Novel Drug & Emergent Therapeutics | Pharmaceuticals | Ophthalmology
Neoadjuvant Uveal Melanoma Monotherapy Darovasertib Granted Breakthrough Drug Status
Time to read: 01:09
Originally published: 31 March 2025
Source: IDEAYA Biosciences
Type of article: Novel Drug & Emergent Therapy News
MedED Catalogue Reference: MNDO001
Category: Ophthalmology
Category tags: ophthalmology, uveal melanoma
| Product Category | Product Name | Company | Status |
|---|---|---|---|
| Breakthrough Drug, Phase 111 |
The U.S. FDA has granted Breakthrough Therapy Designation (BTD) for darovasertib, a first-in-class protein kinase C (PKC) inhibitor, for the neoadjuvant treatment of adult patients with primary uveal melanoma (UM) recommended for enucleation. This designation highlights the potential of darovasertib as a monotherapy in a patient population with no FDA-approved systemic treatments.
Clinical Development and Trial Data
The BTD application was supported by interim data from an ongoing Phase 2 trial (NCT05907954) evaluating darovasertib as a neoadjuvant monotherapy for localized UM. As of September 2024, data showed an 82% ocular tumor shrinkage rate and a 61% eye preservation rate.
Additionally, darovasertib received Fast Track designation for its combination with crizotinib in metastatic UM (MUM).
A Phase 2/3 registration-enabling trial for this combination in first-line HLA-A2-negative MUM is ongoing, with median progression-free survival (mPFS) data expected by late 2025. The drug has also been granted Orphan Drug status, providing regulatory benefits such as tax credits and market exclusivity.
Future Trials and Market Impact
IDEAYA Biosciences plans to launch a Phase 3 registrational trial in the first half of 2025 to assess darovasertib in primary UM patients eligible for enucleation (Cohort 1) or plaque brachytherapy (Cohort 2).
| Clinical Trial Status | ClinicalTrial.gov Registration | Study Name |
|---|---|---|
|
(Neo)Adjuvant IDE196 (Darovasertib) in Patients With Localized Ocular Melanoma |
||
| In progress | NCT05187884 | Neoadjuvant/?Adjuvant Trial of Darovasertib in Ocular Melanoma (NADOM) |
Access the Press Releases
31 March 2025 | IDEAYA Press Release| IDEAYA Biosciences Receives US FDA Breakthrough Therapy Designation for Darovasertib Monotherapy in Neoadjuvant Uveal Melanoma
Back to top
Disclaimer
This article is compiled from a variety of resources. Every effort has been made to correctly attribute quotes and content. Where possible all information has been independently verified. The Medical Education Network bears no responsibility for any inaccuracies which may occur from the use of third-party sources. If you have any queries regarding this article contact us
Fact-checking Policy
The Medical Education Network makes every effort to review and fact-check the articles used as source material in our summaries and original material. We have strict guidelines in relation to the publications we use as our source data, favouring peer-reviewed research wherever possible. Every effort is made to ensure that the information contained here is an accurate reflection of the original material. Should you find inaccuracies, out of date content or have any additional issues with our articles, please make use of the contact us form to notify us.
