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sirolimus (NPC 12G / Hyftor / rapalimus)

✓ Approved

Nobelpharma Co., Ltd. · MTOR

什么是 sirolimus？

sirolimus 是一种治疗药物，由Nobelpharma Co., Ltd.研发。该药已获批，用于治疗相关适应症，给药途径：Topical。

药物档案

商品名NPC 12G, Hyftor, rapalimus
公司Nobelpharma Co., Ltd.
分子靶点MTOR
给药途径Topical
状态Approved
来源： ClinicalTrials.gov, Nobelpharma Co., Ltd.

作用机制

分子靶点

sirolimus 作用于 1 个分子靶点：

MTORmechanistic target of rapamycin kinase (FRAP2, RAPT1)
需要更深入的分析？Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

sirolimus 针对 4 个适应症，涉及 3 个治疗领域。

治疗领域疾病/病症分期
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Angiofibroma✓ Approved
Respiratory, thoracic and mediastinal disordersLymphangioleiomyomatosis✓ Approved
Congenital, familial and genetic disordersLymphatic malformation✓ Approved
Congenital, familial and genetic disordersNeurofibromatosisPhase III

相关研究文献

PubMedHellenic journal of cardiology : HJC = Hellenike kardiologike epitheorese2026-06-13

Clinical Efficacy and Safety of Paclitaxel-coated vs. Limus-coated Balloons in Coronary Interventions: A Review of the literature.

Tsiamis Nikolaos N, Afendoulis Dimitrios D, Papanikolaou Aggelos A, Tsakirian Flora F et al.

The principle of complete vessel restoration in percutaneous coronary intervention (PCI) has highlighted the significance of drug-coated balloons (DCBs) as a crucial substitute for enduring metallic stents, associated with potential hazards like neoatherosclerosis and delayed stent thrombosis. Although paclitaxel-coated balloons (PCBs) are widely accepted as the preferred option for managing in-stent restenosis (ISR) due to their lipophilic and cytotoxic characteristics, sirolimus-coated balloons (SCBs) have surfaced as a potentially less risky alternative, exploiting a cytostatic mode of action. This review synthesizes clinical evidence from 10 randomized controlled trials (RCTs) and 7 meta-analyses published between 2020 and 2025. The analysis focuses on comparative safety and efficacy across major indications: in-stent restenosis (ISR), de novo small vessel disease (SVD), and bifurcation lesions. • In-Stent Restenosis (ISR): Multiple trials (e.g., Scheller et al. 2022, BIO ASCEND ISR) demonstrated the non-inferiority of limus-based platforms compared to PCBs. For instance, late lumen loss (LLL) was nearly identical between groups (0.25 mm for PCB vs. 0.26 mm for SCB). However, the REFORM trial failed to show non-inferiority for a biolimus-coated balloon, highlighting that outcomes are often device-specific rather than a class effect. • De Novo Small Vessel Disease: Outcomes in this category were more heterogeneous. In the TRANSFORM I trial, the MagicTouch SCB failed to meet non-inferiority for net lumen gain compared to the SeQuent Please Neo PCB. Conversely, other studies observed comparable LLL between the two platforms. • Safety Profile: Across most indications, Major Adverse Cardiac Events (MACE) and Target Lesion Failure (TLF) rates were comparable between paclitaxel and sirolimus platforms at 12-month follow-up. PCBs demonstrated a higher frequency of "late lumen enlargement" compared to SCBs. Both paclitaxel and sirolimus-based DCBs are effective for treating in-stent restenosis. However, in de novo lesions, PCBs currently maintain a more consistent evidence base. Clinical performance appears heavily dependent on device-specific factors such as coating technology and excipient formulation rather than a general class effect. Extended follow-up data (3-5 years) are still required to fully evaluate long-term safety and the risk of very late thrombosis.

PMID 42285438
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PubMedOrphanet journal of rare diseases2026-06-13

In vitro study of TSC1 deficiency in preadipocytes: insights into development and treatment options for tuberous sclerosis related lipomatosis.

Friedrich Julika E JE, Hentschel Julia J, Richter Sandy S, Kiep Henriette H et al.

Tuberous sclerosis complex (TSC) is a rare genetic neurocutaneous disorder resulting from mutations in the TSC1 or TSC2 genes, characterized by overgrowth and lesions in multiple organs. While renal angiomyolipomas are commonly seen, lipomas located elsewhere are rarely reported in these patients. We identified a heterozygous TSC1 mutation in a pediatric patient, who developed a lipoma in the gluteal region, which recurred after surgical resection. We observed a loss of heterozygosity in the lipoma tissue, resulting in TSC1 deficiency and subsequent activation of the mechanistic target of rapamycin (mTOR) signaling pathway. Further in vitro experiments showed that silencing TSC1 in adipocyte progenitors led to increased cell proliferation, supporting the hypothesis that TSC1 deficiency contributes to lipoma formation. Treatment with mTOR inhibitors, such as sirolimus and torin-1, as well as the phosphoinositide 3-kinase (PI3K) inhibitor alpelisib reduced cell proliferation and pathway activation in TSC1-deficient cells. This study highlights the need for further investigation into the efficacy of pathway inhibitors in managing TSC-related lipomas in vivo and offers a potential treatment avenue for patients suffering from recurrent lipomatosis.

PMID 42286631
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PubMedWorld journal of transplantation2026-06-12

Gemcitabine-sirolimus synergy associated thrombotic microangiopathy in a renal transplant recipient with mesenteric leiomyosarcoma: A case report.

Motla Vishaal V, Baig Usman U, Mulloy Laura L, Nakkar Talal T et al.

Mesenteric leiomyosarcoma, a rare tumor originating from the smooth muscle of the mesentery, that may require treatment with gemcitabine based chemotherapy in advanced stages. Gemcitabine usage has been associated with the development of thrombotic microangiopathy, a risk which can be increased by concurrent use of sirolimus. We present the case of a young Caucasian woman with a renal transplant who developed an aggressive leiomyosarcoma. While on gemcitabine, she was noted to have acute kidney injury. Biopsy of the renal allograft revealed histological findings consistent with thrombotic microangiopathy. Sirolimus was replaced with tacrolimus, and gemcitabine was discontinued. Despite treatment with eculizumab, the allograft function continued to decline, leading to graft failure, and the patient had to be started on renal replacement therapy. This case highlights the possible synergy of gemcitabine and sirolimus in the pathogenesis of de novo thrombotic microangiopathy.

PMID 42281866
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PubMedPolish archives of internal medicine2026-06-12

Long-term outcomes of sirolimus therapy in sporadic and tuberous sclerosis complex-associated lymphangioleiomyomatosis: a retrospective cohort study.

Nowacka-Ejsmont Joanna J, Bączek Karol K, Bestry Iwona I, Błasinska Katarzyna K et al.

Sirolimus is the standard disease-modifying therapy for lymphangioleiomyomatosis (LAM), but long-term routine-care data integrating pulmonary, lymphatic, extrapulmonary, and biomarker outcomes remain limited. To assess long-term effectiveness and safety of sirolimus in routine clinical practice. We retrospectively analyzed consecutive adults with definite LAM treated with sirolimus at a national tertiary referral center in Poland between 2010 and 2020. Seventy-one patients were included (70 women; 57/71 [80%] with sporadic LAM and 14/71 [20%] with TSC-associated disease). The median duration of available functional follow-up was 5.0 years [IQR, 2.0-5.0], and mean trough sirolimus concentration was 7.85 (2.36) ng/mL. Baseline chylous pleural and/or peritoneal effusions were present in 15/71 (21%), renal angiomyolipomas in 33/71 (46%), and lymphangioleiomyomas in 28/71 (39%) patients. FEV1 increased at 12 months by a median Δ of 0.03 L [IQR, -0.10 to 0.30] from baseline (n=66; P=0.03). FVC and 6-minute walk distance also improved during early follow-up, while TLCO remained generally stable. Chylous effusions resolved in all affected patients by 12 months without recurrence, and renal angiomyolipoma and lymphangioleiomyoma volumes decreased significantly in patients with paired MRI measurements. Higher baseline VEGF-D was associated with lymphatic involvement, and VEGF-D decreased during treatment. Adverse events were mostly mild; permanent discontinuation occurred in approximately 6%. In routine practice, sirolimus was associated with long-term stabilization of lung function, marked improvement of lymphatic disease, reduction of angiomyolipoma burden, and acceptable tolerability. VEGF-D aligned with lymphatic phenotype and declined with treatment, supporting its role as a monitoring biomarker.

PMID 42284501
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PubMedInternational journal of molecular sciences2026-06-12

Electrospun Carbothane-Based Drug-Enriched Scaffolds for Cardiovascular Devices: Drug Release, Hemocompatibility, Endothelialization, and Immunological Characterization.

Nazarkina Zhanna K ZK, Chelobanov Boris P BP, Stepanova Alena O AO, Imenov Aznaur A et al.

Polyurethane (PU) is widely used in medical products due to its biocompatibility and mechanical properties. Electrospinning (ES) was employed to produce PU-based scaffolds intended for cardiovascular devices (CVD) from blends of Carbothane (Carb) with human serum albumin (HSA), dimethylacetamide (DMA), and drugs. Sirolimus (SRL)-an immunosuppressive/anti-proliferative drug-and diclofenac (DF)-a nonsteroidal anti-inflammatory drug-were introduced into ES blends to produce drug-enriched scaffolds that prevent inflammation and cell overgrowth. The biocompatibility, stability, and mechanical properties of the scaffolds and SRL release were studied. The scaffolds possessed good mechanical properties and were stable in PBS and blood plasma (BP) for 120 days. The minimal SRL release rate was observed for the scaffold 3%Carb/10%HSA/DMA/SRL. A study of scaffold interaction with blood demonstrated good hemocompatibility of most scaffolds. A study of human gingival fibroblasts, endothelial cells (HUVEC and EA.hy926), and vascular smooth muscle cell interaction with scaffolds in vitro demonstrated variability in cell viability and pro-inflammatory interleukin IL-6 secretion, depending on both the scaffold composition and the cell type. The incorporation of DF into scaffolds decreased the concentration of IL-6 in the culture medium. The scaffold 3%Carb/10%HSA/DMA/SRL is the best choice for CVD in terms of hemocompatibility, endothelialization, and the induction of minimal inflammation.

PMID 42278604
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PubMedCancer reports (Hoboken, N.J.)2026-06-11

Systemic Treatment of Advanced Chordoma With Molecular Targeted Therapies: A Large Multicentre European Retrospective Case Series.

Lipplaa Astrid A, Hindi Nadia N, Teterycz Pawel P, Young Robin J RJ et al.

This retrospective series reports outcomes and prognostic factors for advanced chordoma (AC) treatment with molecular targeted therapies (MTTs) in different treatment lines. This is a retrospective series of 57 patients with AC treated between 2004 and 2023 at one of seven participating sarcoma centres. Demographics, previous treatment, treatment details and outcomes were recorded. 57 patients were treated with 7 different MTTs. Treatment was received in first (n = 57), second (n = 16), third (n = 5) or fourth (n = 1) line. The most frequently administered agent in first line was imatinib (84.2%) and in second line imatinib + sirolimus (35.7%). Overall median progression free survival (PFS) and overall survival (OS) in first-line treatment was 6.5 (95% CI 4.0-9.0) and 29.5 months (95% CI 24.0-40.4) and in second-line 10.0 (95% CI 4.0-22.0) and 37.2 months (95% CI 9.4-45.9). Partial response according to RECIST 1.1 was seen in 5/79 treatments (6.3%). Dose reductions and interruptions were reported in 19.0% and 27.8% of treatments. PFS and response rates with these MTTs were in line with previous phase II trials and retrospective series. Although the efficacy does not meet the European Society of Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale (MCBS) criteria for single arm studies in orphan diseases, MTTs are frequently used off-label due to the high unmet need and lack of other systemic treatment options. The toxicity profile and limited efficacy rate should be taken into account when counselling patients. Further research is needed to explore other systemic treatment options including (combinations with) immunotherapy.

PMID 42274206
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