Drug Database
SI

sirolimus (NPC 12G / Hyftor / rapalimus)

✓ Approved

Nobelpharma Co., Ltd. · MTOR

What is sirolimus?

sirolimus is a therapeutic agent developed by Nobelpharma Co., Ltd.. It is approved for therapeutic indications via topical.

Drug Profile

Brand NamesNPC 12G, Hyftor, rapalimus
CompanyNobelpharma Co., Ltd.
Molecular TargetMTOR
RouteTopical
StatusApproved
Sources: ClinicalTrials.gov, Nobelpharma Co., Ltd.

Mechanism of Action

Molecular Targets

sirolimus acts on 1 molecular target:

MTORmechanistic target of rapamycin kinase (FRAP2, RAPT1)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

sirolimus is developed for 4 unique indications across 3 therapeutic areas.

Therapeutic AreaConditionPhase
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

Related Research Articles

PubMedJournal of paediatrics and child health2026-06-05

Management of Kaposiform Haemangioendothelioma-A Retrospective Case Series Examining the Use and Effectiveness of Vincristine.

Kirk Bronwen B, Adams Susan S, Wargon Orli O, Tatian Artiene A et al.

Kaposiform haemangioendothelioma is a rare vascular tumour. Management has evolved over the last two decades, with most cases receiving vincristine or sirolimus. We aim to describe our experience with managing this tumour, with a focus on the safety and efficacy of vincristine. A retrospective cohort study of all cases treated at Sydney Children's Hospital over a 19-year period was conducted. Explanatory variables included age, tumour location, imaging, the presence of Kasabach-Merrit Phenomenon, and treatment modality. The primary outcome was disease recurrence and the secondary outcome, complications. Twenty-one cases were identified with 13 (60%) presenting as neonates. The most common site was limb; and imaging included ultrasound (7/21, 33%) and MRI (12/21, 57%). Almost all had consumptive coagulopathy (20/21, 95%) with 6/21 (29%) having severe Kasabach-Merrit Phenomenon. Six tumours were excised and 16 (76%) were treated with vincristine, with three (14%) progressing to treatment with sirolimus. There were no recurrences. Complications of vincristine therapy were common but mild in most cases, with only one proven blood stream infection and one requiring central line replacement. Vincristine remains a highly effective first-line treatment for kaposiform haemangioendothelioma with an acceptable safety profile. Further well-designed multicentre trials are needed to objectively compare its effectiveness and safety with sirolimus.

PMID 42246503
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PubMedNature medicine2026-06-05

AAV gene therapy for homozygous familial hypercholesterolemia: a phase 1 trial.

Zheng Tao T, Gao Ge G, Xu Chenbo C, Li Ruifeng R et al.

Homozygous familial hypercholesterolemia (HoFH) is a rare autosomal disease characterized by severely elevated low-density lipoprotein cholesterol (LDL-C) levels and accelerated atherosclerotic cardiovascular disease. More than 80% of patients with HoFH carry low-density lipoprotein receptor (LDLR) mutations. Here we developed an adeno-associated virus gene therapy designed to express LDLR in the liver and evaluated its safety and efficacy in lowering LDL-C levels in experimental animals and in patients with HoFH. NGGT006, a recombinant adeno-associated virus serotype 8-based vector containing a codon-optimized LDLR complementary DNA, lowered LDL-C levels in Ldlr-/- mice and Ldlr-/- hamsters and decreased aortic plaque size in Ldlr-/- mice. In rhesus monkeys, NGGT006 administration led to transient liver enzyme elevations but no severe adverse events. In an open-label, single-arm, dose-escalation trial, three patients with HoFH received doses of NGGT006 of 7.5 × 1012, 1.5 × 1013 and 3 × 1013 vg kg-1. Primary endpoints included safety and LDL-C reduction over a follow-up of 52 weeks. NGGT006 treatment was well tolerated, with no vector-related severe adverse events. All three patients showed elevations of liver enzymes that were resolved after sirolimus and methylprednisolone therapy. The patient who received the highest dose exhibited a sustained reduction in LDL-C from 11 mmol l-1 to <1.8 mmol l-1 starting at 3 weeks after treatment. These results offer initial insights into the safety and therapeutic potential of NGGT006 and warrant future studies of its safety and efficacy. ClinicalTrials.gov: NCT06125847 .

PMID 42243546
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PubMedIranian journal of kidney diseases2026-06-03

Distribution of CYP3A4*1/*1B (rs2740574) and CYP3A5*1/*3 (rs776746) Genetic Variants in Renal Transplant Recipients Treated with Sirolimus in Urmia (Iran).

Makhdoomi Khadijeh K, Mivefroshan Azam A, Jafari Lale L, Ghelich Khan Zahra Z et al.

Introduction. Rapamycin, also referred to as Sirolimus, is an immunosuppressive medication used in kidney transplantation to prevent organ rejection. Sirolimus is metabolized predominantly by the enzymes CYP3A4 and CYP3A5. The objective of this study was to evaluate the distribution of CYP3A4*1/*1B (rs2740574, -392A > G) and CYP3A5*1/*3 (rs776746, 6986A > G) genetic variants in renal transplant recipients treated with Sirolimus in Urmia (Iran). Methods. This assessment involved thirty-nine renal transplant recipients in Urmia Imam Khomeini University Hospital, Urmia (Iran) who were treated with a daily dose of 1mg/day Sirolimus. The CYP3A4*1/*1B and CYP3A5*1/*3 allelic variants were identified using the Restriction Fragment Length Polymorphism-Polymerase Chain Reaction (RFLP-PCR) technique.  Results. The frequencies of CYP3A4 *1/*1 (-392AA), CYP3A4 *1/*1B (-392AG), and CYP3A4 *1B/*1B (-392GG) genotypes and CYP3A4 *1 (-392A) and CYP3A4 *1B (-392G) alleles were 37(94.87%), 2(5.13%), 0(0%), 76(97%), and 2(3%), respectively. The genotypic distribution for CYP3A5 was 0(0%) for CYP3A5*1/*1 (6986AA), 6(15.38%) for CYP3A5*1/*3 (6986AG), and 33(84.62%) for CYP3A5*3/*3 (6986GG). The allelic frequencies were 6(8%) for CYP3A5*1 (6986A) and 72(92%) for CYP3A5*3 (6986G). Only one patient with CYP3A4.

PMID 42234923
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PubMedFrontiers in oncology2026-06-03

Case report: superficial kaposiform hemangioendothelioma without the Kasabach-Merritt phenomenon.

Chen Yeli Y, Huang Ziyu Z, Huang Feifei F, Chen Jianna J et al.

Kaposiform hemangioendothelioma (KHE) is a rare intermediate-grade malignant vascular tumor that is frequently associated with the Kasabach-Merritt phenomenon (KMP). Cases without KMP are less common and, therefore, prone to misdiagnosis. We report a case of superficial KHE in a 6-month-old male infant without associated KMP. Magnetic resonance imaging revealed a poorly demarcated lesion with T1 hypointensity and T2 hyperintensity, demonstrating marked enhancement and an arterial flow void. The patient was hospitalized because KHE was suspected and received a single course of intralesional pingyangmycin therapy. One week later, histopathological examination showed proliferating spindle-shaped endothelial cells in a glomeruloid pattern. The immunohistochemical results revealed CD31 and CD34 positivity and GLUT1 and D2-40 negativity. GLUT1 negativity helped exclude infantile hemangioma, whereas D2-40 negativity ruled out lymphatic malformation. Upon confirming KHE diagnosis, we initiated a combined sirolimus and prednisone treatment. At the 3-month follow-up, clinical observation showed regression of the lesion, prompting prednisone discontinuation while continuing sirolimus maintenance therapy. In this report, we also discuss the clinical manifestations, imaging features, histopathological characteristics, and treatment options for this KHE subtype and review relevant literature. This case highlights the diagnostic features and therapeutic considerations of superficial KHE without KMP, emphasizing the importance of multidisciplinary evaluation.

PMID 42232551
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PubMedFrontiers in cardiovascular medicine2026-06-03

Drug-coated balloons vs. drug-eluting stents for coronary artery disease: an updated systematic review and meta-analysis of randomized controlled trials with lesion-specific insights.

Wang Yuting Y, Shi Yaqi Y, Wang Qiong Q, Yang Shengxiang S et al.

To compare the efficacy and safety of drug-coated balloons (DCB) vs. drug-eluting stents (DES) in patients with coronary artery disease (CAD) undergoing percutaneous coronary intervention, with a focus on lesion-specific and presentation-specific outcomes. We systematically searched PubMed, Embase, CENTRAL, and Web of Science for randomized controlled trials (RCTs) comparing DCB with DES from inception to March 2026. Outcomes included major adverse cardiac events (MACEs), target lesion revascularization (TLR), device-oriented composite endpoint (DoCE), patient-oriented composite endpoint (PoCE), cardiac death, myocardial infarction (MI), all-cause death, thrombosis (definite/probable), and angiographic endpoints. Pooled odds ratios (ORs) or mean differences (MDs) were calculated using random-effects models. Subgroup analyses were performed by lesion type [de novo vs. in-stent restenosis (ISR)], vessel size, clinical presentation (STEMI vs. NSTEMI vs. unstable angina), and DCB type (paclitaxel vs. sirolimus). Twenty-three RCTs comprising 8,123 patients were included. DCB was associated with significantly higher risks of TLR (OR 2.22, 95% CI 1.49-3.33), DoCE (OR 1.86, 95% CI 1.49-2.31), PoCE (OR 1.43, 95% CI 1.20-1.72), and cardiac death (OR 1.53, 95% CI 1.11-2.10) compared with DES. No significant differences were observed for MACEs, MI, all-cause death, or thrombosis. In the critical subgroup analysis, for ISR, DES was superior to DCB (OR for TLR with DCB vs. DES: 3.54, 95% CI 2.05-6.09), whereas for de novo lesions, DCB was associated with a higher risk of TLR compared to DES (OR 1.76, 95% CI 1.03-3.02). In small vessel disease, TLR did not differ significantly between the two strategies (OR 1.17, 95% CI 0.64-2.14). The increased risk of cardiac death with DCB was observed only in trials using paclitaxel-coated balloons (OR 1.63, 95% CI 1.17-2.28), while no signal was seen with sirolimus-coated balloons (OR 0.96, 95% CI 0.19-4.81). However, this finding is exploratory, derived from post-hoc subgroup analyses with limited events and shorter follow-up in sirolimus studies, and should be interpreted with caution. Exploratory analysis by clinical presentation showed no significant interaction between treatment effect and STEMI, NSTEMI, or unstable angina (P-interaction = 0.34 for MACEs). The comparative effectiveness of DCB vs. DES is lesion-specific. For ISR, DES remains the superior treatment. DCB represents a viable alternative to DES in de novo small vessel disease. However, in de novo lesions of non-small vessels, DES remains superior. The increased cardiac death signal appears to be driven by paclitaxel-coated balloons and warrants further investigation. Clinical presentation (STEMI/NSTEMI/unstable angina) did not modify the relative treatment effect, but these analyses were exploratory and limited by sample size. https://www.crd.york.ac.uk/PROSPERO/view/CRD420261355942, PROSPERO CRD420261355942.

PMID 42232313
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PubMedJournal of visualized surgery2026-06-03

Surgical strategies for spontaneous pneumothorax: a narrative review.

Tsuboshima Kenji K

Spontaneous pneumothorax encompasses both primary spontaneous pneumothorax (PSP) and secondary spontaneous pneumothorax (SSP), which differ in etiology and recurrence risk. Surgical management aims to control air leaks and prevent recurrence while minimizing invasiveness. This narrative review synthesizes current strategies, adjunctive methods, and special scenarios, and outlines future perspectives. A narrative search of PubMed and major thoracic surgery journals was conducted for English-language publications published between January 1991 and January 2026, focusing on surgical indications; minimally invasive approaches [multiportal and uniportal video-assisted thoracoscopic surgery (VATS), and awake VATS]; adjuncts to reduce recurrence; and emerging technologies. For SSP, the scope was limited to lymphangioleiomyomatosis (LAM), Birt-Hogg-Dubé syndrome (BHDS), and thoracic endometriosis-related pneumothorax (TERP). Key clinical guidelines and pivotal studies were prioritized. For PSP, stapled bullectomy remains the standard approach and is frequently combined with pleural reinforcement. Staple-line coverage with absorbable sheets has been associated with lower recurrence rates. Mechanical pleurodesis provides recurrence control comparable to pleurectomy in selected cases, whereas pleurectomy carries higher morbidity. Similarly, chemical pleurodesis has shown benefit as an adjunctive strategy. SSP requires individualized surgical planning based on the underlying disease; awake VATS may be considered for frail patients when feasible. In LAM and BHDS, total pleural covering has shown favorable outcomes, and sirolimus may reduce recurrence after surgery in LAM. Robotic-assisted thoracoscopic surgery (RATS) demonstrates ergonomic and precision advantages in thoracic surgery, but its role in spontaneous pneumothorax remains to be defined. Artificial intelligence (AI)-based imaging remains investigational without established surgical applications. Contemporary surgery for spontaneous pneumothorax is defined by minimally invasive techniques supported by recurrence-preventive adjuncts tailored to patient characteristics. Evidence supports coverage techniques and selective chemical or mechanical pleurodesis, whereas disease-specific strategies remain essential for LAM, BHDS, and TERP.

PMID 42232039
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