Shanghai, China, April 7th, 2022 - Shanghai Henlius Biotech, Inc. (2696.HK) 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.
According to GLOBOCAN 2020, lung cancer (LC) is the second most common cancer around the world. There were 2.2 million new LC cases and 1.8 million new deaths in 2020 worldwide, and LC is the leading cause of cancer deaths. SCLC accounts for 15%–20% of all cases and is the most aggressive type of LC. It is classified into two stages: limited stage (LS-SCLC) and extended stage (ES-SCLC), with both exhibiting high malignancy, strong invasiveness, early metastasis, fast disease progression, and a poor prognosis. The advent of immune checkpoint inhibitors has been proved to bring hope to patients with ES-SCLC but fraught with challenges. In recent years, a number of PD-1 mAbs have failed in the area. On the other hand, the standard treatment regimens for LS-SCLC is surgery, chemotherapy and concurrent radiotherapy. Traditional chemotherapeutic drugs did not exhibit significant progress in patients with LS-SCLC, and most patients tend to develop drug resistance or rapid relapse[2-4].
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 the 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.
In the future, Henlius will continue to emphasize unmet clinical needs and actively promote the combination immunotherapy of serplulimab and international regulatory registration to benefit more patients around the world.
 Sung H, Ferlay J, Siegel RL, Laversanne M, Soerjomataram I, Jemal A, Bray F. Global Cancer Statistics 2020: GLOBOCAN Estimates of Incidence and Mortality Worldwide for 36 Cancers in 185 Countries. CA Cancer J Clin. 2021 May;71(3):209-249.
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