QTORINTM rapamycin 3.9% is a novel topical therapy being studied for Pachyonychia Congenita (PC). We expect to begin enrolling a phase 3 study in PC in late 2021.
PC: A Disease of Limited Mobility and Pain
PC is a rare, chronically debilitating and lifelong disease in which mutations of genes responsible for keratin production lead to dysregulated keratinocyte proliferation, increased skin fragility, and impaired skin barrier function on the feet. Affected individuals have difficulty walking, and often require ambulatory aids or crawling.
Palvella estimates that there are 14,000 people with PC diagnosed and treated by Dermatologists and Podiatrists in the US.
The potential for rapamycin in PC was discovered by leading scientists and geneticists in the field of PC research who demonstrated a direct mechanism of action of mTOR inhibitors on translationally repressing the mutant keratin genes which are the root cause of PC.
QTORINTM rapamycin 3.9% has been awarded Fast Track Designation, FDA Orphan Drug Designation, and EMA orphan drug designation for PC.
The VAPAUS Study
We plan to start the VAPAUS study in the second half of 2021. The VAPAUS study is a pivotal phase 3 multicenter clinical study evaluating the safety and efficacy of QTORINTM rapamycin 3.9% in adults with moderate to severe pachyonychia congenita. To stay updated on progress or to inquire about participating in studies for Pachyonychia Congenita, please contact PC Project.
Able and willing to comply with all protocol-required activities
Resources for PC
Pachyonychia Congenita Project
PC Project is the leading patient advocacy group for PC, connecting patients, researchers, and physicians throughout the world in a united effort to help those with Pachyonychia Congenita.
2018 FDA Externally-led Patient-Focused Drug Development Meeting for PC
PC project co-organized an Externally-led Patient Focused Drug Development Meeting (EL-PFDD) for FDA officials on April 6, 2018. Approximately 75 Pachyonychia Congenita (PC) patients and caregivers shared their voices through panel presentations, electronic polling, and moderated audience discussions.
National Organization for Rare Disorders
NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 280 patient organizations members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.
Our Advocacy Work
Collaboration with patients and patient advocacy groups is at the heart of our work, bringing both drive and focus to everything we do.
2019 Pachyonychia Congenita Patient Support Meeting - Boston, MA
2018 London PC Patient Support Meeting
Wes Kaupinen (CEO, Palvella Therapeutics) and Jan Schwartz (Executive Director, PC Project) at the 2018 London PC Patient Support Meeting
FDA Patient-Focused Drug Development Meeting, 2018
We’re uncovering more therapies every day. Stay updated with our progress.