FDA approval summary: Crizotinib for pediatric and young adult patients with relapsed or refractory systemic anaplastic large cell lymphoma.
Margret MerinoYvette KasamonHongshan LiLian MaRuby LeongJiaxi ZhouGregory ReamanWiley ChambersNicholas C RichardsonMarc TheoretRichard PazdurNicole GormleyPublished in: Pediatric blood & cancer (2022)
In January 2021, the U.S. Food and Drug Administration (FDA) approved crizotinib for pediatric patients 1 year and older and young adults with relapsed or refractory systemic anaplastic large cell lymphoma (sALCL). This is the first approval for pediatric sALCL. Approval was based on a single-arm trial of crizotinib monotherapy that included 26 patients, aged 1-20 years, with previously treated sALCL. Efficacy was based on centrally assessed objective response rate (88%) and duration of response. Herein, we highlight unique aspects of the regulatory review, including extension of the indication to young adults, postmarketing safety, and dose optimization strategies.
Keyphrases
- drug administration
- diffuse large b cell lymphoma
- young adults
- acute lymphoblastic leukemia
- advanced non small cell lung cancer
- single cell
- acute myeloid leukemia
- end stage renal disease
- middle aged
- newly diagnosed
- cell therapy
- multiple myeloma
- chronic kidney disease
- ejection fraction
- clinical trial
- prognostic factors
- transcription factor
- phase iii
- open label
- patient reported outcomes
- mesenchymal stem cells
- risk assessment
- climate change
- childhood cancer
- human health
- patient reported
- epidermal growth factor receptor
- double blind