Palvella Announces FDA Acceptance and Priority Review of QTORIN™ Rapamycin NDA; February 28, 2027 PDUFA Target Action Date Set
Key Highlights
- ➤FDA accepted Palvella’s NDA for QTORIN rapamycin and granted Priority Review.
- ➤February 28, 2027 PDUFA target action date set for QTORIN rapamycin.
- ➤Phase 3 SELVA achieved statistical significance across all six efficacy endpoints, all p<0.001.
- ➤95% of evaluable participants aged 6 and older improved on the mLM-IGA endpoint.
- ➤Palvella is preparing a potential standalone U.S. launch in the first half of 2027.
Expert Statements
Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics
“FDA acceptance of our NDA and granting of Priority Review represent significant milestones in the development of QTORIN™ rapamycin as a potential first FDA-approved therapy for microcystic lymphatic malformations”
Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics
“Palvella was founded to serve rare disease patients by developing and commercializing first-in-disease therapies that meaningfully improve their lives.”
Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics
“With compelling Phase 3 SELVA results and a February 28, 2027 PDUFA target action date, we believe we are closer than ever to delivering on that mission.”
Wes Kaupinen, Founder and Chief Executive Officer of Palvella Therapeutics
“We have assembled an exceptional team across our medical and commercial organizations as we prepare for a potential U.S. launch and work toward bringing QTORIN™ rapamycin to patients who currently have no FDA-approved therapies.”
QTORIN™ rapamycin could become the first FDA-approved therapy for microcystic lymphatic malformations, a serious, lifelong rare disease affecting an estimated more than 30,000 diagnosed children and adults in the U.S.
Phase 3 SELVA study achieved highly statistically significant results across all six prespecified efficacy endpoints (all p<0.001), with 95% of participants aged 6 years and older who completed the efficacy evaluation period demonstrating improvement on the mLM-IGA primary endpoint
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