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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

PubMedExpert opinion on investigational drugs2026-10-15

Zovegalisib for PIK3CA-driven vascular and lymphatic malformations: a drug evaluation.

Seront Emmanuel E, Van Damme An A, Coulie Julien J, Vikkula Miikka M et al.

PIK3CA-driven overgrowth and vascular malformations are mosaic developmental disorders in which chronic pathway inhibition may be required from childhood into adulthood. Alpelisib established PI3Kα as a clinically actionable target, but inhibition of wild-type PI3Kα can produce on-target toxicities, principally hyperglycemia, diarrhea and stomatitis, that are relevant to long-term therapy. Zovegalisib (RLY-2608) is an oral allosteric, pan-mutant-selective PI3Kα inhibitor designed to preferentially inhibit mutant over wild-type PI3Kα. This Drug Evaluation reviews PIK3CA biology, the current systemic-treatment landscape, zovegalisib pharmacology, and the preliminary Phase 2 ReInspire data. PubMed/MEDLINE, ClinicalTrials.gov, regulatory sources, and publicly available conference materials and company disclosures were reviewed through 20 August 2026. At the formal 15 April 2026 cutoff, early ReInspire results showed a 60% week-12 volumetric response rate in 20 evaluable adults/adolescents; one unconfirmed response reported after the cutoff in a sponsor press release increased the descriptive rate to 65%. Activity after prior sirolimus and/or alpelisib and encouraging short-term tolerability support further development. Mutant selectivity could be especially valuable in chronic nonmalignant disease, but superiority over alpelisib is unproven.

PMID 42875408
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PubMedAME case reports2026-10-14

Giant mixed vascular lymphatic malformation in a pediatric patient: a case report.

Qin Hui H, Wu Meixian M, Lu Jiang J, Qin Xiaoxin X et al.

Giant Mixed vascular lymphatic malformation-is a relatively rare congenital anomaly, particularly in pediatric populations. Mediastinal vascular malformations are uncommon in children, and their exact incidence remains poorly defined in the literature. It is not a true neoplasm but rather a low-flow vascular malformation arising from aberrant development of the vascular system during embryogenesis. Due to nonspecific clinical manifestations (e.g., cough, dyspnea) and the deep location of the mediastinum-adjacent to the heart, great vessels, and airways-diagnosis and management pose considerable challenges. Misdiagnosis is common, potentially leading to inappropriate treatment and life-threatening complications. Thus, accurate preoperative imaging is essential for developing safe and effective treatment strategies. We report the case of a 3-year-old girl who presented with a persistent cough lasting more than 20 days. Imaging revealed a cystic-solid mass measuring approximately 53 mm × 37 mm × 67 mm in the left middle-upper mediastinum. Contrast-enhanced computed tomography (CT) showed mild progressive enhancement of the lesion, with distinct "branch-like" vascular structures communicating with the left brachiocephalic vein, consistent with a low-flow vascular malformation. No definite arterial feeding vessel was identified. The mass was well-demarcated from the aorta and heart but caused localized compression and narrowing of the left main bronchus. These imaging findings were consistent with a low-flow vascular malformation. A biopsy obtained via video-assisted thoracoscopic surgery (VATS) confirmed the diagnosis of mixed vascular malformation. Given the high-risk location adjacent to vital mediastinal structures, complete surgical resection was considered extremely hazardous. The multidisciplinary team therefore adopted a non-surgical approach consisting of oral sirolimus as targeted therapy. Following treatment, the child's cough improved significantly. This case illustrates that in children with cystic-solid mediastinal masses, the presence of "progressive enhancement" and "clearly enhanced vascular channels" on contrast-enhanced CT or magnetic resonance imaging (MRI) should raise suspicion for vascular malformations. Comprehensive imaging assessment is fundamental for accurate diagnosis and formulation of individualized management. For complex mediastinal vascular malformations with high surgical risk, oral sirolimus represents a safe and effective targeted therapeutic alternative, capable of alleviating symptoms and controlling disease progression.

PMID 42871551
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PubMedACG case reports journal2026-10-13

Mimic of Peritoneal Carcinomatosis: Protein-Losing Enteropathy Revealing HRAS-Driven Generalized Lymphatic Anomaly.

Kamalumpundi Vijayvardhan V, Saey Stephanie S, Khanna Sahil S, Peterson Joshua J et al.

Complex lymphatic anomalies can present with a protein-losing enteropathy. A 25-year-old woman presented with steatorrhea, weight loss, anasarca, and hypoalbuminemia. Abdominal computed tomography revealed diffuse peritoneal soft tissue lesions concerning for carcinomatosis. Omental biopsies and magnetic resonance lymphangiography established generalized lymphatic anomaly. Targeted genetic sequencing identified a novel postzygotic HRAS insertion mutation. She was started on a low-fat diet, sirolimus, and trametinib monotherapy with modest symptomatic response. However, she continued to have severe malnutrition. Alternating therapy of sirolimus and trametinib is ongoing. This is the first reported case of protein-losing enteropathy as the presenting manifestation of an HRAS-driven generalized lymphatic anomaly.

PMID 42866326
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PubMedPediatric blood & cancer2026-10-12

Successful Use of Sutimlimab in two Pediatric Patients With Refractory Cold Antibody-Mediated Autoimmune Hemolytic Anemia.

Zaheer Sibgha S, Friedmann Rachel R, Cheng Kimberly K, Young Guy G et al.

Cold antibody-mediated autoimmune hemolytic anemia, or cold agglutinin syndrome (CAS), in pediatrics is typically transient, although it can often be severe and require transfusion support. Recurrence in children is rare, but those with underlying immune dysfunction can have a refractory course with limited options available. We report a 4-year-old girl with Evans syndrome and life-threatening CAS refractory to transfusions, plasmapheresis, steroids, and immunosuppression as well as a 12-month-old boy status post liver transplant with CAS refractory to steroids, rituximab, and tacrolimus cessation with transition to sirolimus, both of whom received sutimlimab, a C1s inhibitor, with rapid improvement in hemolysis, transfusion independence, and a sustained remission. To our knowledge, these are the first reported uses of sutimlimab in the pediatric population.

PMID 42862050
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PubMedJAMA2026-10-12

Heart Transplant for Advanced Heart Failure: A Review.

Amancherla Kaushik K, Shah Ravi R, Shah Ashish S AS, Schlendorf Kelly H KH et al.

Heart failure affects more than 6 million US adults; approximately 4% to 5% progress to advanced disease despite optimal therapy. Heart transplant is a class I guideline-recommended therapy for select individuals with advanced heart failure. Early referral for heart transplant is guided by the "I NEED HELP" mnemonic, a tool that identifies patients with advanced heart failure based on criteria such as use of inotropes, persistent symptoms (New York Heart Association class III or IV), end-organ dysfunction, left ventricular ejection fraction, defibrillator shocks, hospitalizations for heart failure, persistent edema or escalating use of diuretics, and low blood pressure. Each criterion is associated with higher risk of death or first heart failure hospitalization over a median follow-up of 260 days (adjusted hazard ratio, 1.19 [95% CI, 1.11-1.27] for each additional criterion). Typical absolute contraindications to heart transplant include active systemic infection, malignancy with limited life expectancy, severe and irreversible end-organ dysfunction (eg, cirrhosis, severe pulmonary hypertension), active substance use, major untreated psychiatric illness, inadequate social support, and severe frailty. Approximately 4600 heart transplants are performed annually in the US. The mortality rate was 8.5 deaths/100 patient-years for adults on the transplant waiting list in 2023. Leading causes of early death after transplant include primary graft dysfunction (39.5%), multiorgan failure (18.5%), and infection (13.9%). Among adults, contemporary survival rates after heart transplant are 91.5% at 1 year and 80.3% at 5 years. Lifelong maintenance immunosuppression typically combines a calcineurin inhibitor, antiproliferative agents (typically mycophenolate mofetil or azathioprine) and corticosteroids, with selective use of induction therapy (intensive immunosuppression immediately after transplant). Acute rejection requiring treatment occurs in 20% to 25% of recipients within a year, and may be treated with high-dose corticosteroids, antithymocyte globulin, and B-cell-targeted therapies (such as rituximab or bortezomib). Rejection surveillance with endomyocardial biopsies of the right ventricular septum is performed 5 to 10 times within the first year after transplant. Cardiac allograft vasculopathy (chronic rejection that causes diffuse coronary narrowing) affects 29% of heart transplant recipients within 5 years and 46.8% by 10 years and is the leading cause of late mortality. Cardiac allograft vasculopathy is treated with statins, mechanistic target of rapamycin inhibitors (such as sirolimus or everolimus), and retransplant for severe cases. Infections (62.5% are bacterial, 30.0% are viral, 6.7% are fungal, and 0.8% are parasitic) and malignancy (10.7% within 5 years after heart transplant) are other leading causes of late mortality. Heart transplant is the definitive therapy for carefully selected patients with advanced heart failure despite receiving optimal, guideline-directed therapy. After heart transplant, lifelong multidisciplinary care is necessary to manage immunosuppression, prevent and treat rejection and cardiac allograft vasculopathy, and monitor for infection and malignancy.

PMID 42862549
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PubMedAging and disease2026-10-11

Lysosomal Competence in Skeletal Muscle Aging: Autophagic Flux, Measurement Gaps, and Human Translation.

Dogsom Oyungerel O, Kim Jae Gyu JG, Lee Yeon-Hee YH, Jahr Holger H et al.

Age-related deterioration of skeletal muscle contributes to loss of mobility, frailty, and disability. Altered autophagy-lysosome competence has been proposed as a mechanism linking impaired proteostasis, mitochondrial damage, inflammation, and regenerative failure to sarcopenia. Chronological muscle aging and clinically defined sarcopenia overlap, but evidence from healthy aging, frailty, disuse, cachexia, obesity, or chronic disease cannot be treated as interchangeable. This narrative review examines how lysosomal acidification, degradative capacity, biogenesis, and turnover influences autophagic flux in aging muscle and evaluates the strength of evidence across experimental models and human studies. Genetic and pharmacological studies show that severe disruption of autophagy or specific lysosomal functions can cause myofiber damage, mitochondrial impairment, defective regeneration, and weakness. However, direct evidence that lysosomal acidification, fusion, hydrolase activity, membrane integrity, recycling, cargo degradation, or completed autophagic flux is impaired in aging human muscle remains limited. Much of the human literature relies on static abundance of LC3-II, SQSTM1/p62, lysosomal proteins, or transcriptional signatures, which cannot distinguish increased autophagosome formation from impaired degradation. We therefore separate causal experimental evidence from human associations and focus on V-ATPase, TMEM175, LAMP proteins, TFEB/TFE3, lysophagy, and crosstalk with mitophagy and the ubiquitin-proteasome system. Exercise improves clinically relevant outcomes in older adults, but mediation through completed autophagic flux has not been established. Recent trials of metformin and once-weekly sirolimus did not improve prespecified functional outcomes in the tested populations and regimens, whereas urolithin A produced endpoint-specific signals while missing key primary outcomes.

PMID 42858326
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