FDA Grants Fast Track Designation to Pembrolizumab Plus Personalized Immunotherapy for Metastatic Melanoma


The FDA has given fast track designation to peptide-based immunotherapy agent EVX-01 in combination with pembrolizumab for metastatic melanoma.

The FDA has granted fast track designation to EVX-01, a personalized cancer vaccine, plus pembrolizumab (Keytruda) for patients with metastatic melanoma, according to a press release from Evaxion Biotech.

The fast track designation for EVX-01 plus pembrolizumab followed the FDA’s go-ahead for a phase 2b clinical trial (NCT05309421) in which investigators are evaluating the combination in patients with metastatic melanoma.

Investigators are assessing EVX-01 plus pembrolizumab in patients with metastatic melanoma in an ongoing phase 2b clinical trial.

Investigators are assessing EVX-01 plus pembrolizumab in patients with metastatic melanoma in an ongoing phase 2b clinical trial.

“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,” Per Norlén, chief executive officer at Evaxion, said in the press release. “This is first and foremost to the benefit of the patients.”

EVX-01 is a peptide-based cancer immunotherapeutic that generates a individualized treatment for each patient based on a gene analysis of their tumors and on matching with their immune system. A proprietary AI platform called PIONEER serves as a primary component to the EVX-01 treatment process.

Investigators of the ongoing phase 2b study are evaluating EVX-01 in combination with pembrolizumab at clinical sites across the United States, Europe, and Australia. In a single experimental arm, patients will begin treatment with 25 mg/ml of pembrolizumab for up to 18 treatment cycles followed by immunization with EVX-01 administered intramuscularly at week 12 of treatment. Patients will receive a total of 10 doses of EVX-01, with 6 doses given 2 weeks apart and 4 booster shots administered at later times.

The primary end point of this trial is change in best overall response for up to 102 weeks per RECIST v1.1 criteria. Secondary end points include overall response rate, progression-free survival, overall survival, adverse effects (AEs) and serious AEs, immunologic responses, and the percentage of patients who received EVX-01.

Patients 18 years and older with histologically confirmed metastatic or unresectable stage III or IV melanoma were eligible to enroll on the trial. Additional inclusion criteria included having measurable disease per RECIST v1.1 criteria as assessed by a local site investigator 4 weeks prior to study treatment, and a willingness to provide a new or archival tumor tissue from an unresectable or metastatic site of disease for biomarker analysis. An ECOG performance status of 0 or 1 was also required.

Patients who received prior treatment with an anti–PD-1, anti–PD-L1, or anti–PD-L2 agent or any prior systemic anti-cancer therapy within 4 weeks prior to treatment were unable to enroll on the study. Patients were also unsuitable for enrollment if they received prior radiotherapy within 2 weeks of beginning study treatment or had received a live or live-attenuated vaccine within 30 days prior to starting study treatment.


Evaxion receives FDA fast-track designation for personalized cancer immunotherapy. News release. Evaxion Biotech. News release. January 19, 2023. Accessed January 19, 2023. bit.ly/3CZiIBo

Related Videos
Experts on myeloma
Related Content