FDA lifts remaining hold on Pharvaris’ HAE drug, clearing path to phase 3

Pharvaris’ preventative oral medicine for hereditary angioedema (HAE) has had its remaining clinical hold lifted by the FDA weeks after the treatment proved its worth in a phase 2 study.

Two oral versions of deucrictibant—a capsule for rapid onset to treat acute attacks and an extended-release pill for prophylaxis—are being developed by Pharvaris with the goal of reducing the treatment burden for patients with HAE.

Both programs were placed on hold by the FDA back in August 2022, with the Switerzland-based company later hinting that the cause was related the safety concerns tied to preclinical data. The biotech later pledged to conduct a 26-week rodent toxicology study to resolve the holds, and by June 2023 the FDA had agreed to release the investigational new drug application for deucrictibant for the on-demand treatment of HAE.

However, the trial of deucrictibant for prophylaxis had remained on hold, with Pharvaris saying at the time that it was “still progressing” the rodent study with the aim of submitting data by the end of 2023.

Now, those efforts appear to have paid off. The biotech announced this morning that the FDA had indeed lifted the hold after reviewing the data from the 26-week toxicology study.

“The lift of the clinical hold in the U.S. enables us to progress the global development of deucrictibant for long-term prophylaxis, including resuming the open-label portion of CHAPTER-1, our phase 2 proof-of-concept study of deucrictibant for the prevention of HAE attacks, in the U.S.,” CEO Berndt Modig said in the Jan. 22 release.

“We are pleased to have worked collaboratively with the FDA to address the requests of the agency with the submission of additional nonclinical data, and we appreciate the agency’s comments and recommendations regarding study conduct,” Modig added. “We will request an end-of-phase 2 meeting with the FDA to align on key elements of CHAPTER-3, the anticipated global phase 3 study of deucrictibant extended-release tablets for the prophylactic treatment of HAE attacks.”

The FDA’s decision comes the month after Pharvaris posted data from the phase 2 CHAPTER-1 trial outside the U.S. showing that patients with HAE taking deucrictibant experienced an 84.5% reduction in monthly attacks.