Pharvaris AAE-C1INH burden study published in Frontiers Immunology
Pharvaris has announced publication of the first in-depth qualitative study characterising the patient experience of acquired angioedema due to C1 inhibitor deficiency (AAE-C1INH), with results appearing in Frontiers in Immunology. The Swiss-based, Nasdaq-listed company said findings from the research directly shaped the design and endpoint selection of its ongoing Phase 3 CREAATE study (NCT07266805), which is investigating deucrictibant for both prophylaxis and on-demand treatment of AAE-C1INH attacks.
The study drew on interviews with patients diagnosed with AAE-C1INH, capturing disease manifestations, daily-life impact, and perspectives on treatment benefit. Participants reported frequent, painful swelling attacks that disrupted routine activity, employment, and travel, often preceded by prolonged periods of misdiagnosis and emergency-care episodes. All interviewees relied on off-label therapies, underlining the absence of any approved treatment in this indication.
Endpoint validation
A central objective of the study was to assess whether established patient-reported outcome (PRO) instruments are meaningful in the AAE-C1INH population. The researchers evaluated three tools: the Patient Global Impression of Change (PGI-C), Patient Global Impression of Severity (PGI-S), and Patient Global Assessment (PGA). Results indicated that all three are relevant and interpretable for this patient group. Notably, a PGI-C rating of "better" was the threshold most consistently identified as meaningful by participants at time points up to four hours post-treatment, a clinically actionable anchor for future trial readouts.
Danny M. Cohn, principal investigator in CREAATE and a physician at Amsterdam UMC, said: "Despite this, there are currently no therapies approved for the prevention or treatment of AAE-C1INH attacks. This study is an important step forward, capturing the patient experience in a rigorous way and helping to ensure that clinical studies evaluate outcomes that are truly meaningful to patients."
Peng Lu, President of Pharvaris, noted the research aligns with FDA guidance on capturing patient experience in drug development, describing the publication as strengthening the scientific foundation for evaluating treatment benefit in what the company calls an underserved population.
Market context and regulatory read-across
AAE-C1INH is an ultra-rare condition distinct from hereditary angioedema (HAE), where the competitive landscape is already well-populated. Approved HAE therapies include plasma-derived C1 inhibitor concentrates, the subcutaneous monoclonal antibody lanadelumab, and the oral kallikrein inhibitor berotralstat. Pharvaris is seeking to differentiate deucrictibant as an oral bradykinin B2 receptor antagonist with what it describes as injectable-like efficacy, a positioning that, if borne out in Phase 3, could address both HAE and the currently treatment-void AAE-C1INH segment.
The FDA's increasing emphasis on patient-centred outcome measures in rare-disease development, reflected in multiple recent guidance documents, means that a rigorously validated PRO framework can ease regulatory dialogue during a new drug application review. For Pharvaris, which has a separate NDA under review for deucrictibant in on-demand HAE treatment, the publication also serves to signal methodological rigour across its broader pipeline. Investors and clinicians will look for CREAATE recruitment updates and interim readout timelines as the next substantive indicators of the programme's progress.