Pharvaris’ hereditary angioedema medication reduces attacks in the wake of the FDA hold

Pharvaris has reported a significant breakthrough in the treatment of hereditary angioedema (HAE), a genetic disorder characterized by recurrent severe swelling. The company announced that its prophylactic oral medication, deucrictibant, exhibited a significant 84.5% decrease in monthly attacks in a phase 2 clinical trial. This result represents a remarkable improvement for the drug, especially considering it was subject to a clinical hold imposed by the FDA last year.

The latest development involves the immediate-release capsule formulation of deucrictibant, which successfully met the primary endpoint in the mid-stage CHAPTER-1 trial. The trial aimed to assess whether oral therapy could function as a long-term prophylaxis against angioedema attacks for individuals with type 1 and type 2 of the condition. Over 12 weeks, 34 patients were randomly assigned to receive one of two doses or a placebo.

The primary endpoint measured the number of confirmed HAE attacks during the treatment duration. The 40 mg daily dose of deucrictibant demonstrated an impressive 84.5% reduction in attacks compared to the placebo.

Dr. Peng Lu, Chief Medical Officer at Pharvaris, emphasized that the results provide compelling evidence that deucrictibant can alleviate the treatment burden for HAE patients. The hallmark of the condition is the repeated occurrence of intense swelling in the limbs, face, intestinal tract, and airways.

Principal investigator Dr. Marc A. Riedl emphasized the importance of these results. He noted that the CHAPTER-1 data represent a crucial advancement in HAE treatment, meeting the community’s demand for highly effective, well-tolerated, and less burdensome therapies.

Beyond the primary endpoint, the therapy exhibited positive effects on secondary endpoints, including a decrease in attack severity and a reduction in the number of attacks requiring on-demand medication. The trials showed that deucrictibant was well-tolerated, with no reported serious adverse events, severe treatment-emergent adverse events, or events leading to discontinuation.

Pharvaris did not provide specific details about the future trajectory of deucrictibant but announced intentions to submit the results to the FDA. This clinical success follows the FDA’s lift of the hold on deucrictibant’s use as an on-demand treatment in the United States, which required Pharvaris to complete a 26-week rodent toxicology study. The company continued its research efforts globally, exploring two oral versions of deucrictibant for acute attack treatment and prophylaxis, respectively—an immediate-release capsule and an extended-release pill undergoing phase 1 testing.

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