FDA Grants Fast Track Designation to EVX-01 Plus Pembrolizumab in Metastatic Melanoma


The FDA has granted a fast track designation to EVX-01 in combination with pembrolizumab for the treatment of patients with metastatic melanoma.

The FDA has granted a fast track designation to EVX-01 in combination with pembrolizumab (Keytruda) for the treatment of patients with metastatic melanoma.1

EVX-01 is a personalized peptide-based therapeutic vaccine comprised of multiple peptides, each representing a neoepitope only found in the tumor of an individual patient, in combination with a liposomal adjuvant (CAF09b), to create a single vaccine formulation.2

“We are extremely pleased that our cancer vaccine candidate EVX-01 has received the FDA fast track designation, as it enables a potentially faster approval of the vaccine. This is first and foremost to the benefit of the patients. And it is a great validation of our AI platform, PIONEER, and our drug development candidate,” Per Norlén, chief executive officer at Evaxion, stated in a news release.

Data from a phase 1 trial (NCT03715985) showed that EVX-01 monotherapy produced long-lasting EVX-01–specific T-cell responses in all patients with metastatic melanoma, and no severe adverse effects were reported.3 Investigators also observed a manufacturing time of 48 to 55 days for EVX-01, allowing patients to begin treatment within 60 days from baseline biopsy.

The combination of EVX-01 plus pembrolizumab is under investigation in a phase 2 trial (NCT05309421) in patients with unresectable or metastatic melanoma, which the FDA approved to proceed in December 2022.4

The study is enrolling patients who are at least 18 years of age with histologically confirmed, and not amenable to local therapy, metastatic or unresectable stage III or IV melanoma. Patients cannot have uveal or ocular melanoma, must be naïve to checkpoint inhibitor therapy, and must have testing for a BRAF mutation prior to study entry. Other inclusion criteria include measurable disease per RECIST v1.1 criteria and an ECOG performance status of 0 or 1.

Key exclusion criteria include prior systemic therapy, including investigational agents, within 4 weeks of study treatment; prior radiotherapy within 2 weeks of study treatment; or receipt of a live or live-attenuated vaccine within 30 days prior to the first dose of study treatment.

All patients enrolled in the single-arm study are receiving EVX-01 in combination with standard-of-care pembrolizumab.

The primary end point of the trial is best overall response through study completion. Secondary end points include overall response rate, progression-free survival, overall survival, and safety.


  1. Evaxion receives FDA fast-track designation for personalized cancer immunotherapy. News release. Evaxion. January 19, 2023. Accessed January 20, 2023. http://bit.ly/3wkvJBu
  2. Long GV, Ferrucci PF, Khattak A, et al. KEYNOTE - D36: personalized immunotherapy with a neoepitope vaccine, EVX-01 and pembrolizumab in advanced melanoma. Future Oncol. 2022;18(31):3473-3480. doi:10.2217/fon-2022-0694
  3. Mørk SK, Kadivar M, Bol KF, et al. Personalized therapy with peptide-based neoantigen vaccine (EVX-01) including a novel adjuvant, CAF®09b, in patients with metastatic melanoma. Oncoimmunology. 2022;11(1):2023255. doi:10.1080/2162402X.2021.2023255
  4. A single arm trial evaluating the efficacy and safety of EVX-01 in combination with pembrolizumab in adults with unresectable or metastatic melanoma. ClinicalTrials.gov. Updated January 9, 2023. Accessed January 20, 2023. https://clinicaltrials.gov/ct2/show/NCT05309421
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