FDA Expands Methotrexate Indication to Included Children With Acute Lymphoblastic Leukemia

The FDA expanded the indication of methotrexate (Jylamvo; Shorla Oncology) to include the treatment of pediatric individuals with acute lymphoblastic leukemia and polyarticular juvenile idiopathic arthritis. As of this approval, the drug is the only oral liquid methotrexate approved for adult and pediatric indications.¹

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“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,” Sharon Cunningham, CEO of Shorla Oncology, said in a news release. “We are pleased to offer a convenient, patient-friendly alternative for both adult and pediatric patients in the US as we continue to develop innovative solutions for those with limited treatment options.”¹

The drug comes in an orange flavor and has a dedicated dosing syringe. Further, it has a room temperature stability for 3 months after opening, which eliminates the need of refrigeration before dispensing.¹

According to a 2017 study published in Cancer, acute lymphoblastic leukemia is the most common childhood malignancy worldwide, which accounts for 20% to 30% of overall cancer incidence. In the study, investigators included 17,500 children to determine the net survival of pediatric patients in the United States.

They found that at 1 year, there was 95% survival at 1 year, 90% at 3 years, and 86% at 5 years for individuals diagnosed during 2001 and 2003, with the net survival rising to 96%, 91%, and 88%, respectively, for individuals diagnosed during 2004 to 2009. However, the investigators also noted that there are still racial disparities present for pediatric patients after diagnosis, finding that Black children had lower initial 5-year survival rates compared with White children, and the improvement in survival was also lower.²

The drug comes in an orange flavor and has a dedicated dosing syringe. Further, it has a room temperature stability for 3 months after opening, which eliminates the need of refrigeration before dispensing.¹

In April 2024, the FDA accepted the new drug application for methotrexate in the oral liquid form to treat leukemia and other cancers, according to a news release. The oral liquid is a treatment that slows or stops the growth of certain forms of leukemia and other cancers.³

In November 2022, the FDA approved the drug for the treatment of adults with acute lymphoblastic leukemia as part of a combination chemotherapy maintenance regimen, adults with mycosis fungoides as a single agent or as a combination therapy, adults with relapsed or refractory non-Hodgkin lymphomas as part of a metronomic combination, adults with rheumatoid arthritis, and adults with severe psoriasis.⁴

The most common adverse reactions with the drug include ulcerative stomatitis, leukopenia, nausea, and abdominal distress, and the drug is contraindicated for those who are pregnant for the treatment of non-neoplastic disease, and patients with a history of severe hypersensitivity reactions to methotrexate.⁴

REFERENCES

1. Shorla Oncology Announces U.S. Food and Drug Administration (FDA) Expanded Approval of JYLAMVO™ (methotrexate), an Oncology and Autoimmune Drug for Pediatric Indications. News release. Shorla Oncology. October 29, 2024. Accessed October 29, 2024. https://www.businesswire.com/news/home/20241029627376/en/Shorla-Oncology-Announces-U.S.-Food-and-Drug-Administration-FDA-Expanded-Approval-of-JYLAMVO%E2%84%A2-methotrexate-an-Oncology-and-Autoimmune-Drug-for-Pediatric-Indications

2. Tai EW, Ward KC, Bonaventure A, Siegel DA, Coleman MP. Survival among children diagnosed with acute lymphoblastic leukemia in the United States, by race and age, 2001 to 2009: Findings from the CONCORD-2 study. Cancer. 2017;123 Suppl 24(Suppl 24):5178-5189. doi:10.1002/cncr.30899

3. Shorla Oncology Announces FDA Filing Acceptance of New Drug Application to Treat Certain Forms of Leukemia and Other Cancers. News release. Shorla Oncology. April 8, 2024. Accessed October 29, 2024. https://www.businesswire.com/news/home/20240408742912/en/Shorla-Oncology-Announces-FDA-Filing-Acceptance-of-New-Drug-Application-to-Treat-Certain-Forms-of-Leukemia-and-Other-Cancers

4. Jylamvo (methotrexate) – New drug approval. OptumRx. November 29, 2022. Accessed October 29, 2024. https://professionals.optumrx.com/publications/library/drugapproval_jylamvo_2022-1206.html