FDA Approves Jylamvo for Pediatric Patients, Expanding Methotrexate Options

Shorla Oncology recently received US Food and Drug Administration (FDA) approval to expand Jylamvo’s (methotrexate) use for children with acute lymphoblastic leukemia and polyarticular juvenile idiopathic arthritis.

Jylamvo, an oral liquid methotrexate, can now treat pediatric patients with acute lymphoblastic leukemia and polyarticular juvenile idiopathic arthritis. This makes Jylamvo the only liquid methotrexate approved for both children and adults, which is important because it fills a gap for kids with oncology and autoimmune conditions who need methotrexate but may struggle to take pills.

Originally, Jylamvo — acquired from UK-based Therakind in 2023 — was approved to treat adults with cancers like acute lymphoblastic leukemia and non-Hodgkin lymphoma, as well as rheumatoid arthritis and severe psoriasis.

Methotrexate, the active ingredient, works by blocking folic acid — a key nutrient that helps cells divide. This action helps control abnormal cell proliferation in cancers and reduces inflammation in autoimmune diseases. Jylamvo has an orange flavor and comes with a dosing syringe, which makes it easier to swallow and measure.

Shorla’s work on Jylamvo fits well with its overall goal of making treatments easier to handle and more patient-friendly. Earlier, the company developed Tepylute, an injectable cancer drug that’s ready to use and doesn’t need complex mixing, making it faster to prepare and more accurate to dose. This highlights Shorla’s ongoing focus on simplifying medication prep and dosing.

Lastly, Jylamvo has some practical advantages. It remains stable at room temperature for up to 90 days after opening, unlike other liquid methotrexate solutions that need refrigeration. This makes Jylamvo more convenient for patients and caregivers by easing the storage process.

By making Jylamvo accessible to children and maintaining its user-friendly design, Shorla aims to meet the needs of both adult and pediatric patients, especially those with limited treatment options.

Although methotrexate has long been a cornerstone in treating cancers and autoimmune diseases, its limitations, including potential resistance and side effects, have inspired research into new alternatives.

Earlier this year, the National Center for Advancing Translational Sciences (NCATS), shared details from a study that explored versortrexate, a methotrexate-based PROTAC (proteolysis targeting chimera) designed to target and break down dihydrofolate reductase (DHFR), methotrexate’s primary target. This selective degradation could reduce toxicity, enhancing safety and usability for patients.

DeepCure, meanwhile, introduced DC-9476 as a potential methotrexate alternative. Unlike methotrexate’s single-target approach, DC-9476 — a selective BRD4 (BD2) inhibitor — addresses multiple inflammatory pathways. In early studies, it outperformed standard treatments, including JAK inhibitors and anti-TNF therapies, particularly for rheumatoid arthritis patients unresponsive to methotrexate.

Beyond Jylamvo, Shorla’s pipeline includes SH-201, a palatable oral liquid for leukemia and certain gastrointestinal cancers, which is currently under FDA review with a decision expected in November 2024. Another candidate, SH-110, an oral liquid for glioma, a type of brain tumor, is in preparation for clinical trials after a positive pre-Investigational New Drug (IND) meeting with the FDA.

These versatile efforts could bring new potential to tackle complex autoimmune and cancer-related conditions.