QTORIN rapamycin 3.9 percent gel

Microcystic lymphatic malformations can cause lymphorrhea, bleeding, pain, itching, malodor, functional impairment, and recurrent infection or inflammation. Tongue involvement can impair function and quality of life. The sponsor estimates more than 30,000 diagnosed individuals in the United States. A separate physician-survey study estimated 79,920 annually managed individuals with lymphatic malformations having a cutaneous component, corresponding to 24.1 per 100,000 population. That estimate includes microcystic and mixed malformations and is not an estimate of the total national prevalence of microcystic lymphatic malformations.

Historical United States inpatient evidence for pediatric lymphatic malformations reported average charges of $30,995 per admission using data from 2000, 2003, 2006, and 2009. These are historical hospital charges for lymphatic malformations broadly, not current costs specific to microcystic disease.

SELVA enrolled 51 participants, of whom 50 initiated QTORIN rapamycin treatment. The primary efficacy population comprised 49 participants aged at least 6 years. At week 24, the mean change on the mLM-IGA was +2.13 points, with p<0.001. Among 43 participants aged at least 6 years who completed the efficacy evaluation period, 41 experienced at least a 1-point improvement.

Treatment-related adverse events occurred in 17 of 50 participants (34%). Four participants (8%) experienced serious adverse events assessed as unrelated to study treatment. SELVA was a single-arm, baseline-controlled study without a concurrent placebo or active-treatment comparator.

The sponsor presented a potential annual pricing range of approximately $100,000 to $200,000 per patient. The presentation does not identify that range as ASP or WAC. A potential annual price range alone does not establish payer budget impact, which also depends on eligible population, uptake, treatment duration, reimbursement, and changes in other healthcare utilization. No QTORIN-specific budget impact model is included in the evidence collected for this report.

QTORIN rapamycin is an investigational topical sirolimus formulation. The sponsor announced completion of its rolling NDA submission on August 31, 2026. Submission does not establish regulatory approval.

The available phase 3 evidence describes improvement during a 24-week treatment period in a single-arm study of superficial/cutaneous microcystic lymphatic malformations. The study excluded participants with severe systemic symptoms requiring systemic therapy. These findings do not establish comparative efficacy against surgery, sclerotherapy, other topical sirolimus formulations, or systemic treatment. The pricing projection does not establish cost effectiveness or budget impact.