FDA Removes of Clinical Hold on Pharvaris’ Therapy for Hereditary Angioedema Attacks

Rare Daily Staff

The U.S. Food and Drug Administration has lifted the clinical hold on Pharvaris’ Investigational New Drug application for deucrictibant for the on-demand treatment of hereditary angioedema attacks following review of data from a preplanned interim analysis of the ongoing 26-week nonclinical study.

Pharvaris expects to submit data from the 26-week nonclinical study by the end of 2023 to address the remaining hold on the IND application for deucrictibant for prophylactic treatment of hereditary angiodedema.

Hereditary angioedema (HAE) is a rare genetic disorder that results in recurring attacks of edema, or swelling, in various parts of the body, including the abdomen, face, feet, genitals, hands and throat. The swelling can be debilitating and painful. Attacks that obstruct the airways can cause asphyxiation and are potentially life threatening. HAE attacks may occur very early in childhood. Potentially fatal upper airway angioedema has been reported in patients as young as 3 years old. HAE diagnosis can take an average of 8.4 years after symptom onset. HAE affects is often under recognized, under diagnosed, and under treated. There are currently no long-term prophylactic treatments approved for HAE patients younger than 6 years.

Current therapies are limited by invasive routes of drug administration (injection or infusion), inconvenient dosing regimens, or undesired side effects. Surveys of patients with HAE overwhelmingly show that they seek additional treatment options.

Pharvaris has a platform of novel, potent, and selective B2-receptor antagonist small molecules for the treatment of B2-receptor-mediated diseases. PHA121, the company’s most advanced program, is an oral small molecule antagonist of the bradykinin B2 receptor designed to treat all subtypes of HAE, which the company believes may provide an effective and more convenient way to manage HAE and improve quality of life.

“Our team plans to request an end of phase 2 meeting with the agency and is preparing for RAPIDe-3, our global Phase 3 study of PHVS416 for the on-demand treatment of HAE, to include U.S. sites,” said Peng Lu, chief medical officer of Pharvaris. “Based on current enrollment, we confirm that top-line data from CHAPTER-1, our phase 2 proof-of-concept study of PHVS416 for the prophylactic treatment of HAE, remains on track to be announced by the end of the year.”

Photo: Peng Lu, chief medical officer of Pharvaris