RARE Daily

FDA Places Clinical Hold on Pharvaris HAE Clinical Trials in the United States

August 22, 2022

Pharvaris said the U.S. Food and Drug Administration verbally informed the company that, based on its review of nonclinical data, the agency is placing a clinical hold on the clinical trials of PHA121 in the United States under two Pharvaris Investigational New Drug applications for the treatment of hereditary angioedema attacks.

Photo: Berndt Modig, chief executive officer of Pharvaris

The FDA indicated it will provide a formal clinical hold letter to Pharvaris in approximately 30 days. The company plans to provide additional updates following interactions with the FDA.

“We are fully committed to working closely with the FDA to address the agency’s concerns,” said Berndt Modig, chief executive officer of Pharvaris. “Pharvaris remains dedicated to providing new therapeutic choices for the treatment of HAE and is working diligently to bring PHA121 to people living with HAE.”

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 an estimated 1 in 50,000 people worldwide and 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.

Author: Rare Daily Staff

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