The FDA has granted approval to nelarabine injection (SH-111) in a larger, 375-mg/75-mL vial size for adult and pediatric patients with T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL).1
This larger vial size is the second FDA approval for the same formulation of nelarabine injection, the first of which was for a 250-mg/50-mL vial. According to Shora, the drug’s developer, this vial size was introduced to address ongoing product shortages in the United States (US). However, the new vial size introduces greater dosing adaptability for pediatric patients, and allows adult patients the options of a higher dose.
“We are delighted to offer this new larger vial size of nelarabine injection to better serve adult and pediatric patients with T-cell leukemia and lymphoma,” Sharon Cunningham, chief executive officer and co-founder of Shorla Oncology, stated in a news release. “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.”
Nelarabine Injection Secures Second FDA Approval With Larger Vial Size
- The FDA approved a new 375 mg vial size of nelarabine injection for adult and pediatric patients with T-ALL/T-LBL.
- This approval represents the second FDA-approved vial size for the same nelarabine formulation, following the 250 mg/50 mL vial, and is intended to improve dosing adaptability for pediatric patients while enabling higher-dose options and reduced waste in adult treatment settings.
- Nelarabine is indicated for patients with relapsed or refractory T-ALL/T-LBL following at least 2 prior chemotherapy regimens, and its approval is supported by complete response data from 2 key trials.
What are the current indications for nelarabine injection?
Of note, nelarabine injection was first FDA approved in March 2023, following the approval of nelarabine (Arranon) for the treatment of patients with T-ALL and T-LBL whose disease has not responded to or has relapsed following treatment with at least 2 chemotherapy regimens in 2005.2,3
Nelarabine injection was approved for the following indications:
- Adult population: intravenous use at a dose of 1500 mg/m² delivered over 2 hours on days 1, 3, and 5 every 21 days for adults
- Pediatric population: 650 mg/m² over 1 hour daily for 5 consecutive days every 21 days for children3
Approval was supported solely by complete response (CR) rates, and randomized trials demonstrating survival or other clinical benefit have not been conducted.
What prior data have been reported with nelarabine in T-ALL/T-LBL?
The safety and efficacy of nelarabine was evaluated in adult patients. Among the 39 treated patients, 28 had T-ALL or T-LBL that had relapsed following or was refractory to 2 or more prior lines of induction therapy.
Nelarabine was administered intravenously at the 1500-mg/m2 dose. In the T-ALL/T-LBL population, 21% (95% CI, 8%-41%) of patients achieved a CR, which included CRs without hematologic recovery (CR*). The duration of response ranged from 4 weeks to 195+ weeks.
In the pediatric population, nelarabine was evaluated among patients who were no more than 21 years of age and had relapsed or refractory T-ALL or T-LBL. Eighty-four of these patients had received 2 or more prior lines of induction therapy, and recieved nelarabine at the 650-mg/m2 dose. Among 39 patients, the CR plus CR* rate was 23% (95% CI, 11%-39%), and the duration of CR ranged from 3.3 weeks to 9.3 weeks.
The most common adverse effects (AEs) seen with nelarabine in at least 20% of adult patients include anemia, thrombocytopenia, neutropenia, nausea, diarrhea, vomiting, constipation, fatigue, pyrexia, cough, and dyspnea. In pediatric patients, frequent AEs include anemia, neutropenia, thrombocytopenia, and leukopenia. Frequently observed neurologic AEs in at least 10% of adult patients include somnolence, dizziness, peripheral neurologic disorders, hypoesthesia, headache, and paresthesia. In pediatric patients, the most common AEs are headache and peripheral neurologic disorders.
“Launching nelarabine injection, as our first product in the US, was a major milestone. Expanding that footprint with a 375-mg vial of nelarabine injection reflects our ongoing commitment to solving real-world challenges for patients with T-cell leukemia by prioritizing improvements in health care delivery, pharmacy workflow, and waste reduction,” Orlaith Ryan, chief technical officer and co-founder of Shorla Oncology, concluded in the news release.1 “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.”
References
- 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://www.businesswire.com/news/home/20260127385856/en/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
- Shorla Oncology announces U.S. FDA approval of nelarabine injection for the treatment of T-cell leukemia. News release. Shorla Oncology. March 9, 2023. Accessed January 28, 2026. https://shorlaoncology.com/shorla-oncology-announces-u-s-fda-approval-of-nelarabine-injection-for-the-treatment-of-t-cell-leukemia/
- Prescribing Information. Arranon. Novartis. Updated July 2019. Accessed January 28, 2026. https://www.novartis.com/us-en/sites/novartis_us/files/arranon.pdf
