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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%

Pachyonychia Congenita

QTORINTM rapamycin 3.9%

Microcystic Lymphatic Malformations

QTORINTM rapamycin 3.9%

Gorlin Syndrome

QTORINTM rapamycin 3.9%

Non-syndromic High-frequency Basal Cell Carcinomas

PTX-014

Undisclosed Rare Disease
QTORINTM rapamycin 3.9%
Phase 3
Pachyonychia Congenita
QTORINTM rapamycin 3.9%
Phase 2
Microcystic Lymphatic Malformations
QTORINTM rapamycin 3.9%
Phase 2b
Gorlin Syndrome
QTORINTM rapamycin 3.9%
Preclinical
Non-syndromic High-frequency Basal Cell Carcinomas
PTX-014
Preclinical
Undisclosed Rare Disease

Our Programs

Pachyonychia Congenita (PC)

Pachyonychia Congenita (PC)

PC is a chronically debilitating rare genetic disease resulting in lifelong limited mobility and severe pain. There are no FDA approved therapies. Our phase 3 clinical study of QTORIN™ rapamycin 3.9% for people with PC is currently enrolling. Palvella has been awarded both 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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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 is conducting a phase 2 clinical study for QTORIN™ rapamycin 3.9%, for people with this disease. Palvella has been awarded both FDA Orphan Drug and Fast Track designation for this program.

Microcystic Lymphatic Malformations

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