FDA Expands Methotrexate (Jylamvo) Indication to Include Pediatric Acute Lymphoblastic Leukemia
Key Insights
The FDA has expanded the indication for methotrexate (Jylamvo) to include pediatric patients with acute lymphoblastic leukemia (search) and polyarticular juvenile idiopathic arthritis (search).
Jylamvo is now the only oral liquid methotrexate approved for both adult and pediatric indications, offering a patient-friendly alternative.
Acute lymphoblastic leukemia (search) is the most common childhood malignancy, accounting for 20% to 30% of overall cancer incidence worldwide.
The U.S. Food and Drug Administration (FDA) has broadened the label for methotrexate (Jylamvo; Shorla Oncology (search)) to encompass the treatment of pediatric patients diagnosed with acute lymphoblastic leukemia (search) (ALL) and polyarticular juvenile idiopathic arthritis (search). This approval marks Jylamvo as the sole oral liquid methotrexate formulation available for both adult and pediatric use.
Sharon Cunningham, CEO of Shorla Oncology (search), stated, "This approval follows Jylamvo’s successful use in adult patients and represents a critical step forward in addressing the unmet needs of pediatric care in oncology and autoimmune diseases." The company aims to provide a more convenient and patient-friendly option for both adult and pediatric patients in the U.S.
Clinical Context and Disease Burden
Acute lymphoblastic leukemia (search) is the most prevalent childhood cancer globally, accounting for 20% to 30% of all cancer diagnoses in children. A 2017 study in Cancer analyzing data from 17,500 children in the United States revealed that 1-year survival rates were 95%, 3-year survival rates were 90%, and 5-year survival rates were 86% for those diagnosed between 2001 and 2003. These rates improved to 96%, 91%, and 88%, respectively, for patients diagnosed between 2004 and 2009. However, the study also highlighted persistent racial disparities, with Black children exhibiting lower initial 5-year survival rates compared to White children.
Jylamvo Formulation and Benefits
Jylamvo is presented as an orange-flavored liquid with a dedicated dosing syringe to facilitate accurate administration. It maintains stability at room temperature for up to three months after opening, eliminating the need for refrigeration prior to dispensing.
The FDA had previously accepted the new drug application for methotrexate in oral liquid form in April 2024, recognizing its potential to slow or halt the progression of leukemia and other cancers. In November 2022, the FDA approved methotrexate for various adult indications, including acute lymphoblastic leukemia (search) (as part of combination chemotherapy), mycosis fungoides (search) (as a single agent or in combination), relapsed or refractory non-Hodgkin lymphomas (search) (in metronomic combinations), rheumatoid arthritis (search), and severe psoriasis (search).
Safety Considerations
Common adverse reactions associated with methotrexate include ulcerative stomatitis, leukopenia, nausea, and abdominal distress. The drug is contraindicated in pregnant women for non-neoplastic diseases and in patients with a history of severe hypersensitivity reactions to methotrexate.