(May 29, 2025, Shanghai, China）— On 29 May, 2025, Shanghai Fosun Pharmaceutical (Group) Co., Ltd. (“Fosun Pharma”; Stock Code: 600196.SH, 02196.HK), announced that its self-developed innovative drug Luvometinib Tablets (Chinese trade name:复迈宁^®, project no.: FCN-159, the “New Drug”) has been officially approved for marketing by the National Medical Products Administration (NMPA). As a highly selective MEK1/2 inhibitor, the approved indication is the treatment of adult patients with Langerhans cell histiocytosis (LCH) and histiocytic neoplasms and the treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. The approval of the New Drug addresses unmet treatment needs in rare tumors and provides new treatment options for patients.

Xingli WANG, ExecutivePresident and CEO of Global R&D Center of Fosun Pharma said:“The approval of Luvometinib Tablets is an important milestone for Fosun Pharma's deep dive into the oncology and rare disease fields. As an innovation-driven global healthcare group, we are committed to addressing unmet medical needs, accelerating rare disease drug development, filling treatment gaps in related diseases and improving access to innovative therapies for patients with rare conditions. looking forward, Fosun Pharma will continue to advance the development of the New Drug for additional indications to benefit more patients.”

The New Drug is the first and only drug approved in China for both adult LCH/histiocytic neoplasms and NF1-associated PN in children aged two and above. As an innovative small molecule chemical drug self-developed by Fosun Pharma (i.e., the Company and its subsidiaries/units, the same applies below), the New Drug works by selectively inhibiting MEK1/2, blocking abnormal activation of the MAPK pathway, suppressing tumor cell growth, and inducing apoptosis.^[1],[2],[3]Based on clinical data, the New Drug demonstrates proven efficacy, good safety, and faster onset than current therapies, providing a valuable new option for clinical use. Abnormal MAPK activation is commonin many solid tumors and rare diseases, such as adult histiocytic neoplasms and NF1-associated PN in children. The semutations are considered key drivers of disease onset and progression.^[4] Compared to traditional chemotherapy and surgery, MEK-targeted therapies offer greater precision and manageable side effects, providing patients with a more effective treatment option^[5].

In addition, the New Drug is at the stage of Phase III clinical trial in China (excluding Hong Kong, Macao and Taiwan regions for the purpose of this announcement, the same applies below) for the treatment of adults with neurofibromatosis type 1, and the New Drug is at the stage of Phase II clinical trial in China for the treatment of low-grade glioma, extracranial arteriovenous malformations, children with Langerhans cell histiocytosis. The New Drug has been granted breakthrough therapy designation by the Center for Drug Evaluation of the NMPA for the treatment of two indications of adults with inoperable or post-operative residual/recurrent NF1-associated PNs and children with Langerhans cell histiocytosis.

According to the latest data from IQVIA MIDAS™^[6], the sales of MEK1/2 selective inhibitor worldwide amounted to approximately US$2,068 million in 2024. As of the end of 2024, only several MEK1/2 inhibitors had been approved worldwide^[7]. In China, no drugs have been approved to date for adult LCH/histiocytic neoplasms. Therefore, the approval of the New Drug offers new treatment hope for patients affected by these conditions.

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About Adult LCH and Histiocytic Neoplasms

LCH and Erdheim-Chester disease (ECD) are rare hematologic cancers, with over 80% of patients experiencing multisystem involvement.^[8],[9],[10]Symptoms may include bone pain, skin lesions, heart conditions (like heart failure, infarction, pericarditis), hypoalbuminemia, diabetes insipidus, exophthalmos, dyspnea, and liver failure. Patients often face poor prognosis and limited treatment options. Traditional therapies, such as surgery, chemo-radiation, bone marrow transplant, have limited effects. In a Phase II clinical trial, the New Drug demonstrated a confirmed objective response rate (ORR) of 82.8%, as assessed by an independent review committee (IRC) using PET Response Criteria (PRC). The complete metabolic response (CMR) rate was 62.1%, with a 2-year duration of response (DOR) rate of 91.1%. The median time to response was only 2.9 months, significantly improving patient outcomes.^[11]

About NF1-associated PN in children

Neurofibromatosis type 1 (NF1) is an autosomal dominant genetic disorder with a clear familial inheritance pattern. It is the most common form of neurofibromatosis (NF), accounting for 96% of the rare NF cases, and presents with a wide range of clinical symptoms affecting the skin, eyes, and nervous system. Plexiform neurofibromas (PN) are a common manifestation of NF1, occurring in 30-50% of patients.^[12] These tumors grow rapidly during childhood, often leading to serious complications, and a high rate of recurrence due to incomplete surgical removal. There is an urgent need for effective treatment alternatives. Studies have shown that inhibiting MEK activity can effectively suppress neoplasm growth and proliferation, making it a promising target for therapy.1-3In a Phase II clinical trial of the New Drug, with a median follow-up of 25.1 months, the confirmed ORR assessed by investigators based on REiNS criteria was 60.5%, with a 1-year DOR rate of 87.6%. With a faster onset of action, the New Drug achieved a median time to remission of just 4.7 months. It effectively relieved tumor-related pain and showed good tolerability, offering a non-invasive targeted treatment option for pediatric patients.