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FDA D.I.S.C.O. Burst Edition: FDA approval of Ojemda (tovorafenib) for relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement, or BRAF V600 mutation

Podcast

Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we’ll provide a quick update on a recent FDA cancer drug approval.

On April 23, 2024, the FDA granted accelerated approval to tovorafenib (brand name Ojemda) for patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.

This represents the first FDA approval of a systemic therapy for the treatment of patients with pediatric LGG with BRAF rearrangements, including fusions.

Efficacy was evaluated in 76 patients enrolled in FIREFLY-1, a multicenter, open-label, single-arm trial in patients with relapsed or refractory pediatric low-grade glioma harboring an activating BRAF alteration detected by a local laboratory who had received at least one line of prior systemic therapy. Patients were required to have documented evidence of radiographic progression and at least one measurable lesion. Patients with tumors harboring additional activating molecular alterations or with a known or suspected diagnosis of neurofibromatosis type 1 were excluded. Patients received tovorafenib based on body surface area once weekly until they experienced disease progression or unacceptable toxicity.

The major efficacy outcome measure was overall response rate, as defined as the proportion of patients with complete response, partial response, or minor response by blinded independent central review based on Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma criteria. Additional efficacy outcome measures included duration of response. The overall response rate was 51% and median duration of response was 13.8 months.

The most common adverse reactions reported in more than 30% of patients were rash, hair color changes, fatigue, viral infection, vomiting, headache, hemorrhage, pyrexia, dry skin, constipation, nausea, dermatitis acneiform, and upper respiratory tract infection. The most common Grade 3 or 4 laboratory abnormalities reported in more than 2% were decreased phosphate, decreased hemoglobin, increased creatinine phosphokinase, increased alanine aminotransferase, decreased albumin, decreased lymphocytes, decreased leukocytes, increased aspartate aminotransferase, decreased potassium, and decreased sodium.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

Full prescribing information for these approvals can be found on the web at www.fda.gov/drugsatfda.

Health care professionals should report serious adverse events to www.fda.gov/medwatch.

Follow the Division of Drug Information on X (formally Twitter) @FDA_Drug_Info and the Oncology Center of Excellence @FDAOncology. Send your feedback via email to FDAOncology@fda.hhs.gov. Thanks for tuning in to the D.I.S.C.O. Burst Edition.

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