Erythroid Neoplasm — Drugs

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Drug	Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
Dabrafenib	US FDA	Tumor-agnostic	the treatment of adult and pediatric patients 1 year of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response (DoR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). • the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. • TAFINLAR is not indicated for treatment of patients with wild-type BRAF solid tumors. ( 5.2 )accelerated	approved	Mar 16, 2023	openfda
Dabrafenib	US FDA	Tumor-agnostic	the treatment of adult and pediatric patients 1 year of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response (DoR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). • the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. • TAFINLAR is not indicated for treatment of patients with wild-type BRAF solid tumors. ( 5.2 )accelerated	approved	May 29, 2013	openfda
Dostarlimab	US FDA	Tumor-agnostic	as a single agent for the treatment of adult patients with mismatch repair deficient (dMMR) recurrent or advanced EC, as determined by an FDA-approved test, that has progressed on or following prior treatment with a platinum-containing regimen in any setting and are not candidates for curative surgery or radiation. • Mismatch Repair Deficient Recurrent or Advanced Solid Tumors • as a single agent for the treatment of adult patients with dMMR recurrent or advanced solid tumors, as determined by an FDA-approved test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options. 1 • 1 This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).accelerated	approved	Apr 22, 2021	openfda
Entrectinib	EU EMA	Tumor-agnosticLung Non-Small Cell Carcinoma	Rozlytrek as monotherapy is indicated for the treatment of adult and paediatric patients 12 years of age and older with solid tumours expressing a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and who have not received a prior NTRK inhibitor who have no satisfactory treatment options. Rozlytrek as monotherapy is indicated for the treatment of adult patients with ROS1 positive, advanced non small cell lung cancer (NSCLC) not previously treated with ROS1 inhibitors.conditional	approved	Jul 31, 2020	ema
Entrectinib	US FDA	Tumor-agnostic	Adult and pediatric patients older than 1 month of age with solid tumors that: have a neurotrophic tyrosine receptor kinase ( NTRK) gene fusion, as detected by an FDA-approved test without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.accelerated	approved	Oct 20, 2023	openfda
Entrectinib	US FDA	Tumor-agnostic	Adult and pediatric patients older than 1 month of age with solid tumors that: have a neurotrophic tyrosine receptor kinase ( NTRK) gene fusion, as detected by an FDA-approved test without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.accelerated	approved	Aug 15, 2019	openfda
Larotrectinib	EU EMA	Tumor-agnostic	Vitrakvi as monotherapy is indicated for the treatment of adult and paediatric patients with solid tumours that display a Neurotrophic Tyrosine Receptor Kinase (NTRK) gene fusion, who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and who have no satisfactory treatment options.conditional	approved	Sep 19, 2019	ema
Larotrectinib	US FDA	Tumor-agnostic	VITRAKVI is indicated for the treatment of adult and pediatric patients with solid tumors that: have a neurotrophic receptor tyrosine kinase ( NTRK ) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. Select patients for therapy based on an FDA-approved test [see Dosage and Administration (2.1) ]. VITRAKVI is a kinase inhibitor indicated for the treatment of adult and pediatric patients with solid tumors that: have a neurotrophic receptor tyrosine kinase ( NTRK ) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. Select patients for therapy based on an FDA-approved test. ( 1 , 2.1 )	approved	Nov 26, 2018	openfda
Larotrectinib	US FDA	Tumor-agnostic	VITRAKVI is indicated for the treatment of adult and pediatric patients with solid tumors that: have a neurotrophic receptor tyrosine kinase ( NTRK ) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. Select patients for therapy based on an FDA-approved test [see Dosage and Administration (2.1) ]. VITRAKVI is a kinase inhibitor indicated for the treatment of adult and pediatric patients with solid tumors that: have a neurotrophic receptor tyrosine kinase ( NTRK ) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. Select patients for therapy based on an FDA-approved test. ( 1 , 2.1 )	approved	Nov 26, 2018	openfda
Pembrolizumab	US FDA	Tumor-agnostic	for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy. • Limitations of Use : KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy. Urothelial Cancer in combination with enfortumab vedotin-ejfv, for the treatment of adult patients with locally advanced or metastatic urothelial cancer. • Microsatellite Instability-High or Mismatch Repair Deficient Cancer for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors, as determined by an FDA-authorized test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. • in combination with fluoropyrimidine- and platinum-containing chemotherapy, for the first-line treatment of adults with locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-authorized test. • in combination with lenvatinib, for the first-line treatment of adult patients with advanced RCC. • for the adjuvant treatment of patients with RCC at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions. • Tumor Mutational Burden-High (TMB-H) Cancer for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-authorized test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. 1 • Limitations of Use : The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established. Cutaneous Squamous Cell Carcinoma (cSCC) for the treatment of patients with recurrent or metastatic cSCC or locally advanced cSCC that is not curable by surgery or radiation. • Ovarian Cancer in combination with paclitaxel, with or without bevacizumab, for the treatment of adult patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal carcinoma whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-authorized test, and who have received one or two prior systemic treatment regimens. • 1 This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.accelerated	approved
Repotrectinib	EU EMA	Tumor-agnosticLung Non-Small Cell Carcinoma	Augtyro as monotherapy is indicated for the treatment of adult patients with ROS1-positive advanced non-small cell lung cancer (NSCLC). Augtyro as monotherapy is indicated for the treatment of adult and paediatric patients 12 years of age and older with advanced solid tumours expressing a NTRK gene fusion, and who have received a prior NTRK inhibitor, or  have not received a prior NTRK inhibitor and treatment options not targeting NTRK provide limited clinical benefit, or have been exhausted (see sections 4.4 and 5.1)conditional	approved	Jan 13, 2025	ema
Repotrectinib	US FDA	Tumor-agnostic	adult and pediatric patients 12 years of age and older with solid tumors that: • have a neurotrophic tyrosine receptor kinase ( NTRK ) gene fusion and • are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity. • have progressed following treatment or have no satisfactory alternative therapy. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.accelerated	approved	Nov 15, 2023	openfda
Ropeginterferon Alfa-2b	US FDA	Polycythemia Vera	The treatment of adults with polycythemia vera.	approved	Nov 12, 2021	openfda
Ruxolitinib	US FDA	Polycythemia Vera	polycythemia vera in adults who have had an inadequate response to or are intolerant of hydroxyurea.	approved	May 1, 2026	openfda
Ruxolitinib	US FDA	Polycythemia Vera	polycythemia vera in adults who have had an inadequate response to or are intolerant of hydroxyurea.	approved	Nov 16, 2011	openfda
Selpercatinib	EU EMA	Tumor-agnostic	Retsevmo as monotherapy is indicated for the treatment of adults with: advanced RET fusion positive non small cell lung cancer (NSCLC) not previously treated with a RET inhibitor. For biomarker-based patient selection, see section 4.2.  Retsevmo as monotherapy is indicated for the treatment of adults and paediatric patients 2  years of age and older with: advanced RET fusion positive thyroid cancer who are radioactive iodine-refractory (if radioactive iodine is appropriate) . For biomarker-based patient selection, see section 4.2. advanced RET mutant medullary thyroid cancer (MTC) . For biomarker-based patient selection, see section 4.2. advanced RET fusion positive solid tumours, when treatment options not targeting RET provide limited clinical benefit, or have been exhausted (see sections  4.4 and 5.1). For biomarker-based patient selection, see section 4.2.	approved	Feb 11, 2021	ema
Selpercatinib	US FDA	Tumor-agnostic	Adult and pediatric patients 2 years of age and older with locally advanced or metastatic solid tumors with a RET gene fusion, as detected by an FDA-approved test, that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options	approved	Apr 10, 2024	openfda
Selpercatinib	US FDA	Tumor-agnostic	Adult and pediatric patients 2 years of age and older with locally advanced or metastatic solid tumors with a RET gene fusion, as detected by an FDA-approved test, that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options	approved	May 8, 2020	openfda
Trametinib	US FDA	Tumor-agnostic	the treatment of adult and pediatric patients 1 year of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). • the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.accelerated	approved	Mar 16, 2023	openfda
Trametinib	US FDA	Tumor-agnostic	the treatment of adult and pediatric patients 1 year of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). • the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.accelerated	approved	May 29, 2013	openfda
Trastuzumab Deruxtecan	US FDA	Tumor-agnostic	HER2-Positive Locally Advanced or Metastatic Gastric Cancer as monotherapy for the treatment of adult patients with locally advanced or metastatic HER2-positive (IHC 3+ or IHC 2+/ISH positive) gastric or gastroesophageal junction adenocarcinoma who have received a prior trastuzumab-based regimen. • HER2-Positive (IHC 3+) Unresectable or Metastatic Solid Tumors as monotherapy for the treatment of adult patients with unresectable or metastatic HER2-positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options* • * These indications are approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. ( 14.3 , 14.5 )accelerated	approved	Dec 20, 2019	openfda
Trastuzumab Deruxtecan	US FDA	Tumor-agnostic	HER2-Positive Locally Advanced or Metastatic Gastric Cancer as monotherapy for the treatment of adult patients with locally advanced or metastatic HER2-positive (IHC 3+ or IHC 2+/ISH positive) gastric or gastroesophageal junction adenocarcinoma who have received a prior trastuzumab-based regimen. • HER2-Positive (IHC 3+) Unresectable or Metastatic Solid Tumors as monotherapy for the treatment of adult patients with unresectable or metastatic HER2-positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options* • * These indications are approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. ( 14.3 , 14.5 )accelerated	approved	Dec 20, 2019	openfda

Drug

Jurisdiction · authority

Cancer

Indication

Status

Approval date

Source

Dabrafenib

US FDA

Tumor-agnostic

the treatment of adult and pediatric patients 1 year of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response (DoR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). • the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. • TAFINLAR is not indicated for treatment of patients with wild-type BRAF solid tumors. ( 5.2 )accelerated

approved

Mar 16, 2023

Therapy	Evidence items (count)	Sensitivity / response	Resistance
Fedratinib	1	1	0
Peginterferon Alfa-2b	1	1	0

Approvals by jurisdiction

Therapies appearing in curated evidence