FDA Grants Orphan Drug Status to Serplulimab for Small Cell Lung Cancer


The FDA has granted orphan drug designation to serplulimab for the treatment of patients with small cell lung cancer.

The FDA has granted orphan drug designation to serplulimab (HLX10) for the treatment of patients with small cell lung cancer (SCLC), according to a press release issued by its developer, Shanghai Henlius Biotech, Inc.1

Serplulimab is the first dual mAbs combination therapy with the ability to target PD-L1- and VEGF-expressing tumors. The agent has investigational new drug approval from the National Medical Products Administration in China for the treatment of advanced solid tumors.2

Due to an unmet medical need in the SCLC field, the developer has multiple ongoing studies of serplulimab, exploring its safety and efficacy in patients with limited stage SCLC, and extensive-stage SCLC (ES-SCLC).

Recently, the randomized, double-blind, multicenter, phase 3 study of serplulimab plus chemotherapy in previously-untreated patients with ES-SCLC demonstrated improvement in overall survival (OS) compared with placebo and chemotherapy, meeting the study’s primary end point.3

“SCLC is a type of lung cancer with strong invasion and poor prognosis, among which ES-SCLC cancer cells are prone to metastasis. At present, ES-SCLC is still mainly treated with chemotherapy or chemotherapy combined with PD-L1 inhibitors. It is easy to progress after chemotherapy, and the 5-year survival rate is generally less than 5%. The prognosis has not been improved for a long time. The results of the Phase 3 trial of serplulimab bring a new option for anti-PD-1 mAb as a first-line ES-SCLC therapy,” said Jie Wan, MD, PhD, the lead investigator of the study and professor at Cancer Hospital Chinese Academy of Medical Sciences, in a statement about the study results.

According to interim analysis data reviewed by an independent data monitoring committee serplulimab and chemotherapy also had a positive safety profile, with no reports of new safety signals.

Overall, 567 patients with previously untreated ES-SCLC have been included in the study and randomly assigned 2:1 to receive either serplulimab plus chemotherapy consisting of carboplatin and etoposide or placebo plus chemotherapy. Eligible patients were those with histologically or cytologically confirmed disease, no prior systemic therapy for ES-SCLC, and adequate organ function.4

The study excludes patients with mixed SCLC according to histology or cytology, known history of severe allergy to any monoclonal antibody, known hypersensitivity to carboplatin or etoposide, as well as those who are pregnant, have a known history of psychotropic drug abuse or drug addiction, and those with any clinical feature that could lead to early treatment termination based on the investigators’ discretion. The study is recruiting eligible patients with ES-SCLC at a site in Shanghai, China.

To evaluate OS, patients in the study are stratified by whether they have positive, negative, or unavailable PD-L1 expression level, the presence of brain metastases, and age (≥ 65 years versus < 65 years).


1. Henlius' serplulimab granted orphan-drug designation in the United States for small cell lung cancer. News release. Shanghai Henlius Biotech, Inc. April 7, 2022. Accessed April 7, 2022. https://prn.to/3NRJgIv

2. Serplulimab. Shanghai Henlius Biotech, Inc website. Accessed April 7, 2022. https://bit.ly/3r4ICh1

3. 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 7, 2022. https://bit.ly/3GkUfFI

4. A randomized, double-blind, placebo controlled phase III study to investigate efficacy and safety of HLX10 + chemotherapy (Carboplatin- Etoposide) in patients with extensive stage small cell lung cancer (ES-SCLC). Clinicaltrials.gov. Updated December 17, 2020. Accessed April 7, 2022. https://bit.ly/3DSuQU0

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