HUTCHMED Announces NDA Acceptance in China for Sovleplenib for the Treatment of Primary Immune Thrombocytopenia with Priority Review Status
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Insights
The acceptance of the New Drug Application (NDA) for sovleplenib by the China National Medical Products Administration (NMPA) with Priority Review status is a pivotal step in the drug's regulatory pathway. Sovleplenib, a spleen tyrosine kinase (Syk) inhibitor, represents a potential advancement in the treatment of adult primary immune thrombocytopenia (ITP), a condition characterized by a low platelet count that can lead to bleeding and bruising. The designation of sovleplenib as a Breakthrough Therapy underscores the unmet need in this patient population and the drug's potential to offer significant improvements over existing therapies.
The clinical data from the Phase III ESLIM-01 trial is critical here, as it supports the efficacy and safety profile of sovleplenib. The trial's success in meeting both primary and secondary endpoints indicates a durable response rate, which is a key measure of long-term benefit for ITP patients. This could translate into fewer bleeding episodes and a reduced need for other therapies or interventions, potentially improving the quality of life for patients with ITP.
Furthermore, the impact of sovleplenib on the market for hematological treatments could be substantial, given the current limitations of available therapies. The novel mechanism of action of Syk inhibition could set a new standard for treatment efficacy. However, the long-term safety profile will be a critical factor in its market adoption, as ITP patients often require long-term treatment.
The announcement regarding sovleplenib's NDA acceptance and Priority Review by the NMPA is likely to be closely monitored by investors and competitors in the pharmaceutical industry. Sovleplenib's potential market entry in China, a significant pharmaceutical market, could disrupt the current ITP treatment landscape and capture a sizeable share due to its novel mechanism of action and the Breakthrough Therapy designation, which suggests expedited development and review processes.
For HUTCHMED, the successful trial and subsequent regulatory milestones could lead to an increase in stock valuation, as investors often react positively to developments that expand a company's product pipeline, especially in large markets such as China. The market will also be looking at the company's ability to successfully commercialize sovleplenib, including manufacturing and distribution capabilities, pricing strategy and market access. The company's performance in these areas will be essential for translating clinical success into financial success.
It is also important to note that the competitive dynamics of the ITP treatment market could be affected by the introduction of sovleplenib. As a result, companies with existing ITP treatments might need to reassess their market strategies, which could include price adjustments, increased marketing efforts, or even accelerated development of their own novel therapies.
From a health economics perspective, the introduction of sovleplenib as a treatment option for adult primary ITP could have significant implications for healthcare spending and resource allocation. The Priority Review status indicates a potential for a quicker path to market, which could lead to earlier access for patients and potential cost savings if the drug can reduce the need for hospitalizations and other costly interventions associated with ITP complications.
However, the pricing of sovleplenib will be a crucial factor in determining its cost-effectiveness and subsequent adoption by healthcare providers. Innovative treatments often come at a premium and reimbursement decisions by insurers and health authorities will depend on the drug's ability to demonstrate value in terms of improved patient outcomes and overall healthcare savings.
The economic burden of ITP is not solely limited to direct medical costs but also includes indirect costs such as lost productivity due to illness. If sovleplenib can significantly improve patient quality of life and reduce disease-related absenteeism, it could offer broader economic benefits. The long-term impact will depend on the balance between the drug's cost and its effectiveness in reducing the overall burden of ITP.
— NDA accepted and granted Priority Review following its Breakthrough Therapy designation granted in January 2022 —
— NDA is supported by data from successful Phase III ESLIM-01 trial in patients with adult primary immune thrombocytopenia who have received at least one previous therapy —
HONG KONG and SHANGHAI and FLORHAM PARK, N.J., Jan. 10, 2024 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (“HUTCHMED”) (Nasdaq/AIM:HCM; HKEX:13) today announces that the New Drug Application (“NDA”) for sovleplenib for the treatment of adult patients with primary immune thrombocytopenia (“ITP”) has been accepted for review and granted priority review by the China National Medical Products Administration (“NMPA”). Sovleplenib is a novel, selective, oral inhibitor targeting spleen tyrosine kinase (“Syk”), being developed for the treatment of hematological malignancies and immune diseases.
The NDA is supported by data from ESLIM-01, a randomized, double-blinded, placebo-controlled Phase III trial in China of sovleplenib in 188 adult patients with primary ITP who have received at least one prior line of standard therapy. In August 2023, HUTCHMED announced that the trial had met its primary endpoint of demonstrating a clinically meaningful and a statistically significant increase in durable response rate in patients treated with sovleplenib as compared to patients treated with placebo. Secondary endpoints including response rate and safety were also met. Full results will be published in due course. Results from the proof of concept study that led to the ESLIM-01 study were published in The Lancet Haematology.
The NMPA granted Breakthrough Therapy designation (“BTD”) to sovleplenib for the indication studied in ESLIM-01 in January 2022. The NMPA granted this designation to sovleplenib as a new drug that could treat a serious condition for which there are no effective treatment options, and where clinical evidence demonstrates significant advantages over existing therapies.
“We are pleased to have initiated the rolling submission of an NDA for sovleplenib in China as we look to bring this novel treatment to ITP patients,” said Dr. Weiguo Su, Executive Director, Chief Executive Officer and Chief Scientific Officer of HUTCHMED. “Our submission includes data from the successful Phase III ESLIM-01 trial in China which demonstrated a durable response rate of sovleplenib for patients. There is a significant need for new therapies in adult primary ITP which can significantly impair the quality of life for patients.”
About Sovleplenib
Sovleplenib is a novel, selective inhibitor of Syk for once daily oral administration. Syk is a major component in B-cell receptor and FcR signaling and is an established target for the treatment of multiple subtypes of B-cell lymphomas and autoimmune disorders.
Results from the Phase I/II study in China study published in The Lancet Haematology showed a rapid and durable increase in platelet counts in previously treated patients with ITP. Among the patients who received the recommend Phase II dose of 300mg once daily (“RP2D”),
Sovleplenib is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
HUTCHMED currently retains all rights to sovleplenib worldwide. In addition to ITP, sovleplenib is also being studied in warm antibody autoimmune hemolytic anemia (NCT05535933) and indolent non-Hodgkin’s lymphoma (NCT03779113).
About ITP
ITP is an autoimmune disorder characterized by immunologic destruction of platelets and decreased platelet production. Patients with ITP are at increased risk of excessive bleeding and bruising.1 ITP is also associated with fatigue (reported in up to
Adult ITP is a heterogeneous disease that can persist for years, even with best available care, and treatments are infrequently curative. Despite availability of several treatments with differing mechanisms of action, chronicity of disease continues to be a problem. Many patients develop resistance to treatment and thereby are prone to relapse.9 Thus, there remains a significant population of patients who have limited sensitivity to currently available agents and are in need of new treatments.
As platelet destruction in ITP is mediated by Syk-dependent phagocytosis of FcγR-bound platelets, Syk inhibition represents a promising approach to management of ITP.10
About HUTCHMED
HUTCHMED (Nasdaq/AIM: HCM; HKEX: 13) is an innovative, commercial-stage, biopharmaceutical company. It is committed to the discovery, global development and commercialization of targeted therapies and immunotherapies for the treatment of cancer and immunological diseases. It has approximately 5,000 personnel across all its companies, at the center of which is a team of about 1,800 in oncology/immunology. Since inception, HUTCHMED has focused on bringing cancer drug candidates from in-house discovery to patients around the world, with its first three oncology medicines now approved marketed in China, the first of which is also marketed in the U.S. For more information, please visit: www.hutch-med.com or follow us on LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the U.S. Private Securities Litigation Reform Act of 1995. These forward-looking statements reflect HUTCHMED’s current expectations regarding future events, including its expectations regarding the submission of a NDA for sovleplenib for the treatment of ITP with the NMPA and the timing of such submission, therapeutic potential of sovleplenib for the treatment of patients with ITP and the further development of sovleplenib in this and other indications. Forward-looking statements involve risks and uncertainties. Such risks and uncertainties include, among other things, assumptions regarding the timing and outcome of clinical studies and the sufficiency of clinical data to support NDA approval of sovleplenib for the treatment of patients with ITP or other indications in China or other jurisdictions, its potential to gain approvals from regulatory authorities on an expedited basis or at all, the safety profile of sovleplenib, HUTCHMED’s ability to fund, implement and complete its further clinical development and commercialization plans for sovleplenib, the timing of these events, and the impact of COVID-19 on general economic, regulatory and political conditions. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. For further discussion of these and other risks, see HUTCHMED’s filings with the U.S. Securities and Exchange Commission, The Stock Exchange of Hong Kong Limited and on AIM. HUTCHMED undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.
CONTACTS
| Investor Enquiries | +852 2121 8200 / ir@hutch-med.com |
| Media Enquiries | |
| Ben Atwell / Alex Shaw, FTI Consulting | +44 20 3727 1030 / +44 7771 913 902 (Mobile) / |
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| Zhou Yi, Brunswick | +852 9783 6894 (Mobile) / HUTCHMED@brunswickgroup.com |
| Nominated Advisor | |
| Atholl Tweedie / Freddy Crossley / Daphne Zhang, Panmure Gordon | +44 (20) 7886 2500 |
1 Zufferey A, Kapur R, Semple JW. Pathogenesis and Therapeutic Mechanisms in Immune Thrombocytopenia (ITP). J. Clin. Med. 2017, 6(2), 16.
2 McMillan R, Bussel JB, et al. Self-reported health-related quality of life in adults with chronic immune thrombocytopenic purpura. Am J Hematol. 2008 Feb;83(2):150-4.
3 Snyder CF, Mathias SD, Cella D, et al. Health-related quality of life of immune thrombocytopenic purpura patients: results from a web‑based survey. Curr Med Res Opin. 2008 Oct;24(10):2767-76.
4 Doobaree IU, Nandigam R, Bennett D, et al. Thromboembolism in adults with primary immune thrombocytopenia: a systematic literature review and meta-analysis. Eur J Haematol. 2016 Oct;97(4):321-30.
5 Sarpatwari A, Bennett D, Logie JW, et al. Thromboembolic events among adult patients with primary immune thrombocytopenia in the United Kingdom General Practice Research Database. Haematologica. 2010 Jul;95(7):1167-75.
6 Sarpatwari A, Watson S, Erqou S, et al. Health-related lifestyle in adults and children with primary immune thrombocytopenia (ITP). Br J Haematol. 2010 Oct;151(2):189-91.
7 Lambert MP, Gernsheimer TB. Clinical updates in adult immune thrombocytopenia. Blood. 2017 May 25;129(21):2829-2835.
8 Clarivate Landscape & Forecast for Immune Thrombocytopenic Purpura, 2018.
9 Provan D, Arnold DM, Bussel JB, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019;3(22):3780-3817.
10 Crowley MT, Costello PS, Fitzer-Attas CJ et al. A critical role for Syk in signal transduction and phagocytosis mediated by Fcγ receptors on macrophages. J. Exp. Med. 186(7), 1027–1039 (1997).
FAQ
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