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Gene Therapy for Certain Types of ALL Approved
Novartis news release; 2017 Aug 30
The FDA has approved the first gene therapy for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).
Indications: Kymriah is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.
Dosage and administration:
- For patients 50 kg or less, 0.2 to 5.0 x 106 chimeric antigen receptor (CAR)-positive viable T cells per kg body weight intravenously.
- For patients above 50 kg, 0.1 to 2.5 x 108 total CAR-positive viable T cells (non-weight based) intravenously.
Efficacy and safety: Approval is based on trial results showing that the overall remission rate within 3 months of treatment was 83%.
Side effects/risks: The most common adverse reactions are cytokine release syndrome, hypogammaglobulinemia, infections-pathogen unspecified, pyrexia, decreased appetite, headache, encephalopathy, hypotension, bleeding episodes, tachycardia, nausea, diarrhea, vomiting, viral infectious disorders, hypoxia, fatigue, acute kidney injury, and delirium.
FDA approval brings first gene therapy to the United States [news release]. Silver Spring, MD: FDA. August 30, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm. Accessed September 4, 2017.
Kymriah [package insert]. East Hanover, NJ: Novartis Pharmaceuticals Corporation. 2017. https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/kymriah.pdf. Accessed September 4, 2017.