Evicenter
P&T meetings

Tovorafenib (DAY101)

RAF-altered pediatric low-grade glioma requiring first-line systemic therapy

Also known as TAK-580
Regulatory submission
Not announced
Launch
Not announced
Last updated
155 sources

Product information and disease descriptionProduct description

Phase of product development

14 evidence topics · 15 sources

Summary: regulatory and development status as of September 2026

Tovorafenib is approved in the United States (accelerated approval, 23 April 2024), the European Union (conditional marketing authorisation, April 2026), and Japan (16 September 2026). The US and EU indications are limited to relapsed or refractory (EU: progressed after one or more prior systemic therapies) pediatric low-grade glioma with a BRAF fusion or rearrangement or BRAF V600 mutation, based on the single-arm phase 2 FIREFLY-1 trial. The FDA listed the accelerated approval as ongoing (clinical benefit not yet verified) as of 16 September 2026. No approved indication, supplemental new drug application, or EU variation for first-line use was identified. The first-line trial LOGGIC/FIREFLY-2 (tovorafenib vs standard-of-care chemotherapy) completed enrollment of approximately 400 participants in May 2026; the sponsor expects topline data by mid-2027. FIREFLY-2 is also the confirmatory trial for the US accelerated approval (interim report due April 2028, final report April 2032) and the specific obligation for the EU conditional authorisation (final report due 30 April 2032). The EMA overview page gives 20 April 2026 as the authorisation date, while the Ipsen announcement is dated 22 April 2026. Day One Biopharmaceuticals became a wholly owned subsidiary of Servier after the tender offer closed on 22 April 2026.

United States: accelerated approval for relapsed or refractory pLGG

“On April 23, 2024, the Food and Drug Administration granted accelerated approval to tovorafenib (Ojemda, Day One Biopharmaceuticals, Inc.) for patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.” (opens the source at this quote in a new tab)

“This represents the first FDA approval of a systemic therapy for the treatment of patients with pediatric LGG with BRAF rearrangements, including fusions.” (opens the source at this quote in a new tab)

“This application was granted accelerated approval based on overall response rate and duration of response. Continued approval may be contingent upon verification of clinical benefit in confirmatory trials.” (opens the source at this quote in a new tab)

United States: approval pathway stated in the approval letter

“It is approved under accelerated approval pursuant to section 506(c) of the Federal Food, Drug, and Cosmetic Act (FDCA) and 21 CFR 314.510, effective on the date of this letter, for use as recommended in the enclosed agreed-upon labeling.” (opens the source at this quote in a new tab)

“Your application for Ojemda was not referred to an FDA advisory committee because the application did not raise significant safety or efficacy issues that were unexpected for a drug in the intended population.” (opens the source at this quote in a new tab)

United States: expedited programs and designations

“This application was granted priority review, breakthrough designation and orphan drug designation.” (opens the source at this quote in a new tab)

“Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma and from the European Commission for the treatment of glioma.” (opens the source at this quote in a new tab)

United States: breakthrough therapy and orphan designation dates

“31 Aug 2020 IND 108340 Breakthrough Therapy Designation granted by FDA in the treatment of pediatric patients with low-grade glioma harboring an activating RAF alteration who have progressed after one or more therapies.” (opens the source at this quote in a new tab)

“24 Sep 2020 IND 108340 Orphan Drug Designation granted by FDA for tovorafenib for the treatment of malignant glioma.” (opens the source at this quote in a new tab)

United States: rare pediatric disease priority review voucher

“We also inform you that you have been granted a rare pediatric disease priority review voucher, as provided under section 529 of the FDCA.” (opens the source at this quote in a new tab)

United States: August 2025 labeling supplements

“Prior Approval sNDA 002 provides for updates to the U.S. Prescribing Information (USPI) and Patient Information labeling, based on safety and efficacy data generated from Study DAY101-001/PNOC026 (FIREFLY-1); and references the availability of an FDA-approved companion diagnostic.” (opens the source at this quote in a new tab)

““Changes Being Effected” sNDA 003 provides for a new 20 count carton configuration comprised of four blister cards each containing five 100 mg tablets to support the 500 mg once weekly dose packaging configuration.” (opens the source at this quote in a new tab)

United States: accelerated approval status in September 2026

“Patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.” (opens the source at this quote in a new tab)

First-line pLGG: FIREFLY-2 enrollment completion and expected data timing

“Trial aims to extend OJEMDA's indication beyond relapsed/refractory setting to address significant unmet need; topline data expected by mid-2027” (opens the source at this quote in a new tab)

“today announced that it has completed enrollment in the FIREFLY-2 clinical trial, which is evaluating the efficacy, safety and tolerability of tovorafenib vs standard of care chemotherapy in the front-line setting of therapy for patients aged 6 months to 25 years with low-grade glioma.” (opens the source at this quote in a new tab)

“It is being conducted at approximately 140 sites across the U.S., Canada, Europe, Australia, South America, Middle East and Asia, and in just over three years has enrolled approximately 400 participants who are receiving either tovorafenib once weekly or one of four standard of care (SoC) chemotherapy regimens.” (opens the source at this quote in a new tab)

“The primary analysis is expected to occur approximately 12 months after the last patient is enrolled; preliminary insights are expected to be available in 2027.” (opens the source at this quote in a new tab)

First-line pLGG: FIREFLY-2 status at the time of the EU assessment

“Study DAY101-002 [LOGGIC/FIREFLY-2] is a Phase 3, randomized, global, multicenter trial of tovorafenib monotherapy versus standard of care chemotherapy in paediatric low-grade glioma harboring an activating RAF alteration requiring first-line systemic therapy.” (opens the source at this quote in a new tab)

“As of 15 January 2025, there are currently 110 active sites and 169 randomized patients.” (opens the source at this quote in a new tab)

Regulatory submission

No evidence found.

Launch

No evidence found.

European Union: conditional marketing authorisation

“Ojemda has been given conditional authorisation for use in the EU. This means that it has been authorised on the basis of less comprehensive data than are normally required, because it fulfils an unmet medical need.” (opens the source at this quote in a new tab)

“Ojemda received a conditional marketing authorisation valid throughout the EU on 20 April 2026.” (opens the source at this quote in a new tab)

“today announced that the European Commission (EC) has granted conditional marketing authorization for Ojemda® (tovorafenib) as monotherapy for the treatment of patients 6 months of age and older with pediatric low-grade-glioma harboring a BRAF fusion or rearrangement, or BRAF V600 mutation, who have progressed after one or more prior systemic therapies.” (opens the source at this quote in a new tab)

“This EC decision applies across all 27 EU Member States, as well as Iceland, Liechtenstein, and Norway.” (opens the source at this quote in a new tab)

European Union: assessment procedure and orphan designation

“The CHMP did not grant this request, as it considered that, based on the submitted data package, the justification for an accelerated assessment was not sufficiently substantiated.” (opens the source at this quote in a new tab)

“Orphan Designation for the treatment of glioma granted on 20 May 2021 (EU/3/21/2434)” (opens the source at this quote in a new tab)

“The application was received by the EMA on 26 February 2025” (opens the source at this quote in a new tab)

Japan: marketing approval

“IPSEN Co., Ltd. obtained a regulatory approval from the MHLW on September 16, 2026.” (opens the source at this quote in a new tab)

“A key Japanese health ministry panel on August 24 recommended approval of a batch of new medicines and indications including a subcutaneous version of MSD’s PD-1 inhibitor Keytruda (pembrolizumab) and Ipsen’s targeted glioma therapy tovorafenib, known as Ojemda.” (opens the source at this quote in a new tab)

Other countries: approvals and orphan designations reported by the ex-US licensee

“Tovorafenib has also been approved in EAU and has been granted Orphan Drug Designation in Russia, Switzerland, Taiwan, Japan South Korea and Australia.” (opens the source at this quote in a new tab)

United Kingdom, Canada, and Australia: marketing authorisation

No evidence found.