FDA grants orphan drug designation to PAH treatment

The FDA granted orphan drug designation to ZMA001 as a treatment for patients with pulmonary arterial hypertension, according to a press release from Zymedi.

As a first-in-class human monoclonal antibody, the release states that ZMA001 (Zymedi) is capable of blocking inflammation-inducing macrophages from getting into the lungs.

ZMA001 has already displayed “superior efficacy” vs. existing drugs in animal model studies and works well with existing medications, according to the release.

In February, Zymedi reported that the first individual in the placebo-controlled phase 1 trial received the drug. As indicated in a previous release, the purpose of this trial is to evaluate the safety of ZMA001 through different dosages.

“With the orphan drug designation for ZMA001, we hope to provide a valuable treatment option for PAH patients in need of new therapies,” Nam Hoon Kwon, PhD, chief technology officer of Zymedi, said in the release.

Orphan drug designation is given to treatments that address rare diseases affecting fewer than 200,000 people in the United States. The designation provides assistance in developing drugs, tax credits, exemptions from FDA fees and 7 years of marketing exclusivity.

Reference:

• First in human dosed in clinical trial of ZMA001 for treatment of pulmonary arterial hypertension (PAH). https://www.prnewswire.com/news-releases/first-in-human-dosed-in-clinical-trial-of-zma001-for-treatment-of-pulmonary-arterial-hypertension-pah-302056941.html. Published Feb. 7, 2024. Accessed Aug. 5, 2024.