WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) is preparing for a potential first-half 2027 U.S. launch of its experimental treatment for microcystic lymphatic malformations after completing its FDA application and securing a third year of federal funding for the late-stage program.
The Food and Drug Administration’s Office of Orphan Products Development awarded another year of grant funding to support Palvella’s Phase 3 SELVA trial and continuing open-label extension study of QTORIN 3.9% rapamycin anhydrous gel.
The award followed an FDA review of the Wayne-based biotechnology company’s annual progress report, which included results from SELVA.
Palvella completed its New Drug Application for QTORIN rapamycin in August after submitting portions of the application on a rolling basis. The company is now advancing commercial preparations for a U.S. launch in the first half of 2027, contingent on FDA approval.
QTORIN rapamycin is being developed for microcystic lymphatic malformations, a rare condition for which Palvella says there are currently no FDA-approved therapies. The company estimates more than 30,000 pediatric and adult patients in the U.S. have the condition.
The regulatory application follows positive results from SELVA reported in February. The Phase 3 trial met its primary endpoint, a prespecified key secondary endpoint and four additional secondary efficacy endpoints.
All six efficacy endpoints reached statistical significance at p<0.001, according to Palvella.
“Following positive Phase 3 SELVA results and completion of our NDA submission, we are advancing commercial readiness for a potential U.S. launch in the first half of 2027, if approved,” founder and Chief Executive Officer Wes Kaupinen said.
QTORIN rapamycin has received Breakthrough Therapy, Fast Track and Orphan Drug designations from the FDA.
SELVA was one of seven new clinical trials selected from 51 applications for grants through the FDA’s Orphan Products Grants Program in fiscal 2024. It was the only Phase 3 trial awarded funding that year.
The federal program uses independent scientific and technical review to select projects targeting rare diseases. Since its inception, funded clinical trials have contributed to FDA approval of more than 85 products for rare diseases.
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