FDA OKs Larger Nelarabine Vial in T-Cell Leukemia and Lymphoma Populations

The FDA has approved nelarabine injection (SH-111) at a larger vial size of 375 mg/75mL for adult and pediatric patients with T-cell acute lymphoblastic leukemia (T-ALL) or T-cell lymphoblastic lymphoma (T-LBL), according to a press release from the developer, Shorla Oncology.¹

Originally, the FDA approved nelarabine injection for patients with T-ALL in T-LBL at a vial size of 250 mg/50mL.² This regulatory decision was intended to address ongoing product shortages by providing an alternative formulation of reference nelarabine (Arranon).

The larger vial may provide higher dosing flexibility for pediatric populations while allowing for higher-dosing options for adult patients. This approval may permit more precise dosing based on individual needs and preferences.

“We are delighted to offer this new larger vial size of nelarabine injection to better serve adult and pediatric patients with [T-ALL and T-LBL],” Sharon Cunningham, chief executive officer and co-founder of Shorla Oncology, stated in the press release.¹ “Both adult and pediatric patients have differing dose needs, which can make treatment preparation complex. With this FDA approval, we hope to support health care providers in delivering care more efficiently, reducing waste, and improving precision in managing these types of aggressive blood cancers.”

Developers designed nelarabine injection as a nucleoside metabolic inhibitor for use among patients 1 year or older with T-ALL or T-LBL who have not responded to or have relapsed following prior treatment with 2 or more lines of chemotherapy. The product’s label contains warnings and precautions for neurologic adverse reactions, hematologic reactions, embryo-fetal toxicity, and effects on the ability to drive and use machinery.

The most common adverse effects (AEs) reported with nelarabine rejection include anemia, thrombocytopenia, neutropenia, nausea, diarrhea, vomiting, constipation, fatigue, pyrexia, cough, and dyspnea among adult patients as well as neutropenia, anemia, thrombocytopenia, and leukopenia among pediatric populations. Common neurological toxicities have included somnolence, peripheral neurological disorders, dizziness, hypoesthesia, headache, and paresthesia for adults and headaches and peripheral neurologic disorders for pediatric patients.

According to its prescribing information, nelarabine underwent assessment as part of a phase 3 trial (NCT00408005), in which investigators evaluated the injection plus multi-agent chemotherapy among 804 patients with newly diagnosed T-ALL (85%) or T-LBL (15%).³ In this trial, patients were assigned to chemotherapy with (n = 411) or without (n = 393) nelarabine injection.

Data from this trial revealed a single fatal neurological AE in a patient who received nelarabine injection. Additionally, the rates of grade 3/4 abnormal transaminases, motor and sensory neuropathy, nausea and vomiting, and dehydration were higher in the nelarabine injection arm. Any-grade seizures occurred in 3% (n = 14/411) of patients in the nelarabine injection arm, and 2% (n = 7/411) experienced rhabdomyolysis.

In a clinical trial including 28 evaluable adult patients with T-ALL or T-LBL, data showed a complete response (CR) plus CR without hematologic recovery rate of 21% (95% CI, 8%-41%) with nelarabine injection. Additionally, the duration of CR plus CR without hematologic recovery ranged from 4 to more than 195 weeks, and the median overall survival (OS) was 20.6 weeks (95% CI, 10.4-36.4).

In another trial that included 39 evaluable patients with T-ALL or T-LBL who were 21 years and younger, the CR plus CR without hematologic recovery rate was 23% (95% CI, 11%-39%). The duration of CR plus CR without hematologic recovery ranged from 3.3 to 9.3 weeks, and patients experienced a median OS of 13.1 weeks (95% CI, 8.7-17.4).

“This additional vial size strengthens our ability to support both clinicians and patients living with T-ALL and T-LBL, where flexibility and accuracy in dosing is important,” Orlaith Ryan, chief technology officer and co-founder of Shorla Oncology, concluded.¹

References

1. Shorla Oncology® announces U.S. FDA approval of larger vial size for nelarabine intravenous administration for the treatment of T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma. News release. Shorla Oncology. January 28, 2026. Accessed January 28, 2026. https://tinyurl.com/27mdnmvs
2. Shorla Oncology announces U.S. FDA approval of nelarabine injection for the treatment of T-cell leukemia. News release. Shorla Oncology. March 7, 2023. Accessed January 28, 2026. https://tinyurl.com/2ujt7d56
3. NELARABINE injection, for intravenous use. Prescribing information. FDA; 2025. Accessed January 28, 2026. https://tinyurl.com/d35f6jdn