Palvella Therapeutics has received a grant of up to $2.6m from the US Food and Drug Administration (FDA) Office of Orphan Products Development to support the Phase III SELVA trial of QTORIN 3.9% rapamycin anhydrous gel for microcystic lymphatic malformations (LMs).
Palvella’s Phase III trial is one of only seven new studies to receive the funding out of 51 grant applications received for the fiscal year 2024.
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The FDA’s Orphan Products Grants Program provides grants every year to foster the development of medicines that could address the unmet needs of patients with rare diseases or conditions.
Each grant application undergoes a rigorous review for scientific and technical merit by an independent panel of rare disease and regulatory experts.
Consultation with the relevant FDA review division may also occur to assess whether the proposed study will yield data that could contribute to product approval.
Since its inception, the Orphan Products Grants Program funded clinical trials that have led to the approval of more than 85 products.
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By GlobalDataPalvella initiated the SELVA trial of QTORIN rapamycin for microcystic LMs in the third quarter of 2024.
This 24-week, single-arm, baseline-controlled clinical trial’s primary and key secondary endpoints are based on clinician-reported outcomes.
It is expected to enrol a total of 40 subjects at various vascular anomaly centres throughout the US.
Palvella Therapeutics founder and CEO Wes Kaupinen said: “We are pleased to receive this grant from the FDA which will support the advancement of SELVA, our ongoing single-arm, baseline-controlled Phase III trial of QTORIN rapamycin for the treatment of microcystic LMs.
“We believe this grant underscores the high unmet medical need in this serious, rare and chronically debilitating genetic disease, the scientific rationale for targeted inhibition of the causative PI3K/mTOR pathway, and QTORIN rapamycin’s potential to be the first approved therapy and standard of care in the US for microcystic LMs.”
In December 2023, Palvella partnered with Ligand Pharmaceuticals for the Phase III trial of topical Qtorin rapamycin to treat microcystic LMs.
