Palvella Therapeutics Announces Oral Presentation at 15th World Congress of Pediatric Dermatology
Wayne, PA, April 2, 2025 – Palvella Therapeutics, Inc. (Palvella or “the Company”), a clinical-stage biopharmaceutical company dedicated to developing and commercializing innovative therapies for serious, rare genetic skin diseases, is excited to announce an upcoming oral presentation at the 15th World Congress of Pediatric Dermatology. The event will take place from April 8-11, 2025, in Buenos Aires, Argentina.
QTORIN™ rapamycin 3.9% Anhydrous Gel for Microcystic Lymphatic Malformations
The oral presentation will focus on the results of SELVA, a 24-week, Phase 3, single-arm, baseline-controlled clinical trial evaluating the efficacy and safety of QTORIN™ rapamycin 3.9% anhydrous gel for the treatment of microcystic lymphatic malformations (MCMs). MCMs are a type of rare, chronic, and often debilitating skin condition with no approved therapies in the U.S.
Study Design and Key Findings
SELVA enrolled 15 patients with MCMs who received topical QTORIN™ rapamycin 3.9% anhydrous gel twice daily for 24 weeks. The primary endpoint was the percentage change in the lesion size area from baseline to week 24, as assessed by independent blinded investigators using validated visual assessment methods. Key findings from the study include:
- A significant reduction in lesion size area was observed in 11 out of 15 patients (73.3%), with a mean reduction of 43.8%.
- All 15 patients reported improvement in their symptoms, as measured using the Patient and Observer Global Assessment of Efficacy (GOE) scale.
- The treatment was generally well-tolerated, with no serious adverse events reported.
What Does This Mean for Patients?
For patients suffering from microcystic lymphatic malformations, the results of the SELVA trial offer hope for an effective treatment option. Topical application of QTORIN™ rapamycin 3.9% anhydrous gel could lead to significant reductions in lesion size and improvement in symptoms. As the study progresses and more data becomes available, healthcare providers may be able to offer this treatment to their patients, providing relief from the physical and emotional challenges associated with MCMs.
Global Impact of QTORIN™ rapamycin 3.9% Anhydrous Gel
The potential impact of QTORIN™ rapamycin 3.9% anhydrous gel goes beyond the immediate benefits for patients with microcystic lymphatic malformations. The development of effective treatments for rare genetic skin diseases is a significant step forward in the field of dermatology and biopharmaceuticals. By addressing unmet medical needs and filling gaps in the current therapeutic landscape, Palvella Therapeutics and other innovative companies are contributing to improved patient outcomes and advancing the overall understanding of these conditions.
Conclusion
Palvella Therapeutics’ upcoming oral presentation at the 15th World Congress of Pediatric Dermatology marks an important milestone in the development of QTORIN™ rapamycin 3.9% anhydrous gel as a potential treatment for microcystic lymphatic malformations. This Phase 3 trial has shown promising results, with significant reductions in lesion size and improvements in symptoms for most patients. The implications of these findings extend beyond individual patients, as the successful development and commercialization of QTORIN™ rapamycin 3.9% anhydrous gel could pave the way for better treatments and a deeper understanding of rare genetic skin diseases. As the world continues to grapple with the challenges of developing effective therapies for these conditions, the progress made by Palvella Therapeutics and others in the field offers a glimmer of hope for the millions of patients affected by rare and debilitating skin diseases.
Stay tuned for further updates on the SELVA study and the ongoing development of QTORIN™ rapamycin 3.9% anhydrous gel as a potential treatment for microcystic lymphatic malformations.
