Drugs / Pexidartinib
Pexidartinib
also known as
PLX3397 · CML-261 · FP-113 · PEXIDARTINIB (PLX3397) · PEXIDARTINIB HYDROCHLORIDE · PLX-3397 HCL · PLX-3397 HYDROCHLORIDE · Turalio · +1 more
Small molecule targets CSF1, CSF1R, FLT3, KIT via inhibition
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed tenosynovial giant cell tumor 2 milestones | |||||
| Filed | EU (EMA) | — | tenosynovial giant cell tumor | 2019-04-03 | “the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for pexidartinib” daiichisankyo.com ↗ |
| Filed | US (FDA) | — | tenosynovial giant cell tumor | 2019-02-05 | “FDA has accepted a New Drug Application (NDA) and granted Priority Review for pexidartinib” daiichisankyo.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansion | US (FDA) | TURALIO | — | 2022-10-06 | fda.gov ↗ |
| Refused (EMA) | EU (EMA) | Turalio | — | 2020-10-28 | europa.eu ↗ |
| Approved | US (FDA) | TURALIO | — | 2019-08-02 | fda.gov ↗ |
Trials 29
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT04223635 | Jan → Oct 2020 | — | Daiichi Sankyo | Completed | No outcome recorded |
| Phase 112 trials | ||||||
| Phase 1 | NCT03901313 | Apr → May 2019 | — | Daiichi Sankyo | Completed | No outcome recorded |
| Phase 1 | NCT03291288 | Feb → Sep 2018 | — | Daiichi Sankyo | Completed | No outcome recorded |
| Phase 1 | NCT03158103 | Apr 2017 → Apr 2021 | gastrointestinal stromal tumor | Memorial Sloan Kettering Cancer Center | Completed | No outcome recorded |
| Phase 1 | NCT03138759 | Feb → Mar 2017 | — | Daiichi Sankyo | Completed | No outcome recorded |
| Phase 1 | NCT02777710 | Jun 2016 → Nov 2019 | colorectal cancer, malignant pancreatic neoplasm, metastatic malignant neoplasm | Centre Leon Berard | Completed | No outcome recorded |
| Phase 1 | NCT02734433 | Jun 2016 → Jun 2017 | cancer | Daiichi Sankyo | Completed | No outcome recorded |
| Phase 1 | NCT02472275 | Jun 2015 → Aug 2019 | prostate adenocarcinoma | Barbara Ann Karmanos Cancer Institute | Completed | No outcome recorded |
| Phase 1 | NCT02390752 | Apr 2015 → Dec 2026 expected | acute promyelocytic leukemia, plexiform neurofibroma, precursor lymphoblastic lymphoma/leukemia, sarcoma | National Cancer Institute (NCI) | Recruiting | No outcome recorded |
| Phase 1 | NCT01826448 | Nov 2013 → Sep 2014 | melanoma | Daiichi Sankyo | Terminated | No outcome recorded |
| Phase 1 | NCT01525602 | May 2012 → Dec 2017 | cancer | Daiichi Sankyo | Completed | No outcome recorded |
| Phase 1 | NCT01090570 | May → Dec 2010 | rheumatoid arthritis | Plexxikon | Withdrawn | No outcome recorded |
| Phase 1 | NCT01004861 | Oct 2009 → Jan 2018 | cancer | Daiichi Sankyo | Completed | No outcome recorded |
| Phase 1/26 trials · 1 missed | ||||||
| Phase 1/2 | NCT02584647 | Nov 2015 → Jul 2023 | malignant peripheral nerve sheath tumor | Gulam Manji | Terminated | No outcome recorded Stop: Enrollment |
| Phase 1/2 | NCT02452424 | Jul 2015 → Aug 2018 | gastrointestinal stromal tumor, head and neck squamous cell carcinoma, melanoma, non-small cell lung carcinoma +1 | Daiichi Sankyo | Terminated | No outcome recorded |
| Phase 1/2 | NCT02401815 | Mar 2015 → May 2020 | gastrointestinal stromal tumor | Cogent Biosciences, Inc. | Completed | No outcome recorded |
| Phase 1/2 | NCT01790503 | Jul 2013 → Nov 2017 | glioblastoma | Daiichi Sankyo | Completed | Missed primary |
| Phase 1/2 | NCT01596751 | Jul 2012 → Jul 2017 | breast cancer | Hope Rugo, MD | Completed | No outcome recorded |
| Phase 1/2 | NCT01349049 | Nov 2011 → Jan 2015 | acute myeloid leukemia | Daiichi Sankyo | Completed | No outcome recorded |
| Phase 26 trials | ||||||
| Phase 2 | NCT04703322 | Mar 2021 → Mar 2023 | tenosynovial giant cell tumor | Daiichi Sankyo | Completed | No outcome recorded |
| Phase 2 | NCT02071940 | Oct 2015 → Mar 2021 | melanoma | The Christie NHS Foundation Trust | Completed | No outcome recorded |
| Phase 2 | NCT01499043 | May 2012 → Mar 2013 | prostate cancer | Daiichi Sankyo | Terminated | No outcome recorded Stop: Business |
| Phase 2 | NCT01349036 | Dec 2011 → Nov 2013 | glioblastoma | Daiichi Sankyo | Terminated | No outcome recorded Stop: Business |
| Phase 2 | NCT01217229 | Mar 2011 → Apr 2012 | classic Hodgkin lymphoma | Daiichi Sankyo | Completed | No outcome recorded |
| Phase 2 | NCT01042379 | Mar 2010 → Dec 2030 expected | HER2 positive breast carcinoma, angiosarcoma, female breast carcinoma, hormone receptor-positive breast cancer +1 | QuantumLeap Healthcare Collaborative | Recruiting | No outcome recorded |
| Phase 32 trials · 1 met primary | ||||||
| Phase 3 | NCT04488822 | Sep 2020 → Oct 2021 overdue | tenosynovial giant cell tumor | Daiichi Sankyo | Active not recruiting | No outcome recorded |
| Phase 3 | NCT02371369 | May 2015 → Mar 2017 | tenosynovial giant cell tumor, diffuse type | Daiichi Sankyo | Completed | Met primary |
| Phase 41 trial | ||||||
| Phase 4 | NCT04526704 | Oct 2020 → Jul 2023 | tenosynovial giant cell tumor | Daiichi Sankyo | Completed | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT02975700 | Jan 2017 → Aug 2018 | melanoma | Daiichi Sankyo | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 8
| Date | Issuer | Release |
|---|---|---|
| 2020-06-29 | Daiichi Sankyo | Regulatory Daiichi Sankyo Provides Update on CHMP Review of Pexidartinib, a CSF1R Inhibitor for the Treatment of Patients with TGCT daiichisankyo.com ↗
Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the Marketing Authorization Application (MAA) for pexidartinib for the treatment of a subset of adult patients with severe tenosynovial giant cell tumor (TGCT). |
| 2019-08-05 | Daiichi Sankyo | Regulatory FDA Approves Daiichi Sankyo’s TURALIO™ (pexidartinib) for the Treatment of Select Patients with TGCT, a Rare and Debilitating Tumor daiichisankyo.com ↗
Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) today announced that the U.S. Food and Drug Administration (FDA) approved TURALIO™ (pexidartinib) as the first and only treatment for adult patients with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery. |
| 2019-05-15 | Daiichi Sankyo | Regulatory FDA Oncologic Drugs Advisory Committee Votes in Favor of Daiichi Sankyo’s Pexidartinib for the Treatment of Select Patients with TGCT, a Rare, Debilitating Tumor daiichisankyo.com ↗
FDA Oncologic Drugs Advisory Committee Votes in Favor of Daiichi Sankyo’s Pexidartinib for the Treatment of Select Patients with TGCT, a Rare, Debilitating Tumor |
| 2019-04-04 | Daiichi Sankyo | Regulatory EMA Validates Daiichi Sankyo’s Marketing Authorization Application for Pexidartinib for Treatment of Patients with TGCT, a Rare, Debilitating, Non-Malignant Tumor daiichisankyo.com ↗
EMA Validates Daiichi Sankyo’s Marketing Authorization Application for Pexidartinib for Treatment of Patients with TGCT, a Rare, Debilitating, Non-Malignant Tumor |
| 2019-02-06 | Daiichi Sankyo | Regulatory FDA Grants Priority Review for Daiichi Sankyo’s New Drug Application for CSF1R Inhibitor Pexidartinib for Treatment of Patients with TGCT, a Rare, Debilitating Tumor daiichisankyo.com ↗
FDA Grants Priority Review for Daiichi Sankyo’s New Drug Application for CSF1R Inhibitor Pexidartinib for Treatment of Patients with TGCT, a Rare, Debilitating Tumor |
| 2018-06-05 | Daiichi Sankyo | Results Daiichi Sankyo Presents Phase 3 ENLIVEN Study of Pexidartinib, Demonstrating Statistically Significant Clinical Improvement Across Multiple Endpoints in Patients with Tenosynovial Giant Cell Tumor at 2018 American Society of Clinical Oncology (ASCO) Annual Meeting daiichisankyo.com ↗
Oral pexidartinib significantly reduced tumor size (39 percent overall tumor response rate), compared to no tumor response in patients treated with placebo. |
| 2017-10-31 | Daiichi Sankyo | Results Daiichi Sankyo Announces ENLIVEN Phase 3 Study of Pexidartinib Met Primary Endpoint in Tenosynovial Giant Cell Tumor daiichisankyo.com ↗
Daiichi Sankyo Announces ENLIVEN Phase 3 Study of Pexidartinib Met Primary Endpoint in Tenosynovial Giant Cell Tumor |
| 2015-11-02 | Daiichi Sankyo | Regulatory FDA Grants Breakthrough Therapy Designation for Daiichi Sankyo and Plexxikon’s Investigational CSF-1R Inhibitor Pexidartinib (PLX3397) in Tenosynovial Giant Cell Tumor daiichisankyo.com ↗
Daiichi Sankyo, Inc. and Plexxikon Inc., a member of the Daiichi Sankyo Group, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to its investigational oral CSF-1R inhibitor pexidartinib (formerly PLX3397) for the treatment of tenosynovial giant cell tumor (TGCT) where surgical removal of the tumor would be associated with potentially worsening functional limitation or severe morbidity. |
All press releases naming this drug 9 releases
Evidence & citations 14 cited values
Every value below carries the sentence it was read from. 32 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | pexidartinib | “evaluated the safety, tolerability, and efficacy of the oral FLT3 inhibitor PLX3397 (pexidartinib), which has activity against the FLT3 TKI-resistant F691L gatekeeper mutation...” PMID 32330242 ↗ Apr 202013“Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors” NCT02390752 ↗ “Single oral 400 mg (2 × 200 mg capsules) dose of pexidartinib in the morning under fasting conditions” NCT03901313 ↗ |
| Known as | CML-261 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3813873 ↗ |
| Known as | FP-113 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3813873 ↗ |
| Known as | PEXIDARTINIB (PLX3397) | ChEMBL registry synonym — accepted as the source's own label CHEMBL3813873 ↗ |
| Known as | PEXIDARTINIB HYDROCHLORIDE | ChEMBL registry synonym — accepted as the source's own label CHEMBL3813873 ↗ |
| Known as | PLX-3397 HCL | ChEMBL registry synonym — accepted as the source's own label CHEMBL3813873 ↗ |
| Known as | PLX-3397 HYDROCHLORIDE | ChEMBL registry synonym — accepted as the source's own label CHEMBL3813873 ↗ |
| Known as | PLX3397 | “Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors” NCT02390752 ↗ |
| Known as | Turalio | “pexidartinib (brand name, Turalio™), a medication taken by mouth” PMID 32755241 ↗ Aug 2020 |
| Known as | TURALIO(R) | “Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors” NCT02390752 ↗ |
| Action | Inhibit | “PLX3397 is a selective inhibitor of Fms, Kit, and oncogenic Flt3 activity.” NCT01004861 ↗ |
| Modality | Small molecule | “TURALIO(R) is an orally available small molecule inhibitor” NCT02390752 ↗ |
| Route | Oral | “orally administered PLX3397” NCT01004861 ↗ |
| Target | FLT3 | “oral FLT3 inhibitor PLX3397” PMID 32330242 ↗ Apr 2020 |