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sirolimus (Excel / Excel II)

✓ Approved

JW Medical Systems · MTOR · Small Molecule

What is sirolimus?

sirolimus is a small molecule developed by JW Medical Systems. It is approved for therapeutic indications via surgical implantation.

Drug Profile

Brand NamesExcel, Excel II
CompanyJW Medical Systems
Drug ClassSmall Molecule
Molecular TargetMTOR
RouteSurgical Implantation
StatusApproved
Sources: ClinicalTrials.gov, JW Medical Systems

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 1 unique indication across 1 therapeutic area.

Therapeutic AreaConditionPhase
Injury, poisoning and procedural complicationsRestenosis✓ Approved

Related Research Articles

PubMedJournal of clinical medicine2026-10-16

Contemporary Endovascular Treatment of Femoropopliteal Disease: Lesion Preparation, Antiproliferative Therapies, and Emerging Technologies.

Río Solá María Lourdes Del MLD, Fernández Sandra Pérez SP, Asensio Rodríguez Sergio S

Background: Endovascular treatment of femoropopliteal atherosclerotic disease has evolved from plain balloon angioplasty and routine stenting toward lesion-oriented strategies that combine plaque assessment, selective vessel preparation, antiproliferative drug delivery, and provisional scaffolding. The increasing availability of drug-coated balloons, drug-eluting stents, atherectomy, intravascular lithotripsy, intravascular imaging, and sirolimus-based technologies has improved therapeutic options but has also made device selection more complex. Objective: To synthesize comparative evidence and present a practical lesion-oriented framework for selecting vessel preparation, antiproliferative therapy, and selective scaffolding. Methods: A structured narrative review of contemporary evidence was performed, prioritizing randomized controlled trials, head-to-head comparisons, systematic reviews, meta-analyses, and consensus documents relevant to femoropopliteal intervention. Results: Lesion morphology-particularly length, chronic total occlusion, calcification pattern, popliteal involvement, and in-stent restenosis-strongly influences procedural strategy and durability. Atherectomy and specialty balloons can optimize acute vessel preparation in selected lesions, although consistent long-term superiority has not been demonstrated. Intravascular lithotripsy has the strongest randomized evidence for heavily calcified lesions, reducing flow-limiting dissection and bailout stenting. Paclitaxel-coated balloons remain a central leave-nothing-behind therapy, with contemporary evidence not confirming the previously proposed late mortality signal. Sirolimus-coated balloons have now entered the field with randomized noninferiority data versus paclitaxel through 2 years. Drug-eluting and other contemporary stents remain important when recoil, dissection, or complex anatomy make a purely scaffold-free strategy unreliable. Conclusions: Contemporary femoropopliteal intervention is best approached as a sequence of lesion characterization, selective preparation, antiproliferative therapy, and selective scaffolding. Treatment should be matched to lesion morphology and the final mechanical result, with focal repair or stenting when dissection or recoil compromises flow.

PMID 42882111
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PubMedThe Journal of clinical and aesthetic dermatology2026-10-15

Keratosis Pilaris Rubra Faciei: Assessing the Unmet Therapeutic Need.

Snow Katherine K, Ryan Irene I, Maguiness Sheilagh S, Siegfried Elaine E

Keratosis pilaris rubra faciei (KPRF) is a clinical variant of keratosis pilaris characterized by prominent facial erythema and follicular papules that can cause significant aesthetic concern. The condition is underrecognized, frequently self-diagnosed, and lacks established evidence-based treatment guidelines. To characterize the demographics, diagnostic pathways, treatment experiences, and psychosocial impact among individuals with KPRF and to explore perceived responses to commonly used therapies, including topical sirolimus. A 24-item survey was distributed to members of a Reddit-based KPRF support group. Survey domains included demographics, diagnostic history, symptom triggers, treatments trialed, and mental health impact. Descriptive statistics were calculated using RStudio. Seventy-nine respondents completed the survey. Most were men (73.4%) aged 18 to 30 years (70.9%) and resided outside the United States (69.6%). Symptom onset typically occurred before age 18, though diagnosis was frequently delayed; 38% reported self-diagnosis with social media input. The face was the most affected site (98.7%). Nearly all participants (98.7%) reported a negative impact on mental health. Common treatments included pulsed dye laser, topical keratolytics, retinoids, and topical sirolimus. Among participants who used compounded topical sirolimus, 75% reported improvement during treatment. The primary limitations of this study include the relatively small sample size and potential response bias inherent in self-reported survey data. KPRF is associated with delayed diagnosis, frequent reliance on social media for information, and substantial psychosocial burden. Self-reported improvement with topical sirolimus highlights the need for controlled studies and greater clinical awareness of this underrecognized condition.

PMID 42877935
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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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PubMedJournal of clinical oncology : official journal of the American Society of Clinical Oncology2026-10-15

First-In-Human Study of 211At-CD45 Radioimmunotherapy With Nonmyeloablative Conditioning Hematopoietic Cell Transplantation for Adults With Hematologic Malignancies.

Vo Phuong T PT, Walter Roland B RB, Gooley Ted A TA, Hamlin Donald K DK et al.

Relapse is the dominant cause of failure after allogeneic hematopoietic cell transplantation (HCT) for aggressive hematologic malignancies. CD45-directed radioimmunotherapy (RIT) has been investigated to intensify conditioning and augment antitumor efficacy. After gaining clinical experience with β-emitting radionuclides, attention is now shifting toward α-emitters, such as astatine-211 (211At), for their more potent and precise radiation delivery with minimized toxicity to surrounding tissues. To our knowledge, in this first-in-human, two-stage dose-finding study, we administered a single infusion of 211At-labeled anti-CD45 monoclonal antibody before nonmyeloablative conditioning with fludarabine/2-3 Gy total body irradiation and allografting from HLA-matched or single-allele-mismatched donors in 45 adults (38-74 years of age) at a high relapse risk from myeloid malignancies or acute leukemia. Seventeen patients were treated in stage I with 85-591 μCi/kg of 211At. The patient receiving 591 μCi/kg developed veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS), thereby establishing 500 μCi/kg as the protocol-defined maximum tolerated dose and prompting initiation of stage II at that dose in 28 patients. Infusion reactions were mild. 211At-CD45 RIT did not delay hematopoietic recovery, impair donor chimerism, or increase graft-versus-host disease (GVHD) rates. In stage II, six patients (all receiving sirolimus as part of GVHD prophylaxis for unrelated donor HCT) experienced VOD/SOS. Among the 13 patients with circulating blasts at baseline, blasts cleared in 12 before initiation of fludarabine/total body irradiation, demonstrating immediate cytotoxic activity. Estimated 1-year relapse-free and overall survival were 42% (95% CI, 28%-56%) and 49% (34%-62%), respectively. 211At-CD45 RIT can be incorporated into nonmyeloablative conditioning, with liver toxicity defining activity limits. Evidence of antitumor efficacy supports phase II evaluation.

PMID 42879732
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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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