Cemiplimab in locally advanced or metastatic cutaneous squamous cell carcinoma: prospective real-world data from the DRUG Access Protocol.

Background: The DRUG Access Protocol provides patients with cancer access to registered anti-cancer drugs that are awaiting reimbursement in the Netherlands and simultaneously collects prospective real-world data (RWD). Here, we present RWD from PD-1 blocker cemiplimab in patients with locally advanced or metastatic cutaneous squamous cell carcinoma (laCSCC; mCSCC).
Methods: Patients with laCSCC or mCSCC received cemiplimab 350 mg fixed dose every three weeks. Primary endpoints were objective clinical benefit rate (CBR), defined as objective response (OR) or stable disease (SD) at 16 weeks, physician-assessed CBR, defined as clinician's documentation of improved disease or SD based on evaluation of all available clinical parameters at 16 weeks, objective response rate (ORR), and safety, defined as grade ≥ 3 treatment related adverse events (TRAEs) occurring up to 30 days after last drug administration. Secondary endpoints included duration of response (DoR), progression-free survival (PFS), and overall survival (OS).
Findings: Between February 2021 and December 2022, 151 patients started treatment. Objective and physician-assessed CBR were 54.3% (95% CI, 46.0-62.4) and 59.6% (95% CI, 51.3-67.5), respectively. ORR was 35.1% (95% CI, 27.5-43.3). After a median follow-up of 15.2 months, median DoR was not reached. Median PFS and OS were 12.2 (95% CI, 7.0-not reached) and 24.2 months (95% CI, 18.8-not reached), respectively. Sixty-eight TRAEs occurred in 29.8% of patients. Most commonly reported TRAE was a kidney transplant rejection (9.5%).
Interpretation: Cemiplimab proved highly effective and safe in this real-world cohort of patients with laCSCC or mCSCC, confirming its therapeutic value in the treatment of advanced CSCC in daily clinical practice.
Funding: The DRUG Access Protocol is supported by all participating pharmaceutical companies: Bayer, Janssen, Lilly, Merck, Novartis, Roche, and Sanofi.
Competing Interests: John B.A.G. Haanen reports research grants to the institution from Asher Bio, Amgen, BioNTech, Bristol Myers Squibb, Novartis, and Sastra Cell Therapy, consultation fees to the institution from Achilles Tx, AstraZeneca, BioNTech, Bristol Myers Squibb, CureVac, Eisai, GlaxoSmithKline, Imcyse, Immunocore, Instil Bio, Iovance Bio, Merck, Merck Sharp & Dohme, Neogene Tx, Novartis, Obsidian Tx, Roche, Sanofi, Sastra Cell Therapy, Third Rock Ventures, and T-Knife, and stock options from Neogene Tx and Sastra Cell Therapy. Mathilde Jalving received compensation to the institution for advisory roles for Pierre Fabre, Merck, and Novartis. Lot A. Devriese received compensation to the institution for advisory roles and education for Bristol Myers Squibb, Merck Sharp & Dohme, and Incyte. Astrid A.M. van der Veldt received compensation to the institution for advisory roles for Bristol Myers Squibb, Merck Sharp & Dohme, Eisai, Ipsen, Novartis, Pierre Fabre, Pfizer, Roche, and Sanofi. Maureen J.B. Aarts reports research grants to the institution from Merck-Pfizer and consultation fees to the institution from being on the advisory board from Amgen, Bristol Myers Squibb, Novartis, Merck Sharp & Dohme, Merck-Pfizer, Pierre Fabre, Sanofi, Astellas, and Bayer. Carla M.L. van Herpen reports research grants to the institution from Astra Zeneca, Bayer, Bristol Myers Squibb, Elevar, Ipsen, Merck Sharp & Dohme, Merck, Novartis, and Sanofi, payments for lectures to the institution from ICCR, NIFU, RIVM, Roije Congressen, Nederlands Paramedisch Instituut, Elevar, and Uitgeverij Jaap, and payments to the institution for participating on an advisory board to the institution from Elevar. Mariette Labots received compensation to the institution for education for Bristol Myers Squibb and Janssen. Sahar Barjesteh van Waalwijk van Doorn-Khosrovani reports the PRIME-ROSE—A European precision cancer medicine trial network and implementation initiative funded by the EU Cancer Mission; grant no. 101104269 paid to the Leiden University Medical Center and travel expenses for attending meetings at the Nordic Precision Medicine Forum and Center for Innovation in Regulatory Science. Egbert F. Smit reports compensation to the institution for advisory roles at AstraZeneca, Bristol Myers Squibb, Boehringer Ingelheim, Daiichi Sankyo, Eli Lilly, Janssen, Merck Sharp & Dohme, Roche, Sanofi, and Takeda, personal compensation for education from Boehringer Ingelheim and Daiichi Sankyo, and compensation to the institution for participation in the data safety monitoring board of DSI. The other authors have no conflicts of interests to disclose.
(© 2024 The Author(s).)