Shanghai Henlius Biotech announced that the United States Food and Drug Administration (FDA) has granted Orphan-Drug Designation (ODD) for Hansizhuang (serplulimab) for the treatment of small-cell lung cancer (SCLC). This is the first such designation granted to Hansizhuang. The ODD granted by the FDA is beneficial for the continuous development of Hansizhuang and the enjoyment of certain policy support in terms of registration and commercialization in the United States. The company plans to file its NDA in China and MAA in the EU in 2022, making Hansizhuang potentially to be the world's first PD-1 inhibitor for the first-line treatment of SCLC.

Orphan drugs refer to pharmaceutical products developed for the prevention, diagnosis, and treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S. at the time of designation and to offer certain policy support, including but not limited to: 1) tax credits for clinical trial costs; 2) waiver of application fees for new drugs; and 3) seven years of market exclusivity without being affected by patent.

Based on the unmet clinical needs, Henlius promotes a series of immunotherapy studies in the field of SCLC, with the goal of providing more effective treatment approaches for patients. Henlius has undertaken multiple trials to investigate therapy options for both LS-SCLC and ES-SCLC, including the global multi-centre phase 3 study of Hansizhuang for the first-line treatment of ES-SCLC (ASTRUM-005) and the investigational new drug application (IND) of international multi-centre phase 3 studies of Hansizhuang (serplulimab) approval from the National Medical Products Administration (NMPA). In detail, ASTRUM-005 is a randomised, double-blind, international, multi-centre, phase 3 clinical study aimed to compare the efficacy and safety of HANSIZHUANG with placebo when combined with chemotherapy (carboplatin-etoposide) in previously untreated patients with SCLC. This study has set up about 128 sites in China, Turkey, Poland, Georgia, etc. and 585 subjects were enrolled, among whom 31.5% were Caucasian. In Dec 2021, ASTRUM-005 has met the primary study endpoint of overall survival (OS), reducing the risk of death by 38% of the overall population (41% in the Asian subgroup). Based on the excellent readouts, the company intends to file NDA for the indication shortly.