Programs & Research
Explore our current research and clinical trials involving the QTORIN™ platform.
Our Pipeline
Palvella Therapeutics is leading the way in developing new treatments that have the power to change lives for thousands with rare genetic skin diseases.
Microcystic Lymphatic Malformations
Microcystic Lymphatic Malformations (Micro LM) is a rare genetic disease of the lymphatic system characterized by abnormal vessels or cysts that lead to chronic lymphorrhea (abnormal flow of lymph fluid), bleeding, and a risk of severe life-threatening infections.
While most individuals are diagnosed early in life, the disease can become more pronounced as a person ages and goes through puberty, and there are no FDA-approved therapies.
Targeting Micro LM
QTORIN™ rapamycin 3.9% is a novel targeted topical therapy being studied for Microcystic Lymphatic Malformations (Micro LM). QTORIN™ rapamycin 3.9% is designed to target the PI3K/mTOR pathway, which is overactivated in Micro LM. QTORIN™ rapamycin 3.9% topical gel delivers rapamycin deep into the dermis, the area where the disease originates.
Palvella has successfully completed a phase 2 clinical study for people with this disease and has been awarded FDA Breakthrough Therapy Designation, FDA Orphan Drug, and Fast Track Designation for this program.
Interested in enrolling in a clinical study?
Our multicenter phase 2 study evaluating the safety and effectiveness of QTORIN™ rapamycin 3.9% in those with microcystic lymphatic malformations is now complete. Subscribe to our email list to get updates on future clinical trials.
Venous Malformations
While rare, congenital venous malformation (VM) is the most common type of congenital vascular malformation (CVM) comprising up to two-thirds of all CVMs. VMs develop as a result of an error in vascular embryogenesis resulting in the formation of thin-walled, dilated venous channels with sparse, clumped smooth muscle cells.
VMs generally present as localized malformations with variations in age of presentation, lesion size, and location. There are no FDA-approved treatments indicated for VM. The treatment of patients with VMs is medically challenging, and most VMs cannot be completely excised or cured. As such, VMs are managed as a chronic disease with the goal of providing symptomatic relief and minimizing further progression.
Other mTOR-Driven Skin Diseases
Palvella is continuing research into other mTOR-driven diseases, building on our clinical experience with QTORIN™ rapamycin. Subscribe to our email list to get updates on future clinical trials.