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QTORIN rapamycin

Phase 3

Pachyonychia Congenita | Small molecule | Rare Disease |Palvella Therapeutics, Inc.|Trials Updated: Mar 27, 2026

QTORIN rapamycin target and mechanism

ModalitySmall molecule

Also known as QTORIN 3.9% rapamycin anhydrous gel, QTORIN 3.9% rapamycin anhydrous

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials3
Total Enrollment153

FDA Designations

FAST_TRACKORPHAN_DRUG

QTORIN rapamycin clinical trials

QTORIN rapamycin · 3 trials · 3 indications

Phase 3 2Phase 2 1
NCT06239480SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic MalformationsMicrocystic Lymphatic Malformation
ACTIVE NOT_RECRUITING51 Analytics
NCT05180708A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia CongenitaPachyonychia Congenita
COMPLETED87 Analytics
PHASE3ACTIVE NOT_RECRUITING
SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations
Microcystic Lymphatic MalformationUnlock trial analytics
PHASE3COMPLETED
A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita
Pachyonychia CongenitaUnlock trial analytics

Study Endpoints

Primary Endpoints

Overall microcystic lymphatic malformations Investigator Global Assessment (mLM-IGA)
At Week 24

A multi-point scale with higher values indicating improvement.

Patient Global Assessment of Activities Difficulty Scale
6 months
Incidence of Treatment-Emergent Adverse Events
Week 12

Secondary Endpoints

Change in the blinded clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS)
At Week 24
Change in the live clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS)
Change from Baseline to Week 24
Overall patient global impression of change (PGI-C)
Change from Baseline to Week 24
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ActiveEXPERIMENTAL -
VehiclePLACEBO_COMPARATOR -
Single-arm, open-labelEXPERIMENTAL -

Interventions

NameTypeDescription
QTORIN 3.9% Rapamycin Anhydrous GelDRUGQTORIN 3.9% Rapamycin Anhydrous Gel
VehicleDRUGMatching vehicle (QTORIN anyhydrous gel)
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Eligibility Criteria

Age Range3 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites15

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...

Countries:United StatesUnited Kingdom
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Frequently asked questions about QTORIN rapamycin

What is QTORIN rapamycin?

QTORIN rapamycin is an investigational topical small molecule developed by Palvella Therapeutics for dermatologic conditions. It is formulated as a 3.9% rapamycin anhydrous gel and is being studied in cutaneous venous malformations, microcystic lymphatic malformation, and pachyonychia congenita. It is not approved and remains in clinical development.

What is QTORIN rapamycin used for?

QTORIN rapamycin is being developed to treat cutaneous venous malformations, microcystic lymphatic malformation, and pachyonychia congenita. These are dermatologic conditions involving vascular and lymphatic malformations and a rare keratinization disorder. The drug is investigational and has not been approved for any indication.

Who makes QTORIN rapamycin?

QTORIN rapamycin is developed by Palvella Therapeutics, Inc., which trades under the ticker PVLA. The company is advancing the topical rapamycin gel across vascular malformation and pachyonychia congenita programs.

What phase is QTORIN rapamycin in?

QTORIN rapamycin is in Phase 2 and Phase 3 clinical development depending on the indication. A Phase 2 study is evaluating it in cutaneous venous malformations, while Phase 3 studies have been conducted in microcystic lymphatic malformation and pachyonychia congenita. It is investigational and not FDA approved.

What clinical trials is QTORIN rapamycin in?

QTORIN rapamycin has been studied in NCT06653842, a Phase 2 baseline-controlled trial in cutaneous venous malformations; NCT06239480, the Phase 3 SELVA study in microcystic lymphatic malformation; and NCT05180708, a completed Phase 3 randomized, double-blind, vehicle-controlled study in pachyonychia congenita.

Is QTORIN rapamycin the same as QTORIN 3.9% rapamycin anhydrous gel?

Yes. QTORIN rapamycin is also known as QTORIN 3.9% rapamycin anhydrous gel, QTORIN 3.9% rapamycin anhydrous, and QTORIN 3.9% Rapamycin Anhydrous Gel. These names refer to the same investigational topical formulation developed by Palvella Therapeutics.