Once-Weekly Oral Medicine for Kids’ BRAF Brain Tumors

Ojemda is a once-weekly oral kinase inhibitor (tovorafenib) available as 100 mg tablets and as a 25 mg/mL oral suspension. On the US Prescribing Information (revised 08/2025, NDA 218033), it is indicated for patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harbouring a BRAF fusion or rearrangement, or BRAF V600 mutation. The US indication is an accelerated approval based on response rate and duration of response; continued approval may depend on confirmatory trial(s). Manufactured for Day One Biopharmaceuticals, Inc. (Brisbane, CA). In the EU, the marketing authorisation holder is Ipsen Pharma. This is not a cure for every pediatric brain tumour, not for tumours without a qualifying BRAF alteration, and not established under 6 months of age.

Distinct path — not this carton: Some BRAF V600E-directed combinations (for example dabrafenib + trametinib where labelled) are a different molecular door. Ojemda is a type II RAF inhibitor labelled across BRAF fusions/rearrangements and V600 in this pLGG setting after prior systemic therapy. Compare biomarkers and labels with the neuro-oncology clinic; products are not interchangeable.

Where this is taking place

Commercial labelled doors today:

• United States — FDA accelerated approval (23 April 2024 original action; current USPI Revised 08/2025). Prescribe through a clinician experienced in cancer medicines; confirm BRAF alteration before starting. US patient support: EveryDay Support From Day One™ — 1-855-329-1246 (1-855-DAY-1BIO); www.OJEMDA.com / www.everydaysupport.com. AE reporting on USPI highlights: Day One 1-855-329-1246; Patient Information also lists 1-877-204-2820.

• European Union — European Commission conditional marketing authorisation 20 April 2026 (EMA product EMEA/H/C/006140; CHMP opinion 26 Feb 2026). Ipsen Pharma MAH. Additional monitoring. Company must submit further comparative data (ongoing study vs chemotherapy).

• Switzerland — Swissmedic temporary authorisation 5 May 2026 (Art. 9a TPA) for ages ≥6 months with BRAF-altered pLGG after prior therapy; orphan framing on Swissmedic summary.

• Canada — Health Canada Notice of Compliance with Conditions (NOC/c) 13 July 2026 (Ipsen Biopharmaceuticals Canada Inc.; control 301603). DINs 02569817 (100 mg tablets) and 02569876 (25 mg/mL oral suspension). Product Monograph on the Drug Product Database. NOC/c means market authorisation with conditions pending further confirmatory data — authorised is not funded (provincial formulary / private plan separate).

Outside labelled jurisdictions: As of this draft, MHRA, TGA (full ARTG registration), PMDA/MHLW, and NMPA authorisation for Ojemda / tovorafenib not confirmed on sources used here. TGA has a provisional determination only (designation ≠ ARTG licence). A US, EU, Swiss, or Canadian carton is not another country’s label. Do not import on your own.

FIREFLY-1 — NCT04775485: Multicenter, open-label, single-arm trial; US efficacy population N=76 with measurable disease (USPI). Sponsor framing: Day One clinical program. Pivotal evidence supports labelling — that is not the same sentence as open walk-in enrolment into the labelled commercial door without a neuro-oncology clinic and (where required) biomarker confirmation.

The practical door is a pediatric neuro-oncology clinic that can confirm relapsed/refractory pLGG, document the BRAF alteration, calculate body-surface-area dose, choose tablet vs suspension, and monitor bleeding, skin/sun reactions, liver tests, and growth.

Approval matrix

Access by country

• United States: Ask a pediatric neuro-oncology clinic about labelled Ojemda. Path: confirm relapsed/refractory pLGG → document BRAF fusion/rearrangement or V600 (FDA-approved tests listed at fda.gov/companiondiagnostics) → baseline liver tests → BSA-based 380 mg/m² once weekly (max 600 mg) as tablets or suspension → enroll in EveryDay Support From Day One (1-855-329-1246) if the clinic uses that channel. No list price or copay dollars in this article.
• European Union / EEA: Ask the treating oncology centre about EC-conditional Ojemda under the centralised MA. National reimbursement and hospital formulary steps still apply — authorised is not funded.
• Switzerland: Ask the Swiss oncology centre about temporary Swissmedic authorisation and local supply rules.
• Canada: Ask a Canadian pediatric neuro-oncology clinic about labelled Ojemda under Health Canada NOC/c (13 Jul 2026). Path: confirm r/r pLGG → document BRAF fusion/rearrangement or V600 → counsel on NOC/c conditions → prescribe tablets or suspension per Product Monograph → check provincial / hospital formulary (NOC/c ≠ automatic funding). DINs 02569817 / 02569876. No prices in this article.
• UK / Australia / Japan / other countries: Marketing authorisation not confirmed here (AU provisional determination ≠ ARTG). Ask the local clinician what legal paths exist — without treating a labelled-country carton as a foreign carton.
• If your regulator has not authorised it: do not import on your own.

Who is eligible

United States (USPI Rev. 08/2025):

• Indication: Patients 6 months and older with relapsed or refractory pediatric LGG harbouring a BRAF fusion or rearrangement, or BRAF V600 mutation.
• Patient selection: Confirm BRAF alteration in tumour specimens before starting.
• Dose: 380 mg/m² orally once weekly (maximum 600 mg once weekly), with or without food, until progression or unacceptable toxicity. BSA < 0.3 m²: recommended dosage not established.
• Forms: Tablets 100 mg; oral suspension 25 mg/mL.
• Contraindications: None (USPI §4).
• Exclude framing from FIREFLY-1 (trial, informs label population honesty): tumours with additional activating alterations (e.g. IDH1/2, FGFR) or known/suspected NF1 were excluded from the pivotal efficacy cohort — discuss NF1 and molecular profile with the clinic (USPI also warns about NF1-associated tumours).

European Union (EMA overview / indication): Monotherapy for patients 6 months and older with pLGG harbouring BRAF fusion/rearrangement or BRAF V600 mutation who have progressed after one or more prior systemic therapies.

Canada (Health Canada RDS / Product Monograph — Date of Authorization 2026-07-13): Monotherapy for patients 6 months of age and older with relapsed or refractory pediatric LGG harbouring a BRAF fusion or rearrangement, or BRAF V600 mutation, who have received one or more prior systemic therapies. A validated test is required to identify BRAF status. Authorised under NOC/c (conditions pending further data).

Not labelled on sources confirmed for this draft:

• Children younger than 6 months.
• pLGG without a qualifying BRAF alteration on the labelled tests.
• MHRA, TGA full ARTG, PMDA, or NMPA marketing authorisations (not confirmed here).

What the pivotal study showed (FIREFLY-1 / NCT04775485)

Use the USPI, Canadian Product Monograph, local SmPC, or Swiss information for a prescription conversation in that jurisdiction.

• Multicenter, open-label, single-arm; efficacy population N=76 with measurable disease.
• Median age 8.5 years (range 2–21); median 3 prior systemic regimens; 59% prior MAPK pathway inhibitor.
• Major efficacy outcome (RAPNO-LGG by blinded independent review): overall response rate 53% (95% CI 41–64) — 29 partial responses (38%), 11 minor responses (14%); no complete responses in this RAPNO-LGG table.
• Among responders (N=40): median duration of response 18 months (95% CI 12.0–22.8); 65% with observed DoR ≥12 months; 50% ≥18 months. Median time to response 5.4 months.
• Exploratory: ORR 53% with BRAF fusion/rearrangement (n=64); 50% with V600E (n=12).

EMA medicine overview cites a related main-study framing (77 patients; 52.6% response; average response duration about 18 months) — align clinical counselling with the jurisdiction’s product information.

How it is taken

Example NDCs (tablets): weekly 400/500/600 mg cartons 82950-001-16 / -20 / -24; suspension bottle 82950-012-01.

Safety (what the label puts first)

USPI Warnings and Precautions:

1. Hemorrhage — including major/critical-site bleeding; epistaxis and intratumoral hemorrhage reported; monitor and modify dose per severity.
2. Skin toxicity including photosensitivity — limit UV exposure; sunscreen / protective clothing; withhold/reduce/stop per severity.
3. Hepatotoxicity — monitor liver tests before and during treatment.
4. Effect on growth — reductions in growth velocity; routinely monitor growth in pediatric patients.
5. Embryo-fetal toxicity — can cause fetal harm; effective nonhormonal contraception (hormonal contraceptives may be less effective).
6. NF1-associated tumours — increased tumour growth may occur.

Most common adverse reactions (≥30% on USPI highlights): rash, hair colour changes, fatigue, viral infection, vomiting, headache, hemorrhage, pyrexia, dry skin, constipation, nausea, dermatitis acneiform, upper respiratory tract infection.

Avoid moderate/strong CYP2C8 inhibitors or inducers with Ojemda; caution with certain CYP3A substrates and hormonal contraceptives (USPI §7).

How to talk to a doctor

Bring: pathology and imaging confirming pediatric low-grade glioma, molecular report showing BRAF fusion/rearrangement or V600, list of prior systemic therapies, growth chart, bleeding history, skin/sun sensitivity, current medicines, and pregnancy/contraception plans for adolescents of reproductive potential. Ask: “Does my child meet the labelled Ojemda population in this country, and should we use tablets or suspension at 380 mg/m² weekly?” Ask how EveryDay Support (US), Canadian hospital / provincial formulary under NOC/c, or local formulary rules (EU/CH) fit. Do not start or stop cancer medicines without the oncology team.

Research and regulatory team

• FIREFLY-1 (NCT04775485): pivotal evidence base on USPI / FDA accelerated-approval materials; Day One Biopharmaceuticals clinical program.
• US manufactured for: Day One Biopharmaceuticals, Inc., Brisbane, CA 94005.
• EU marketing authorisation holder: Ipsen Pharma, 70 rue Balard, 75015 Paris, France (EMA product page).
• FDA original accelerated approval action: 23 April 2024 (FDA hematology/oncology approvals page).
• EMA conditional MA: 20 April 2026; orphan designation 20 May 2021.
• Swissmedic temporary authorisation: 5 May 2026.
• Canada: Sponsor Ipsen Biopharmaceuticals Canada Inc.; Health Canada NOC/c 13 Jul 2026 (control 301603); DINs 02569817 / 02569876.

Canada zoom: NOC/c is real — provincial funding is separate

Health Canada authorised Ojemda (tovorafenib) with a Notice of Compliance with Conditions on 13 July 2026 (sponsor Ipsen Biopharmaceuticals Canada Inc.; control number 301603). Indication: monotherapy for patients 6 months and older with relapsed or refractory pediatric low-grade glioma harbouring a BRAF fusion or rearrangement, or BRAF V600 mutation, after one or more prior systemic therapies, with a validated BRAF test required (Regulatory Decision Summary RDS1789572712163; Product Monograph on DPD). DINs: 02569817 (100 mg tablets) and 02569876 (25 mg/mL oral suspension). NOC/c means the product can be sold in Canada while the manufacturer completes further studies — patients should be advised of the conditional nature. Provincial formularies, hospital oncology pathways, and private plans decide funding separately. Authorised is not funded. A US or EU carton is still not a Canadian DIN. Do not import on your own.

EU zoom: conditional MA is real — reimbursement is separate

EC conditional authorisation (20 Apr 2026) means Ojemda is authorised across the EU (and EEA countries that apply the centralised MA) while further data are generated. Hospital formulary, HTA, and national funding decisions can still delay the practical door. Authorised is not funded.

Bottom line

Ojemda is a once-weekly oral RAF inhibitor for people 6 months and older with BRAF-altered relapsed/refractory pediatric low-grade glioma, with US accelerated, EU conditional, Swiss temporary, and Canadian NOC/c authorisations. The near-term path is a pediatric neuro-oncology clinic that confirms the biomarker and manages weekly dosing and safety monitoring — not a DIY import, and not an assumption that every plan will fund it.

Primary sources

• Ojemda US Prescribing Information (NDA 218033; Rev. 08/2025)
• FDA accelerated approval announcement — tovorafenib / pLGG
• EMA EPAR — Ojemda
• Swissmedic summary — Ojemda temporary authorisation 5 May 2026
• Health Canada Regulatory Decision Summary — Ojemda (NOC/c 2026-07-13)
• Health Canada Product Monograph — Ojemda (DPD 00085349)
• ClinicalTrials.gov NCT04775485 (FIREFLY-1)
• EveryDay Support From Day One (company patient support) (company — not the regulator)