FDA grants orphan drug status to experimental IPF treatment FS2
Developer Birchbiomed plans to begin first clinical trial next year
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Birchbiomed’s FS2 (kynurenic acid) for the treatment of idiopathic pulmonary fibrosis (IPF).
Orphan drug status is intended to incentivize the development of therapies for rare diseases — defined as those affecting fewer than 200,000 people in the U.S. The designation comes with certain incentives, including tax credits on qualified clinical testing, waiver of certain fees, and seven years of market exclusivity if the therapy is ultimately approved.
“Being granted Orphan Drug Designation by the FDA is a pivotal milestone in BirchBioMed’s development of FS2 for the treatment of IPF,” Mark S. Miller, the company’s chairman and CEO, said in a press release. “The FDA’s designation provides BirchBioMed with development and commercial capabilities to address this high, unmet medical need.”
Pending regulatory clearance, the company plans to begin its first clinical trial of a systemic, or whole-body, formulation of FS2 in people with IPF next year.
“This [orphan drug designation] indicates the scientific value of FS2 in the investigational treatment of IPF and underscores the opportunity for IPF patients to participate in our clinical trials,” said Carlos Camozzi, MD, PhD, Birchbiomed’s chief medical officer.
IPF treatment FS2 promotes breakdown of excess ECM
IPF is a chronic, progressive disease characterized by the production of fibrotic (scar) tissue within the lungs for unknown reasons. Symptoms of IPF include shortness of breath and persistent cough.
In IPF, there is an imbalance between synthesis and degradation in the extracellular matrix (ECM), a network of proteins and other molecules that support cells, along with the abnormal activation of fibroblasts and the excessive production of collagen and fibronectin.
According to Birchbiomed, FS2, along with its precursor, kynurenine, promotes the breakdown of excess ECM by stimulating the production of matrix metalloproteinases. These enzymes help degrade ECM components and curb the formation of collagen and fibronectin. FS2 and kynurenine have also been shown to reduce the proliferation of fibroblasts.
In preclinical studies, FS2 prevented the formation of new scars and reduced existing scars caused by injury, surgery, or disease. The company also noted that a Phase 2 trial showed that a topical FS2 formulation was superior to both a placebo and a comparator available on the market in reducing mature scars.
“As experts in the development of products for scarring and other fibrosis-related disorders, we are laser-focused on FS2’s potential to transform the lives of people suffering from this severe, chronic, deteriorating, irreversible, life-threatening lung disease,” Camozzi said.
About the Author
