sirolimus (NPC 12G / Hyftor / rapalimus)

✓ Approved

What is sirolimus?

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

Drug Profile

Brand Names	NPC 12G, Hyftor, rapalimus
Company	Nobelpharma Co., Ltd.
Molecular Target	MTOR
Route	Topical
Status	Approved

Sources: ClinicalTrials.gov, Nobelpharma Co., Ltd.

Mechanism of Action

Molecular Targets

sirolimus acts on 1 molecular target:

MTOR	mechanistic 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 Area	Condition	Phase
Neoplasms benign, malignant and unspecified (incl cysts and polyps)	Angiofibroma	✓ Approved
Respiratory, thoracic and mediastinal disorders	Lymphangioleiomyomatosis	✓ Approved
Congenital, familial and genetic disorders	Lymphatic malformation	✓ Approved
Congenital, familial and genetic disorders	Neurofibromatosis	Phase III

Related Research Articles

PubMedJournal of mass spectrometry and advances in the clinical lab2026-11-11

Preliminary Analytical Evaluation of the Automated Cobas i601 LC-MS/MS System for Immunosuppressant Quantification in a Clinical Laboratory.

Serres-Gómez Mariana M, Gemma Serrano Olmedo María M, Luisa González-Casaus María M, Fernández-Puntero Belén B et al.

Therapeutic drug monitoring (TDM) of immunosuppressive drugs is essential to maintain concentrations within narrow therapeutic windows and minimize toxicity. Automated liquid chromatography-tandem mass spectrometry (LC-MS/MS) platforms can combine the analytical specificity of mass spectrometry with workflow efficiency suitable for routine laboratories. To preliminarily evaluate the analytical performance of the Cobas i601 automated LC-MS/MS system for quantifying tacrolimus, cyclosporine, everolimus, and sirolimus and to compare results with routine immunoassays and an LC-MS/MS method for sirolimus. Imprecision, bias, and linearity were assessed according to the Clinical Laboratory Standards Institute (CLSI) EP10-A3 standard using quality-control materials. Method comparison and bias estimation versus the Dimension EXL 200 immunoassays and an LC-MS/MS technique for sirolimus followed the CLSI EP09-A3 standard. Regression analyses and predicted biases at therapeutic range concentrations were compared with allowable limits. The Cobas i601 showed good precision and linearity, meeting manufacturer specifications. The highest coefficients of variations for tacrolimus, everolimus, cyclosporine, and sirolimus were 4.0%, 4.7%, 3.6%, and 4.8%, and the maximum EP10-A3 biases were -7.4%, -3.9%, 4.7%, and 5.4%, respectively. In the method comparison, tacrolimus, everolimus, and cyclosporine showed substantial negative biases versus immunoassays (-22.7%, -38.9%, and -37.5%), exceeding allowable limits. Sirolimus exhibited smaller positive bias relative to the LC-MS/MS method (12.5%). Interchangeability between the methodologies cannot be assumed. The Cobas i601 provides reliable automated LC-MS/MS quantification of immunosuppressants, with good precision and linearity. Method-dependent biases highlight the need for careful interpretation during technology transitions. The Cobas i601 shows strong potential for routine LC-MS/MS implementation in TDM in clinical laboratories.

PMID 43011875

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PubMedJournal of the American Academy of Dermatology2026-11-10

Topical sirolimus gel for hyperplastic rosacea: A randomized, double-blind, controlled study and exploration of two-dimensional to three-dimensional approximate protrusion volume (APV) assessment.

Xu Bingyang B, Chen Hao H, Chu Jiali J, Wang Yuanqin Y et al.

Hyperplastic rosacea is difficult to treat. Sirolimus may be a topical option. To evaluate topical sirolimus gel and explore two-dimensional-to-three-dimensional approximate protrusion volume (APV) assessment. In this randomized, double-blind, placebo-controlled trial, 65 patients applied 0.2% sirolimus gel (n = 29) or placebo (n = 36) twice daily. The primary endpoint was an Investigator's Global Assessment (IGA) score ≤1 after a 12-week cycle. Secondary and exploratory endpoints included Clinician's Erythema Assessment (CEA) success, artificial intelligence-estimated APV change, subgroup analyses, and correlations between APV and clinical scores. IGA success was 72.4% versus 33.3% (adjusted relative risk, 1.949; 95% confidence interval [CI], 1.153-3.294; P = .013), and CEA success was 69.0% versus 30.6% (adjusted relative risk, 2.778; 95% CI, 1.627-4.743; P < .001). APV reduction was greater with sirolimus (-61.556 units; P < .001). Baseline APV correlated with IGA (ρ = 0.386; P = .001) and CEA (ρ = 0.314; P = .011). No serious adverse events occurred. Single-center design, small sample, short follow-up, unvalidated algorithm, and no systemic absorption, quality-of-life, or satisfaction assessments. Topical sirolimus gel showed exploratory clinical and APV signals; confirmatory trials are needed.

PMID 43007256

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PubMedJournal of medical case reports2026-11-10

Delayed diagnosis of tuberous sclerosis complex in a young adult with long-term refractory epilepsy: a case report.

Zhao Dapeng D, Yu Ailing A, Chang Yanyan Y, Xu Linying L et al.

Tuberous sclerosis complex (TSC) is a rare autosomal dominant neurocutaneous hamartomatous disorder characterized by widespread multi-system hamartomatous lesions, with epilepsy occurring in the majority of affected patients. When refractory epilepsy serves as the sole long-standing clinical manifestation, subtle cutaneous and radiological hallmarks are easily overlooked, inevitably leading to diagnostic delay. We herein report a 23-year-old female patient presenting with a decade-long history of drug-refractory seizures. Physical examination revealed mild cognitive impairment with a Mini-Mental State Examination (MMSE) score of 22 (technical secondary school education) and no focal neurological deficits. A typical lumbosacral shagreen patch and a surgical scar from prior great toe periungual fibroma resection were identified on cutaneous inspection. Brain MRI demonstrated white matter migration lines on fluid-attenuated inversion recovery (FLAIR) sequences, while cranial CT detected subependymal calcified nodules. Sanger genetic sequencing confirmed a heterozygous pathogenic splice-site variant NM_000368.5:c.107-2A > G in the TSC1. Given her childbearing potential, valproate was gradually tapered and substituted with lamotrigine to minimize teratogenic risks. Sirolimus-mediated mTOR-targeted therapy was initiated alongside renoprotective agents and regular serial imaging surveillance. Optimized combination therapy yielded a remarkable reduction in seizure frequency during follow-up. TSC may manifest insidiously for years with refractory epilepsy as its only prominent clinical feature. Comprehensive screening consisting of thorough cutaneous examination, standardized cranial imaging and molecular genetic testing is mandatory for early identification of this disorder. Individualized antiseizure regimens combined with mechanistic target of rapamycin (mTOR) inhibitors can significantly improve seizure control and long-term clinical outcomes in TSC patients.

PMID 43004460

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PubMedOrphanet journal of rare diseases2026-11-06

Long-term treatment with sirolimus for pediatric vascular anomalies.

Park Jueun J, Kwon Hyunhee H, Kim Dae Yeon DY, Kim Seong Chul SC et al.

Although sirolimus has emerged as a safe and effective treatment modality for unresectable vascular anomalies, comprehensive long-term follow-up data remain scarce in the current literature. This study aims to evaluate the long-term clinical experience of pediatric patients with vascular anomalies who received sirolimus treatment over five years. We retrospectively analysed 17 pediatric patients with vascular anomalies treated with sirolimus over five years in Asan Medical Center. Lesion volumes were measured via 3D volumetric MRI and normalized to body surface area (BSA). Adverse drug effects and therapeutic responses were periodically assessed. Longitudinal response was modeled using generalized additive mixed-effects models (GAMM). Tapering protocols were initiated after 24 months of stability, and alpelisib switch was considered for sirolimus-refractory cases with documented PIK3CA mutations. With a median follow-up of 78 months, the overall response rate was 81.3%. GAMM analysis demonstrated a significant non-linear volume reduction (p < 0.0001), characterized by a rapid initial response within 12-24 months followed by a sustained plateau. Tapering was successful in 70.6% of patients without disease progression. Three patients with suboptimal responses transitioned to alpelisib; those with sufficient follow-up achieved additional volume reduction within 12 months. Long-term sirolimus use was well-tolerated, with no Grade ≥ 3 adverse events reported. Sirolimus provides sustained long-term efficacy and safety in pediatric vascular anomalies. The observed treatment plateau suggests that dose tapering is a viable strategy for sustained responders. Furthermore, genotype-guided transition to alpelisib offers an effective alternative for refractory PIK3CA-mutant cases.

PMID 42989251

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PubMedCardiology in the young2026-11-06

Stopping sirolimus in cardiac rhabdomyoma: is 75% regression the right finish line?

Jindal Meera M, Kamal Murtaza M

PMID 42990024

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PubMedIJU case reports2026-11-06

Bladder Perivascular Epithelioid Cell Neoplasm With Transcription Factor E3 Gene Rearrangement Treated by mTOR Inhibitor and Partial Cystectomy: A Case Report.

Kaeriyama Kimika K, Kakitsuba Tadashi T, Seo Wonseok W, Kurokawa Tetsuyuki T et al.

Perivascular epithelioid cell neoplasms originating in the urinary bladder are rare, and their standard treatment methods have not been established. A 14-year-old girl with intermittent macroscopic hematuria was referred to our hospital. Ultrasonography and magnetic resonance imaging revealed a 5 cm spherical mass in the bladder neck and transurethral biopsy revealed a perivascular epithelioid cell neoplasm. Chromosome G-banding analysis indicated a transcription factor E3 gene rearrangement. The mTOR inhibitor, sirolimus, was administered for six months, followed by partial cystectomy. No tumor recurrence was observed after 15 months. This case suggests the potential efficacy of partial cystectomy combined with neoadjuvant mTOR inhibitor therapy for transcription factor E3 gene rearrangement-positive perivascular epithelioid cell neoplasms.

PMID 42992336

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sirolimus (NPC 12G / Hyftor / rapalimus)

What is sirolimus?

Drug Profile

Mechanism of Action

Molecular Targets

Therapeutic Indications

Related Research Articles

Preliminary Analytical Evaluation of the Automated Cobas i601 LC-MS/MS System for Immunosuppressant Quantification in a Clinical Laboratory.

Topical sirolimus gel for hyperplastic rosacea: A randomized, double-blind, controlled study and exploration of two-dimensional to three-dimensional approximate protrusion volume (APV) assessment.

Delayed diagnosis of tuberous sclerosis complex in a young adult with long-term refractory epilepsy: a case report.

Long-term treatment with sirolimus for pediatric vascular anomalies.

Stopping sirolimus in cardiac rhabdomyoma: is 75% regression the right finish line?

Bladder Perivascular Epithelioid Cell Neoplasm With Transcription Factor E3 Gene Rearrangement Treated by mTOR Inhibitor and Partial Cystectomy: A Case Report.

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