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Our Pipeline

Leading the way for a new era in the treatment of rare genetic diseases.

Preclinical
Phase 1
Phase 2
Phase 3

QTORINTM rapamycin 3.9%

Microcystic Lymphatic Malformations

QTORINTM rapamycin 3.9%

Gorlin Syndrome/BCCs

QTORINTM rapamycin 3.9%

Other Vascular Malformations

PTX-014

Undisclosed Rare Disease
QTORINTM rapamycin 3.9%
Phase 3
Microcystic Lymphatic Malformations
QTORINTM rapamycin 3.9%
Phase 2b
Gorlin Syndrome/BCCs
QTORINTM rapamycin 3.9%
Preclinical
Other Vascular Malformations
PTX-014
Preclinical
Undisclosed Rare Disease

Our Programs

Microcystic LM

Microcystic LM

Microcystic Lymphatic Malformations (Micro LM) is a rare genetic disease of the lymphatic system characterized by abnormal vessels or cysts that leads to chronic lymphorrhea, bleeding, pain, and a risk of severe life-threatening infections. There are no FDA approved therapies. Palvella has successfully completed a Phase 2 clinical study for QTORIN™ rapamycin 3.9%, for people with this disease. Palvella has been awarded FDA Breakthrough Therapy Designation, FDA Orphan Drug, and Fast Track Designation for this program.

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Gorlin Syndrome

Gorlin Syndrome

Gorlin syndrome is a serious rare genetic disease where people may develop thousands of basal cell carcinomas, a form of cancerous skin tumors. There are no FDA approved therapies. Palvella is conducting a phase 2b clinical study of QTORIN™ rapamycin 3.9% for people with Gorlin syndrome, has been awarded FDA Fast Track designation.

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Other Vascular Malformations

Other Vascular Malformations

Palvella is continuing research into other mTOR-driven diseases, building on our clinical experience with QTORIN Rapamycin.

We’re uncovering more therapies every day. Stay updated.