China Approves Only Two Romiplostim Products: Qilu Pharma’s Rulisheng® Emerges as a Scarce TPO Receptor Agonist with New IND Filings

Pharnexcloud data (www.pharnexcloud.com) shows that on April 2, 2024, the National Medical Products Administration (NMPA) of China approved Qilu Pharmaceutical's Romiplostim for Injection N01 (Ruilisheng).^®) has been approved for marketing, becoming the first domestically produced Romiplostim Injection in China. This product isClass 3.4 BiologicsRuilisheng^®Indicated for adult (≥18 years old) patients with chronic immune thrombocytopenia (ITP) who have had an inadequate response to other treatments (e.g., corticosteroids, immunoglobulins). According to PharmaCircle: 250μg/vial.PricingRMB 2980.00 (including tax).

Pharnex Cloud Data www.pharnexcloud.com

The original research product Romiplostim is from Amgen Inc. (USA) / Kyowa Hakko Kirin (Japan), with the trade name Nplate. Currently, there are only two Romiplostim products available on the Chinese market.

Primary Immune Thrombocytopenia (ITP) is an acquired autoimmune bleeding disorder characterized by an unexplained isolated reduction in peripheral blood platelet count. The main pathogenic mechanism involves the loss of immune tolerance to platelet autoantigens, leading to abnormal activation of both humoral and cellular immunity, which jointly mediate accelerated platelet destruction and insufficient platelet production by megakaryocytes.

The annual incidence rate of adult ITP is (2-10)/100,000, with the elderly population over 60 years old being the high-risk group. The clinical manifestations of this disease vary significantly and can include asymptomatic thrombocytopenia, mucocutaneous bleeding, severe internal bleeding, and life-threatening intracranial hemorrhage. The risk of fatal bleeding in elderly patients is notably higher than in younger patients. ITP patients often experience fatigue and anxiety symptoms, which also greatly impact their quality of life.

On September 27-28, 2024, the 2024 American Society of Clinical Oncology Quality Care Symposium (ASCO QCS) Annual Meeting was held, where Qilu Pharmaceutical's Romiplostim for Injection N01 (Ruilisheng) was presented.^®) The latest data from the Phase 2/3 clinical trial for chemotherapy-induced thrombocytopenia (CIT) was presented at the conference.

Romiplostim is a second-generation thrombopoietin (TPO) receptor agonist used for the treatment of primary chronic immune thrombocytopenia (ITP). This clinical study recruits patients with solid tumors or lymphomas experiencing chemotherapy-induced thrombocytopenia (CIT), led by Professor Li Jin from East Hospital affiliated with Tongji University. Part A of the study is an open-label clinical trial, dividing participants into three groups based on different baseline platelet count levels (100~200×10^9/L or <100×10^9/L) and starting doses of Romiplostim N01 (1μg/kg or 2μg/kg), to explore the optimal dosing regimen of Romiplostim N01 in CIT patients. The primary endpoint is the effective response rate on the last day of the chemotherapy cycle. Part B of the study is a double-blind, randomized, placebo-controlled clinical trial evaluating the efficacy and safety of Romiplostim N01 in preventing the occurrence of CIT. The primary endpoint is the proportion of patients showing an effective response during the double-blind period.

Part A included a total of 50 subjects. The first group (baseline platelet count 100-200 × 10^9/L; initial dose 1 μg/kg), the second group (baseline platelet count 100-200 × 10^9/L; initial dose 2 μg/kg), and the third group (baseline platelet count <100 × 10^9/L; initial dose 2 μg/kg) consisted of 15, 15, and 20 cases, respectively. The effective response rates for the three groups were 66.7%, 53.3%, and 90.0%, respectively. Only one patient (from the third group) experienced chemotherapy delay ≥4 days or a chemotherapy dose reduction ≥20%. The incidence rates of treatment-related adverse events (TRAE) in the three groups were 26.7%, 20.0%, and 20.0%, respectively. No treatment-related serious adverse events occurred. There were no treatment-related bleeding or death events.

Part B included a total of 63 subjects who were randomly assigned to the Romiplostim N01 group (41 subjects) or the placebo group (22 subjects). The proportion of patients with an effective response in the two groups was 68.3% and 40.9%, respectively, with an adjusted rate difference of 27.6%. In the Romiplostim N01 group and the placebo group, 75.6% and 40.9% of patients, respectively, did not experience chemotherapy delays, dose reductions, or discontinuations due to thrombocytopenia. The proportion of patients not requiring rescue treatment was 78.0% in the Romiplostim N01 group and 63.6% in the placebo group. The incidence of TRAEs of any grade in the Romiplostim N01 group and the placebo group was 41.5% and 54.4%, respectively, with one case (2.4%) of Grade ≥3 TRAE in the Romiplostim N01 group. No treatment-related bleeding or death events occurred.

In addition, the efficacy data of patients in parts A and B of the study showed that the proportion of patients with an effective response in the N01 group of romiplostim and the placebo group were 73.3% (55/75) and 40.9% (9/22), respectively, with an adjusted rate difference of 37.7%.

Reference:

NMPA/CDE；

Pharnex Cloud Data www.pharnexcloud.com;

FDA/EMA/PMDA；

Relevant companies disclose publicly;

Qilu Pharmaceutical;

Chinese Guidelines for the Diagnosis and Treatment of Adult Primary Immune Thrombocytopenia (2020 Edition). Chinese Journal of Hematology. August 2020, Volume 41, Issue 8: 617-624;

Zhou H, Han SQ, Jin J, Huang RB, Guo XH, Shen XL, et al. Efficacy and safety of QL0911 in adult patients with chronic primary immune thrombocytopenia: A multicenter, randomized, double-blind, placebo-controlled, phase III trial. J Transl Int Med. 2023 Dec 20;11(4):423-432. doi: 10.2478/jtim-2023-0106；

This article is intended to provide scientific information to healthcare professionals only, does not represent the platform's position, and does not make any medication recommendations.