Palvella Therapeutics is set to expand its 24-week Phase III SELVA trial to include subjects aged three to five years old for evaluating Qtorin 3.9% rapamycin anhydrous gel, a treatment for microcystic lymphatic malformations (LMs).
The US Food and Drug Administration (FDA) has communicated its acceptance of the trial’s expansion, which previously only included subjects aged six and older.
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Palvella is enrolling nearly 40 subjects currently in this single-arm, baseline-controlled trial.
The gel has received breakthrough therapy, fast track, and orphan drug designations from the US regulator, for treating this debilitating genetic disease.
Additionally, the FDA’s Office of Orphan Products Development is supporting the study with a grant of up to $2.6m.
SELVA study principal investigator and Stanford University Dermatology and Pediatrics professor Joyce Teng said: “Patients with microcystic LMs often have lymphorrhoea, bleeding, infection, pain, and disfigurement which may lead to difficulty with physical activities as well as more significant complications like cellulitis and hospitalisation.
“Early intervention is essential to minimise disease burden during children’s development which is why I am so excited by the opportunity Qtorin rapamycin presents to the younger paediatric population.”
Qtorin rapamycin, which is still under investigation, has not yet been approved by the FDA or any other regulatory agency.
Palvella is also evaluating this investigational gel in a Phase II trial, TOIVA, for cutaneous venous malformations.
The company is developing a pipeline of product candidates based on its patented Qtorin platform, targeting serious, rare genetic skin conditions.
In the US alone, there are more than 30,000 Microcystic LMs diagnosed patients. It can be caused by dysregulation of the phosphatidylinositol 3-kinase (PI3K)/mammalian target of rapamycin (mTOR) pathway.
In December 2023, the company entered a partnership with Ligand Pharmaceuticals for the Phase III trial of topical Qtorin rapamycin aimed at treating microcystic LMs.
