Tovorafenib (DAY101)

No tovorafenib price specific to first-line use exists, because no first-line indication is approved. In the United States, the WAC as of 1 January 2025 was $35,272.64 per carton of 100 mg tablets and $8,818.16 per single-use 7-day bottle of oral suspension; WAC does not reflect discounts or rebates. The Institute for Clinical and Economic Review reported an annual list price of $330,720 and an estimated annual net price of $297,648, assuming a 375 mg weekly dose. The only price outside the United States identified is the German company dossier, which reported annual therapy costs of €239,561.93 to €479,123.86 per patient based on the oral suspension; IQWiG judged the lower bound plausible and the upper bound overstated because tablets cost less, and the company estimated 6 to 156 patients in the statutory health insurance target population for relapsed or refractory disease. Treatment requires a BRAF alteration; the FDA-approved companion diagnostic FoundationOne CDx had an initial Medicare payment of $3,500.00 (July 2018 to March 2019).

No cost-effectiveness analysis or budget impact model of tovorafenib in any pLGG population has been published, and all health technology assessment activity concerns relapsed or refractory disease. The EU Joint Clinical Assessment found no direct comparative data and no comparative evidence for the full claimed indication, including comparisons with carboplatin plus vincristine or vinblastine; its only included comparison, an unanchored MAIC versus dabrafenib plus trametinib in BRAF V600E-mutated disease, had effective sample sizes of 5.81 to 14.26. In Germany, the G-BA orphan drug benefit assessment was published on 17 August 2026, with a decision expected in early November 2026. In France, HAS granted early access for patients who progressed after at least one systemic treatment and were previously treated with, or ineligible for, dabrafenib plus trametinib. The NICE appraisal (ID6557) was in scoping. A US federal employee health plan policy (October 2025) covers tovorafenib only for relapsed or refractory pLGG and considers other indications investigational.

Economic evidence for first-line targeted therapy exists only for the comparator dabrafenib plus trametinib in BRAF V600E pLGG. NICE (TA977) recommended dabrafenib plus trametinib for children and young people aged 1 year and over who need systemic treatment, applying a 1.2 severity weight to QALYs and an acceptable ICER of around £30,000 per QALY; the LGG ICERs are confidential. A model for England and Wales reported a probabilistic ICER of £26,606 per QALY gained, with a 70.7% probability of cost-effectiveness at £30,000 per QALY. A Canadian microsimulation of first-line dabrafenib plus trametinib versus chemotherapy (2024 Canadian dollars) estimated a gain of 2.21 QALYs at an incremental cost of CAD 554,769, an ICER of CAD 251,027 per QALY with lifetime treatment and CAD 44,740 per QALY with 2 years of treatment, and concluded that a price reduction would be required.

US OJEMDA net product revenue was $57.2 million in 2024 and $155.4 million in 2025 (4,635 prescriptions in 2025), and 2026 guidance was $225 million to $250 million, with the company describing adoption as standard of care in second-line pLGG. The company expected a mid-2027 FIREFLY-2 readout and a potential first-line approval in 2028. Management projections disclosed for the Servier acquisition assumed development of OJEMDA as a front-line therapy and projected total company net revenue, including other products, royalties, and milestones, of $261 million in 2026, rising to $1,131 million in 2035; OJEMDA-only forecasts were not disclosed. The company estimates approximately 1,100 new BRAF-altered pLGG diagnoses per year in US patients younger than 25 years, an addressable pool of 2,000 to 3,000 relapsed, progressive, or refractory patients per year, and a chronic duration of treatment over many years. Because first-line efficacy, treatment duration, uptake relative to chemotherapy and dabrafenib plus trametinib, first-line pricing, and the size of the eligible population are not established, the budget impact of first-line tovorafenib cannot be estimated from public data as of September 2026.