Efficacy and safety of avelumab treatment in patients with metastatic Merkel cell carcinoma: experience from a global expanded access program.

BACKGROUND

Avelumab, a human anti-programmed death-ligand 1 immunoglobulin G1 monoclonal antibody, showed favorable efficacy and safety in patients with metastatic Merkel cell carcinoma (mMCC) in the phase II JAVELIN Merkel 200 trial, leading to approval in multiple countries. We describe real-world experience with avelumab in patients with mMCC from an expanded access program.

METHODS

Eligible patients had mMCC and progressive disease during or after chemotherapy or were ineligible for chemotherapy or clinical trial participation. Patients received an initial 3-month supply of avelumab (administered as 10 mg/kg intravenously every 2 weeks until progressive disease or unacceptable toxicity); resupply was allowed following complete response, partial response, stable disease, or clinical benefit per physician assessment.

RESULTS

Between December 15, 2015, and March 4, 2019, 558 of 620 requests from 38 countries were medically approved, and 494 patients received avelumab. Among 240 evaluable patients, the objective response rate was 46.7% (complete response in 22.9%, including 3 of 16 potentially immunocompromised patients), and the disease control rate was 71.2%. The median duration of treatment in evaluable patients with response was 7.9 months (range, 1.0-41.7) overall and 5.2 months (range, 3.0-13.9) in immunocompromised patients. No new safety signals were identified. The expanded access program closed for new requests on December 31, 2018, as required after regulatory approval; benefitting patients continued to receive avelumab.

CONCLUSIONS

The avelumab expanded access program for patients with mMCC demonstrated efficacy and safety in a real-world setting, consistent with the results from JAVELIN Merkel 200, and provided a treatment for patients with limited options.

Informations de copyright

© Author(s) (or their employer(s)) 2020. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

Déclaration de conflit d'intérêts

Competing interests: CL reports a consulting or advisory role for Amgen, Bristol-Myers Squibb, Merck Sharp & Dohme, Novartis, and Roche; speaker services for Amgen, Bristol-Myers Squibb, Novartis, and Roche; research funding from Bristol-Myers Squibb and Roche; and travel expenses from Amgen, Bristol-Myers Squibb, and Roche. GG reports a consulting or advisory role for Bayer, Eisai, Eli Lilly, Novartis, Pfizer, and PharmaMar; research funding from Bayer, Novartis, and PharmaMar; and travel expenses from PharmaMar. PN reports a consulting or advisory role for AstraZeneca, Bristol-Myers Squibb, Novartis, Immunocure, Roche, and Pfizer. RM reports a consulting or advisory role for Eli Lilly, Merck KGaA, Merck Sharp & Dohme, Pierre Fabre, Roche, and Sanofi; speaker services for Bristol-Myers Squibb, Merck Sharp & Dohme, Novartis, and Roche; research funding from Bristol-Myers Squibb, Eli Lilly, Merck KGaA, Merck Sharp & Dohme, Novartis, and Roche; and travel expenses from Bristol-Myers Squibb, Merck Sharp & Dohme, Roche, and Sanofi. PAA reports a consulting or advisory role for 4SC, Array, AstraZeneca, Bristol-Myers Squibb, Genmab, Idera, Immunocore, Incyte, MedImmune, Merck KGaA, Merck Sharp & Dohme, NewLink Genetics, Novartis, Pierre Fabre, Roche/Genentech, Sandoz, Sanofi, Sun Pharma, Syndax, and Ultimovacs; research funding from Array, Bristol-Myers Squibb, and Roche/Genentech; and travel expenses from Merck Sharp & Dohme. SS reports a consulting or advisory role for Amgen, AstraZeneca, Bristol-Myers Squibb, Janssen, Merck Sharp & Dohme, Novartis, and Roche; and research funding from Amgen, AstraZeneca, Bristol-Myers Squibb, and Merck Sharp & Dohme. EB is an employee of Merck KGaA, and SF, JR, and VK are employees of EMD Serono, a business of Merck KGaA. AE is an employee of Pfizer Pharma. SH is an employee of and has ownership of equity in Pfizer. All other authors declare that they have no competing interests.