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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

PubMedEuropean journal of pharmaceutics and biopharmaceutics : official journal of Arbeitsgemeinschaft fur Pharmazeutische Verfahrenstechnik e.V2026-09-19

Micro-modulation of the intra-structural environment in electrospun drug delivery systems: effects on drug release and hydrolytic degradation.

Wlodarczyk Jakub J, Kurowska Natalia N, Musial-Kulik Monika M, Hajdas Anastazja A et al.

Poor aqueous solubility can limit drug release from biodegradable implants. We developed a multifunctional dual-jet electrospun drug-delivery system (DDS) designed to accelerate the release of poorly water-soluble bioactive substances. Its principle is based on introducing the drug-carrying matrix and release-rate modifier as separate, interlaced fractions. Poly(D,L-lactide-co-glycolide) (PDLGA) nonwovens or poly(ε-caprolactone-co-trimethylene carbonate) (PCLTMC) membranes served as sirolimus carriers, whereas poly(vinyl alcohol) nanofibres loaded with lactic acid (PVA(LA)) were designed to increase incubation-medium accessibility, lower the pH of the aqueous environment and catalyse early-stage matrix hydrolysis. Both copolymers had defined chain microstructures with relatively short comonomer sequences, intended to limit changes in physical properties and molar composition during incubation. Drug release and early-stage hydrolytic degradation were evaluated over 28 days in deionised water and phosphate-buffered saline. Dissolution of the PVA(LA) fraction increased incubation-medium uptake and was associated with accelerated sirolimus release in water. After 28 days, cumulative release increased from 12.76 to 29.32% for PDLGA and from 22.03 to 59.03% for PCLTMC. LA release reduced bulk-medium pH to 3.14-3.21 after one day. Modest hydrolytic changes were detected for PDLGA, whereas the effect on PCLTMC was limited. Both carriers remained amorphous, showed only small changes in glass-transition temperature (Tg) and retained stable molar compositions. Release was diffusion-dominated, with possible contributions from medium uptake and structural reorganisation. This approach integrates a biodegradable drug carrier with an interlaced, dissolving modifier fraction, allowing the release environment to be modified and drug release to be accelerated without altering the chemical composition of the matrix.

PMID 42762970
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PubMedFrontiers in pediatrics2026-09-18

Transcatheter embolization as a rescue therapy for refractory pelvic kaposiform hemangioendothelioma with Kasabachi-Merritt phenomenon: a case report.

Xiong Yi Y, Di Qi Q, Li Sanlin S, Cao Jiajie J et al.

Kasabach-Merritt phenomenon (KMP) is a life-threatening consumptive coagulopathy in children with kaposiform hemangioendothelioma (KHE) or tufted angioma. Until now, there has been no well-accepted treatment regimen in the literature. A pelvic KHE is very rare and its deep anatomical location and invasive growth pattern toward adjacent vital organs pose significant management challenges. Here, we report a 10-month-old girl presenting with KMP secondary to a pelvic KHE. Initial systemic therapy with sirolimus and corticosteroids failed to control her KMP. Transcatheter arterial embolization was performed using polyvinyl alcohol particles and coils. After embolization, the tumor's blood supply decreased significantly and the patient's platelet counts and coagulation markers returned to normal in 3 days. At the 2-year follow-up, her coagulation function remained normal. The perineal cutaneous rash resolved and the tumor's volume decreased substantially. This case demonstrates that transarterial embolization can be an effective therapeutic choice when systemic treatment has failed.

PMID 42756060
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PubMedCase reports in pediatrics2026-09-17

Successful Early Therapy With Sirolimus for an Infant With Primary Intestinal Lymphangiectasia.

Yamamoto Shungo S, Sugino Yoshihiko Y, Yamashita Takahiro T, Honda Kei K et al.

Identifying the cause of protein-losing enteropathy (PLE) is crucial for appropriate treatment. Primary intestinal lymphangiectasia (PIL) is a cause of PLE, and low-strict fat diet with medium-chain triglyceride (MCT) supplementation may be insufficient. Sirolimus has shown efficacy in refractory PIL; however, its use in infants remains uncommon, and the optimal timing of initiation has not been established. A previously healthy 7-month-old Japanese male infant presented with convulsions, generalized edema, and diarrhea. Laboratory findings revealed severe hypoproteinemia (serum albumin: 1.3 g/dL), lymphopenia (absolute lymphocyte count: 822/μL), and electrolyte abnormalities. Scintigraphy using 99mTc-labeled human serum albumin showed tracer exudate in the small intestine, leading to the diagnosis of PLE. Upper endoscopy revealed snowflake-like white spots in the duodenum, and pathological examination confirmed lymphatic dilation, leading to a PIL diagnosis. Despite treatment with a low-strict fat diet with MCT supplementation and corticosteroids, hypoproteinemia persisted. Lymphatic scintigraphy and magnetic resonance lymphangiography confirmed ongoing protein leakage. Sirolimus was initiated at 0.5 mg/day on Day 23. Subsequently, serum albumin began to increase within 1-2 weeks and ultimately reached 4.1 g/dL, and absolute lymphocyte count, 1917/μL immediately before sirolimus initiation, increased to 3843/μL. Lymphatic scintigraphy on Day 74 of sirolimus treatment showed complete resolution of protein leakage. Sirolimus was continued for 121 days and discontinued thereafter. At 17 months of follow-up, no recurrence was observed, with sustained normalization of serum albumin to 4.0 g/dL and absolute lymphocyte count to 4739/μL. This case suggests one of the earliest reported responses to sirolimus in the treatment of PIL, with improvement observed within a few weeks of initiation. Early introduction of sirolimus in patients with persistent protein leakage resistant to low-strict fat diet with MCT supplementation may be associated with rapid improvement in PIL, though this hypothesis warrants further case accumulation.

PMID 42751049
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PubMedWorld journal of pediatrics : WJP2026-09-17

Safety of sirolimus in pediatric vascular anomalies: a BKMR-PopPK exposure-response framework for individualized risk and dosing.

Liu Bo B, Xu Xiao-Lin XL, Wang Jia-Lu JL, Li Jia J et al.

Vascular anomalies are rare, heterogeneous disorders that threaten the health of children. Sirolimus is an important drug therapy, but its high incidence of adverse drug reactions (ADRs) may cause treatment discontinuation and therefore poor disease control. Although prior studies suggest a strong link between sirolimus exposure and ADRs, the quantitative relationship remains unclear. This study aimed to systematically quantify the relationships between steady-state trough sirolimus concentrations (Cmin) and three ADRs: myelosuppression, abnormal liver function, and dyslipidemia. We retrospectively analyzed the data using Bayesian kernel machine regression (BKMR) to characterize associations of Cmin and its determinants with ADRs, and further developed an integrated BKMR-population pharmacokinetic (PopPK) framework for quantitative risk assessment and prediction. A total of 257 patients were involved in this study (766-892 blood samples). BKMR results showed the quantitative exposure-response relationships between Cmin and ADRs, identifying disease complexity and duration of treatment as key risk modifiers. We recommend routine Cmin monitoring within safety windows: 5.0-7.5 ng/mL for simple lesions or milder disease; 8.0-10.2 ng/mL for complex or mixed vascular malformations. Stricter ADR surveillance is warranted in complex malformations, and during long-term therapy (> 6 months), vigilance for myelosuppression and dyslipidemia through regular screening is also warranted. This is the first study to quantify the relationship between Cmin and ADRs in pediatric vascular anomalies. The integrated BKMR-PopPK framework provides a tool for translating pharmacological insights into practice and advancing model-informed precision dosing (MIPD). These findings support therapeutic drug monitoring (TDM)-based target ranges, addressing a knowledge gap and reducing reliance on empirical dosing. This paradigm also informs safety evaluation and early-phase trials in other pediatric rare diseases with limited samples.

PMID 42752806
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PubMedJournal of endovascular therapy : an official journal of the International Society of Endovascular Specialists2026-09-17

Prospective Sirolimus Coated Angioplasty for Arterial Disease in Patients Presenting Chronic Limb-Threatening Ischemia Study (PROSCAD-CLTI STUDY).

Bruno Salvatore S, Mirabella Domenico D, Dinoto Ettore E, Carlotti Giulia G et al.

Angioplasty in chronic limb-threatening ischemia (CLTI) with paclitaxel-coated device has raised doubts on its long-term safety. Consequently, sirolimus-coated balloons (SCBs) have gained more interest given a better safety profile, maintaining good outcomes. This study aims to evaluate the safety and efficacy of SCB (SELUTION SLR; M.A. Med Alliance SA, Nyon, Switzerland) for femoropopliteal arterial disease in patients presenting with CLTI. This prospective, single-center study included patients with CLTI with femoropopliteal arterial disease treated between October 2022 and July 2024. Exclusion criteria included planned primary amputation and long-segment occlusions (>25 cm) of the femoropopliteal axis. Primary endpoints were technical success and primary patency (PP). Primary patency was defined as freedom from restenosis determined by a duplex ultrasound peak systolic velocity ratio ≤2.4. Secondary endpoints included mortality, clinically driven target lesion revascularization, freedom from reintervention, and from major amputation. A total of 24 patients were treated during the study period with a mean lesion length of 152 ± 54.7 mm. All procedures were conducted successfully. One early death occurred after 3 days of the index procedure because of worsening of pre-existing cardiac failure. At 30 months, PP, mortality, and freedom from major amputation were 95.5%, 91.7%, and 95.7%, respectively. These findings suggest that using an SCB is a safe and effective treatment option for femoropopliteal steno-occlusive lesions in CLTI. These results will need to be confirmed by long-term follow-up and randomized controlled trials.Clinical ImpactConcerns about paclitaxel-coated devices have driven the search for safer alternatives. Sirolimus-coated balloons (SCBs) have demonstrated promising results in terms of primary patency and freedom from clinically driven target lesion revascularization. The SELUTION SLR SCB, with its unique drug delivery technology, emerges as a strong candidate in this context. This observational study aims to evaluate its performance in treating femoropopliteal arterial disease in patients with chronic limb-threatening ischemia. Overall, the SELUTION SLR shows encouraging and consistent outcomes in this challenging patient population.

PMID 42751879
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PubMedFamilial cancer2026-09-17

The clinical and phenotypic spectrum of PTEN hamartoma tumor syndrome: a retrospective cohort study.

Ritter Einat E, Shtaya Aasem Abu AA, Kariv Revital R, Lieberman Sari S et al.

PTEN Hamartoma Tumor Syndrome (PHTS) is a rare condition characterized by a complex phenotype including gastrointestinal hamartomas and increased lifetime malignancy risk. To characterize the clinical phenotype, genetic landscape, and spectrum of malignancies within a large cohort of adult PHTS carriers. A retrospective cohort study of adult patients (≥ 18 years) followed at specialized high-risk clinics who met PHTS diagnostic criteria based on either: (1) identification of a germline PTEN pathogenic/likely pathogenic variant, or (2) fulfillment of clinical criteria according to National Comprehensive Cancer Network (NCCN) guidelines. A total of 62 individuals were included (62.9% male; median age at inclusion 39.5, IQR, 28-52). All fulfilled diagnostic criteria for PHTS. PTEN pathogenic or likely pathogenic variant were identified in 50 individuals (80.6%). Colonic polyps were present in 40 patients. Malignancies were reported in 61% (n = 38), most frequently breast (n=13) and thyroid (n=7). Three individuals (4.8%) had rare soft tissue tumors: one with a desmoid tumor at age 18, one with concurrent axillary osteosarcoma and bilateral chest liposarcoma, and one with a skull-base sarcoma. Macrocephaly was observed in 33.8% (n = 21), and developmental delay, including autism spectrum disorder, in 8 patients (12.9%). One case of PTEN mosaicism was identified through tumor sequencing after negative blood genetic testing. Five individuals (8%) were treated with Sirolimus. This large cohort highlights the broad clinical spectrum and substantial cancer burden in PHTS, including rare soft tissue tumors. Constitutional mosaicism should be considered when clinical suspicion persists despite negative germline testing.

PMID 42753013
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