16)29-11-15)5-12-2-4-18(26-7-12)27-9-13-1-3-17(25-8-13)20(22,23)24/h1-4,6-8,10-11H,5,9H2,(H,26,27)(H,28,29)
  • Key:JGWRKYUXBBNENE-UHFFFAOYSA-N

Pexidartinib, sold under the brand name Turalio, is a medication used to treat tenosynovial giant cell tumor (TGCT).[1] It is used in cases which result in significant problems and cannot be treated by surgery.[1] It is taken by mouth.[1]

Common side effects include liver problems, loss of hair color, tiredness, low neutrophils, increased cholesterol, eye swelling, and rash.[2] The liver problems can result in death.[2] Use in pregnancy may harm the baby.[1] It is a kinase inhibitor and works by blocking colony-stimulating factor-1 receptor (CSF-1R).[1]

Pexidartinib was approved for medical use in the United States in 2019.[1] It was refused approval in Europe in 2020 due to minimal benefits and concerns regarding side effects.[3] It is not approved in the United Kingdom.[4] In the United States it costs about 21,200 USD per month.[5]

Medical uses

Dosage

It is taken at a dose of 400 mg twice per day.[1]

History

The approval of pexidartinib was based on the results of a trial of 120 subjects, 59 of whom received placebo.[6] The primary efficacy endpoint was the overall response rate (ORR) analyzed after 25 weeks of treatment.[6] The clinical trial demonstrated a statistically significant improvement in ORR in subjects who received pexidartinib, with an ORR of 38%, compared to no responses in subjects who received placebo.[6] The complete response rate was 15% and the partial response rate was 23%.[6] A total of 22 out of 23 responders who had been followed for a minimum of six months following the initial response maintained their response for six or more months, and a total of 13 out of 13 responders who had been followed for a minimum of 12 months following the initial response maintained their response for 12 or more months.[6]

The U.S. Food and Drug Administration (FDA) granted the application for pexidartinib breakthrough therapy designation, orphan drug designation, and priority review designation.[6] The FDA granted the approval of Turalio to Daiichi Sankyo.[6]

Pexidartinib is available in the US only through the Turalio Risk Evaluation and Mitigation Strategy (REMS) Program.[6] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[7]

References

  1. 1 2 3 4 5 6 7 8 9 "Pexidartinib Monograph for Professionals". Drugs.com. Archived from the original on 21 January 2021. Retrieved 27 October 2021.
  2. 1 2 3 "DailyMed - TURALIO- pexidartinib capsule". dailymed.nlm.nih.gov. Archived from the original on 24 October 2020. Retrieved 27 October 2021.
  3. ↑ "Turalio". Archived from the original on 27 October 2021. Retrieved 27 October 2021.
  4. ↑ "Pexidartinib". SPS - Specialist Pharmacy Service. 12 January 2016. Archived from the original on 27 October 2021. Retrieved 27 October 2021.
  5. ↑ "Turalio Prices, Coupons & Patient Assistance Programs". Drugs.com. Archived from the original on 16 January 2021. Retrieved 27 October 2021.
  6. 1 2 3 4 5 6 7 8 "FDA approves first therapy for rare joint tumor". FDA (Press release). 2 August 2019. Archived from the original on 14 September 2019. Retrieved 17 August 2019. Public Domain This article incorporates text from this source, which is in the public domain.
  7. ↑ "New Drug Therapy Approvals 2019". U.S. Food and Drug Administration. 31 December 2019. Archived from the original on 16 September 2020. Retrieved 15 September 2020.
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