Erdafitinib

tradename	Balversa
medlineplus	a619031
dailymedid	Erdafitinib
routes of administration	By mouth
class	Organonitrogen compounds
atc prefix	L01
atc suffix	EN01
legal ca	Rx-only
legal ca comment	‡R1R‡‡R2R‡
legal us	Rx-only
legal us comment	‡R3R‡
legal eu	Rx-only
legal eu comment	‡R4R‡
cas number	1346242-81-6
pubchem	67462786
pubchemsubstance	347828443
drugbank	DB12147
chemspiderid	35308353
unii	890E37NHMV
kegg	D10927
chembl	3545376
synonyms	JNJ-42756493
iupac name	N'-(3,5-dimethoxyphenyl)-N'-[3-(1-methylpyrazol-4-yl)quinoxalin-6-yl]-N-propan-2-ylethane-1,2-diamine
c	25
h	30
n	6
o	2
smiles	CC(C)NCCN(C1=CC2=NC(=CN=C2C=C1)C3=CN(N=C3)C)C4=CC(=CC(=C4)OC)OC
stdinchi	1S/C25H30N6O2/c1-17(2)26-8-9-31(20-10-21(32-4)13-22(11-20)33-5)19-6-7-23-24(12-19)29-25(15-27-23)18-14-28-30(3)16-18/h6-7,10-17,26H,8-9H2,1-5H3
stdinchikey	OLAHOMJCDNXHFI-UHFFFAOYSA-N

Erdafitinib, sold under the brand name Balversa, is an anti-cancer medication. It is a small molecule inhibitor of fibroblast growth factor receptor (FGFR) used for the treatment of cancer. FGFRs are a subset of tyrosine kinases which are unregulated in some tumors and influence tumor cell differentiation, proliferation, angiogenesis, and cell survival.[5] Astex Pharmaceuticals discovered the drug[6] and licensed it to Janssen Pharmaceuticals for further development.[5]

Medical uses

In the United Statest, erdafitinib is indicated for the treatment of adults with locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations whose disease has progressed on or after at least one line of prior systemic therapy.[3]

History

The efficacy of erdafitinib was studied in a clinical trial (NCT02365597) that included 87 adults with locally advanced or metastatic bladder cancer, with FGFR3 or FGFR2 genetic alterations, that had progressed following treatment with chemotherapy.[7] The overall response rate in these adults was 32.2%, with 2.3% having a complete response and almost 30% having a partial response.[7] The response lasted for a median of approximately five-and-a-half months.[7] The trial was conducted in Asia, Europe, and the United States.[12]

Erdafitinib received an accelerated approval.[7] Further clinical trials are required to confirm erdafitinib's clinical benefit and the sponsor is conducting or plans to conduct these studies.[7] Erdafitinib was also granted breakthrough therapy designation.[7] The FDA granted the approval of Balversa to Janssen Pharmaceutical.[7][13] The FDA also approved the FGFR RGQ RT-PCR Kit, developed by Qiagen Manchester, Ltd., for use as a companion diagnostic with erdafinitib for this therapeutic indication.[7]

In January 2024, the FDA approved erdafitinib for adults with locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations, as determined by an FDA-approved companion diagnostic test, whose disease has progressed on or after at least one line of prior systemic therapy.[10] This approval amends the indication previously granted under accelerated approval for people with metastatic urothelial carcinoma with susceptible FGFR3 or FGFR2 alterations after prior platinum-containing chemotherapy.[10] Efficacy was evaluated in Study BLC3001 Cohort 1, a randomized, open-label trial of 266 participants with metastatic urothelial carcinoma harboring selected FGFR3 alterations who had received 1-2 prior systemic treatments, including a PD-1 or PD-L1 inhibitor.[10] Participants were randomized 1:1 to receive erdafitinib or investigator's choice of chemotherapy (docetaxel or vinflunine).[10] Randomization was stratified by region, performance status, and presence of visceral or bone metastases.[10] FGFR3 alterations were identified from tumor tissue in a central laboratory by the FGFR RGQ RT-PCR kit (Qiagen) in 75% of participants, while the remainder were identified by local next generation sequencing assays.[10]

Erdafitinib

Medical uses

Side effects

History

Society and culture

Legal status

Research

External links

References