Serplulimab Granted Orphan Drug Designation by FDA in SCLC


Serplulimab, which has been granted orphan drug designation by the FDA, is being considered as a treatment for patients with small cell lung cancer.

The FDA had granted serplulimab (Hansizhuang) an orphan drug designation for the treatment of small cell lung cancer (SCLC), according to a press release from developer Shanghai Henlius Biotech.1

The designation was granted to continue the development of serplulimab and to take advantage of a policy that allows for better support of registration and commercialization within the United States. Further plans in 2022 for serpulimab include submitting a new drug application in China and a marketing authorization application in Europe, which could potentially make serplulimab the first PD-1 inhibitor to be used in the frontline setting for patients with SCLC.

Serplulimab was also assessed in combination with chemotherapy in a phase 3 trial (NCT04063163) of patients with previously untreated extensive-stage SCLC (ES-SCLC).2 At the first interim analysis, the combination met the primary end point, yielding a significant improvement in overall survival (OS) compared with chemotherapy alone. Additionally, the combination demonstrated a positive safety profile with no new safety findings.

The trial’s estimated enrollment was 567 patients, and the primary end point was OS. Patients were required to have histologically or cytologically confirmed disease with no previous systemic therapies. Additional inclusion criteria included good major organ function. Those with mixed disease, severe allergies to any monoclonal antibody, and hypersensitivity to carboplatin or etoposide were excluded from the study.

“I’m very excited to see that the phase 3 study of serplulimab in ES-SCLC has met its primary end point OS, and its efficacy and safety have been fully validated. This study is the first and largest ES-SCLC international multicenter clinical study led by Chinese researchers for [and anti–PD-1 monoclonal antibody]. We are looking forward to serplulimab launching as soon as possible,” principal investigator Ying Cheng, MD, director of Department of Medical Oncology at Jilin Cancer Hospital, said in a press release.

Additionally, the phase 3 ASTRUM-005 study assessed serplulimab plus placebo when combined with carboplatin and etoposide in previously untreated SCLC. Among the 585 patients who enrolled on the study, the reduction in risk of death was 38%. A new drug application will likely be filed based on these findings.


  1. Henlius' serplulimab granted orphan-drug designation in the United States for small cell lung cancer. News release. Henlius. April 7, 2022. Accessed April 8, 2022.
  2. Henlius' phase 3 clinical study of novel PD-1 inhibitor serplulimab for the treatment of first-line extensive-stage small cell lung cancer (ES-SCLC) meets primary study endpoint. News release. Henlius. December 7, 2021. Accessed April 8, 2022.
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