Pharvaris N.V. recently announced encouraging findings from its RAPIDe-3 Phase 3 clinical trial, which investigated the efficacy of deucrictibant, an orally administered drug, for the acute management of hereditary angioedema (HAE) attacks. The trial's results highlight deucrictibant's potential to provide swift and effective relief to individuals suffering from these rare, severe swelling episodes. These positive outcomes are poised to form the foundation for upcoming marketing authorization applications, with submissions anticipated to commence in the first half of 2026.
Details of the Clinical Breakthrough
On December 3, 2025, Pharvaris N.V. unveiled the topline data from its RAPIDe-3 Phase 3 study, focusing on deucrictibant's role in the on-demand treatment of hereditary angioedema attacks. The trial involved 134 participants aged 12 and above, all of whom experience HAE, a rare genetic condition characterized by recurrent and severe swelling in various body parts, including the skin, face, limbs, and internal organs. The study's primary objective was to assess the immediate-release (IR) capsule of deucrictibant in providing rapid relief.
The results revealed a remarkable improvement in the median time to the onset of treatment response when deucrictibant was compared to a placebo. Specifically, the time to noticeable symptom relief, as measured by PGI-C3, was reduced to 1.28 hours with deucrictibant, a significant improvement over the more than 12 hours observed in the placebo group. Furthermore, the median time to the end of attack progression was dramatically shortened to 17.47 minutes with the active drug, compared to 228.67 minutes for placebo. Patients receiving deucrictibant also experienced a quicker median time to substantial symptom relief and earlier complete symptom resolution. A significant majority of attacks, 83.0%, were managed with a single dose of deucrictibant IR, and 93.2% of treated attacks did not require any rescue medication. The drug was also well-tolerated, with no serious treatment-related adverse events reported and no participants withdrawing due to side effects. An ongoing open-label extension study, RAPIDe-2 Part B, continues to explore deucrictibant for HAE attack treatment.
In the broader competitive landscape, other advancements have recently emerged for HAE management. In August, the U.S. Food and Drug Administration (FDA) approved Ionis Pharmaceuticals, Inc.'s Dawnzera (donidalorsen) for HAE attack prevention in patients aged 12 and older, demonstrating an 81% reduction in monthly attack rates. Additionally, in July, KalVista Pharmaceuticals Inc. received FDA approval for Ekterly (sebetralstat), marking it as the first and only oral on-demand treatment for acute HAE attacks in the same age group.
The successful trial outcomes for deucrictibant underscore a pivotal moment in the treatment of HAE. As a medical professional, these findings bring immense hope to patients who often face unpredictable and debilitating swelling episodes. The rapid action of deucrictibant, coupled with its favorable tolerability profile, suggests a significant leap forward in improving the quality of life for those afflicted by this rare condition. The competition within the pharmaceutical sector to address HAE is also commendable, indicating a collective commitment to advancing therapeutic options and offering patients a diverse array of effective treatments.