IDEAYA Biosciences Secures US FDA Breakthrough Therapy Designation for Darovasertib in Neoadjuvant Uveal Melanoma
Tuesday, April 01, 2025
IDEAYA Biosciences, Inc, a precision medicine oncology company focused on developing targeted therapeutics, has received Breakthrough Therapy designation (BTD) from the U.S. Food and Drug Administration (FDA) for darovasertib. This potential first-in-class protein kinase C (PKC) inhibitor is intended for the neoadjuvant treatment of adult patients with primary uveal melanoma (UM) who have been advised to undergo enucleation.
The BTD follows the Fast Track designation granted earlier for the combination of darovasertib with crizotinib in treating metastatic uveal melanoma (MUM). A Phase 2/3 trial evaluating this combination in first-line HLA-A2-negative MUM is currently in progress. Additionally, darovasertib has been classified as an Orphan Drug in UM, including MUM, providing IDEAYA with potential benefits such as tax credits, user fee exemptions, and market exclusivity.
The BTD application was supported by interim data from an ongoing Phase 2 open-label trial (NCT05907954) assessing darovasertib monotherapy in the neoadjuvant setting for localised UM. Data presented in September 2024 indicated an 82% tumour shrinkage rate and a 61% eye preservation rate. These findings were also shared at the ASCO 2024 conference. The company plans to present further data on efficacy, safety, radiation reduction, eye preservation, and vision improvement at medical conferences in 2025.
Several clinical updates related to neoadjuvant UM and MUM are scheduled for release throughout 2025, including median overall survival (mOS) data from the Phase 2 study (IDE196-001) in mid-year and late 2025. A median progression-free survival (mPFS) readout from the Phase 2/3 trial of darovasertib and crizotinib in first-line MUM is expected by the end of 2025. Furthermore, a Phase 3 randomised registrational trial for neoadjuvant UM is set to commence in the first half of 2025.
This potential Phase 3 study would assess neoadjuvant darovasertib in primary UM patients eligible for either enucleation (Cohort 1) or plaque brachytherapy (Cohort 2). The estimated annual incidence of neoadjuvant UM across North America, Europe, and Australia is approximately 12,000 patients, highlighting a significant unmet medical need as no FDA-approved systemic therapies currently exist.
BTD is intended to accelerate the development and regulatory review of treatments for serious or life-threatening conditions that show substantial improvement over existing options. The designation allows for enhanced FDA support, cross-disciplinary collaboration, and eligibility for rolling submission and priority review.
Source: prnewswire.com
