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Also known as QTORIN 3.9% rapamycin anhydrous gel, QTORIN 3.9% Rapamycin Anhydrous Gel
QTORIN 3.9% Rapamycin Anhydrous · 3 trials · 3 indications
A multi-point scale with higher values indicating improvement.
| Arm | Type | Description |
|---|---|---|
| Active | EXPERIMENTAL | - |
| Vehicle | PLACEBO_COMPARATOR | - |
| Single-arm, open-label | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| QTORIN 3.9% Rapamycin Anhydrous Gel | DRUG | QTORIN 3.9% Rapamycin Anhydrous Gel |
| Vehicle | DRUG | Matching vehicle (QTORIN anyhydrous gel) |
Key Inclusion Criteria: * Participant must be at least 3 years of age at time of consent/assent * Participant must have a clinically confirmed superficial/cutaneous microcystic lymphatic malformation Key Exclusion Criteria: * Participants who have vascular malformations that interfere with evalua...
QTORIN 3.9% Rapamycin Anhydrous is an investigational topical gel being developed for the treatment of rare diseases, including Pachyonychia Congenita, Cutaneous Venous Malformations, and Microcystic Lymphatic Malformation. It is a small molecule therapy in Phase 3 clinical development for these conditions.
QTORIN 3.9% Rapamycin Anhydrous contains rapamycin, which targets the mTOR pathway. By inhibiting mTOR, it is designed to modulate cellular growth and proliferation, which is relevant to the abnormal tissue growth seen in the rare diseases it is being studied for.
QTORIN 3.9% Rapamycin Anhydrous is being developed by Palvella Therapeutics, Inc., a biopharmaceutical company focused on rare diseases. Palvella Therapeutics is publicly traded under the ticker symbol PVLA.
QTORIN 3.9% Rapamycin Anhydrous is in Phase 3 clinical development. It has received Breakthrough Therapy, Fast Track, and Orphan Drug designations from the FDA for its investigational use in rare disease indications.
QTORIN 3.9% Rapamycin Anhydrous is being studied in several trials. NCT05180708 is a completed Phase 3 study in Pachyonychia Congenita. NCT06239480 is an active Phase 3 study in Microcystic Lymphatic Malformations. NCT06653842 is an active Phase 2 study in Cutaneous Venous Malformations.
QTORIN 3.9% Rapamycin Anhydrous is also known as QTORIN 3.9% rapamycin anhydrous gel. In clinical trials, it has been referred to as PTX-022, as seen in the study of Cutaneous Venous Malformations, where the drug is described as PTX-022 (Sirolimus) Topical Gel 3.9%.