Rapalimus Gel (sirolimus gel) / Nobelpharma - LARVOL DELTA

Topical sirolimus therapy for cutaneous vascular anomalies: A randomized phase II clinical trial. (PubMed, J Dermatol) - "Sirolimus gel reduced lesion size in our cohort. The safety data demonstrated topical sirolimus to have an acceptable safety profile."

Clinical • Journal • P2 data • Dermatitis • Dermatology • Immunology • Oncology

Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC (clinicaltrials.gov) - P3 | N=43 | Completed | Sponsor: Nobelpharma | Active, not recruiting ➔ Completed

Trial completion

Validation of the Index for Facial Angiofibromas: Data analysis from a randomized controlled trial of sirolimus gel treatment in patients with tuberous sclerosis complex. (PubMed, J Dermatol) - P3 | "The IFA score is potentially clinically useful because of its high validity and reliability. A decrease in score from baseline of ≥1.667 may be considered clinically meaningful."

Journal • Fibrosis • Solid Tumor

Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC (clinicaltrials.gov) - P3 | N=43 | Active, not recruiting | Sponsor: Nobelpharma | Trial primary completion date: Dec 2023 ➔ Oct 2024

Trial primary completion date

Treatment- Sensitive, Dependent, and Refractory Chronic Gvhd: Incidence and Clinical Outcomes (ASH 2023) - "The most common second-line cGVHD therapy used in addition to systemic corticosteroids was largely sirolimus, followed by cyclosporine and tacrolimus. Our findings suggest that cGVHD treatment response states are important predictors of clinical outcomes. Additionally, we have shown that long term follow-up after cGVHD treatment is necessary to determine treatment sensitivity and therefore predict long term outcomes."

Clinical • Clinical data • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Pediatrics • Transplantation

Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC (clinicaltrials.gov) - P3 | N=43 | Active, not recruiting | Sponsor: Nobelpharma | Recruiting ➔ Active, not recruiting

Enrollment closed

Verification of the efficacy of topical sirolimus gel for systemic rare vascular malformations: a pilot study. (PubMed, J Dermatol) - "Blue rubber bleb nevus syndrome, a generalized venous malformation, showed the greatest response. In conclusion, 0.2% sirolimus gel was found to be as clinically effective as systemic sirolimus treatment in patients with venous and capillary malformations and more effective for early active lesions, even systemic venous malformations."

Journal • Fabry Disease • Genetic Disorders • Rare Diseases

A Review of Topical Sirolimus for the Treatment of Facial Angiofibromas in Tuberous Sclerosis Complex. (PubMed, Ann Pharmacother) - "Topical sirolimus 0.2% is a first-in-class, newly Food and Drug Administration (FDA)-approved, mammalian target of rapamycin (mTOR) inhibitor that is a promising and safe, noninvasive alternative to surgical procedures for TSC-associated angiofibromas. Topical sirolimus 0.2% gel is a moderately effective treatment for TSC-associated facial angiofibromas with an adequate safety profile."

Journal • Review • Fibrosis • Oncology • Pediatrics • Solid Tumor

Treatment Options for Management of Facial Angiofibroma Associated with Tuberous Sclerosis Complex: A Systematic Review of Evidence of Clinical Effectiveness and Cost-Effectiveness (ISPOR 2023) - "Pooled meta-analysis of 2 trials showed 63% response rate with topical sirolimus 0.2% gel compared with placebo... Topical sirolimus is a low risk, well-tolerated treatment. This first comprehensive review of clinical and cost-effectiveness evidence on treating FA associated with TSC may provide clinical guidance for therapeutic selections."

Clinical • Cost effectiveness • HEOR • Review • Fibrosis • Solid Tumor

Topical Sirolimus 0.2% Gel for Facial Angiofibroma Associated with Tuberous Sclerosis Complex in the United States: A Cost-Effectiveness Analysis (ISPOR 2023) - "Topical sirolimus gel 0.2% is cost-effective compared to BSC for patients with FA associated with TSC in the US."

Cost effectiveness • HEOR • Fibrosis • Solid Tumor

Topical Sirolimus 0.2% Gel for Treatment of Facial Angiofibroma Associated with Tuberous Sclerosis Complex in the United States: A Budget Impact Analysis (ISPOR 2023) - "The introduction of topical sirolimus 0.2% gel for management of facial angiofibroma associated with TSC in the US is likely to have a very low impact on the US health plans budget."

HEOR • Dermatology • Fibrosis • Solid Tumor

Possibility of precision medicine based on functional analysis of keratin mutation in epidermolysis bullosa simplex (ISID 2023) - "To investigate the pathogenic impact of the K14 mutation, we generated three HaCaT cell lines, K14 wild-type (WT), the hotspot mutation K14 R125C, and K14 G364Rfr*118, in a doxycycline-inducible manner and compared cytological impact between the cell lines. Finally, therapeutic experiments showed that topical sirolimus gel slightly improved the healing of our patient's eruption. Treatment options based on underlying pathogenetic mechanisms may promote precision medicine for EBS and improve patients' quality of life."

Topical Sirolimus 0.2% Gel for the Management of Tuberous Sclerosis Complex-Related Cutaneous Manifestations: An Interim Analysis of Postmarketing Surveillance in Japan. (PubMed, Dermatol Ther (Heidelb)) - "Topical sirolimus 0.2% gel is effective in the management of TSC-related cutaneous manifestations and generally well tolerated. Age and duration of usage had a significant association with the effectiveness or safety of topical sirolimus 0.2% gel, whereas total dosage had a significant association with the effectiveness."

Journal • P4 data • Fibrosis • Genetic Disorders • Renal Disease • Solid Tumor

Pilot study for the treatment of cutaneous neurofibromas in neurofibromatosis type 1 patients using topical sirolimus gel. (PubMed, J Am Acad Dermatol) - No abstract available

Journal • Fibrosis • Genetic Disorders • Neurofibromatosis • Solid Tumor

Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC (clinicaltrials.gov) - P3 | N=40 | Recruiting | Sponsor: Nobelpharma

New P3 trial • Solid Tumor

A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I (clinicaltrials.gov) - P3 | N=100 | Terminated | Sponsor: Nobelpharma | Trial completion date: Apr 2022 ➔ Dec 2021 | Active, not recruiting ➔ Terminated | Trial primary completion date: Apr 2022 ➔ Dec 2021; Sponsor decision

Trial completion date • Trial primary completion date • Trial termination • Fibrosis • Genetic Disorders • Neurofibromatosis • Solid Tumor

"EU Filings For Parsaclisib & Sirolimus Gel Submitted For Rare Diseases https://t.co/MKM8BoKSwz #PinkSheet" (@PharmaPinkSheet)

European regulatory • Rare Diseases

A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I (clinicaltrials.gov) - P3; N=100; Active, not recruiting; Sponsor: Nobelpharma; Recruiting ➔ Active, not recruiting

Enrollment closed • Fibrosis • Genetic Disorders • Neurofibromatosis • Solid Tumor