BirchBioMed’s Idiopathic Pulmonary Fibrosis Treatment Receives Orphan Drug Designation

BirchBioMed, a clinical-stage biopharmaceutical company, announced on Dec. 2, 2025 that FDA has granted the company’s FS2 (kynurenic acid) orphan drug designation (ODD) for the investigational treatment ofidiopathic pulmonary fibrosis (IPF) (1). Identified by the University of British Columbia as a potential candidate for clinical research, FS2 (kynurenic acid) is a topical treatment for scars and the systemic treatment of organ fibrosis.

According to BirchBioMed, IPF is a serious, rare chronic lung disease that results in thickening and stiffening of lung tissues surrounding the alveoli in the lungs, potentially causing permanent scarring of the lungs and making breathing difficult. FS2 (kynurenic acid) demonstrated scar prevention during preclinical trials. It also showed a breakdown and reduction of existing scars externally and internally that were the result of injury, surgery, or disease (1).

“A subsequent Phase II randomized double-blind clinical trial comparing the safety and efficacy of topical FS2 cream demonstrated superior, statistically significant efficacy on mature keloid scars as compared not only to the vehicle control but to a market leading scar cream,” the company stated in the press release (1). “FS2 is unique in its therapeutic category as it targets scarring on the molecular level.”

Why is orphan drug designation important?

FDA’s orphan drug designation promotes development of treatments for rare diseases that impact fewer than 200,000 people in the United States by granting companies market exclusivity for seven years post approval, exemption from user fees, tax credits for qualified clinical testing, and more (2). IPF impacts fewer than three million to five million people in the world, and it does not currently have a cure (1).

"Being granted orphan drug designation by [FDA] is a pivotal milestone in BirchBioMed's development of FS2 for the treatment of IPF, a debilitating and fatal disease that has no more than a 5-year life expectancy after diagnosis," Mark S. Miller, chairman and CEO of BirchBioMed, said in the press release (1). "[FDA's] designation provides BirchBioMed with development and commercial capabilities to address this high, unmet medical need."

"This ODD 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,” Carlos Camozzi,chief medical officer, BirchBioMed, stated in the release (1). “BirchBioMed is seeking regulatory approval to begin clinical trials on the systemic use of FS2 for the treatment of organ fibrosis, beginning with IPF early next year. 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."

What more is happening in the rare diseases space?

Pharmaceutical Technology®’s November 2025 Drug Solutions Podcast gathered industry experts to discuss novel modalities for the treatment of rare diseases, including advancements in oncology (3). Listen to Sarah Hein, PhD, co-founder and CEO, March Biosciences; Joe Katakowski, PhD, director of research for the RTW Foundation; and Dan Williams, PhD, co-founder, executive director, and CEO, SynaptixBio, discuss the role of patients’ families in accelerating R&D for these rare conditions in the podcast at PharmTech.com.

For more on orphan drugs, be sure to read the article by Hibreniguss Terefe, director, Product Development Somerset at Ardena, which outlines common chemistry, manufacturing, and controls challenges that steal momentum and create costly detours in the development or orphan drugs (4).

References

1. BirchBioMed. U.S. FDA Grants BirchBioMed Orphan Drug Designation for FS2 in the Treatment of Idiopathic Pulmonary Fibrosis. Press Release. Dec. 2, 2025.
2. FDA. Designating an Orphan Product: Drugs and Biological Products. FDA.gov. https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products (accessed Dec. 2, 2025).
3. Lavery, P. Drug Solutions Podcast: Innovations in Orpah Drugs for Rare Diseases. PharmTech.com. Nov. 27, 2025. https://www.pharmtech.com/view/drug-solutions-podcast-innovations-in-orphan-drugs-for-rare-diseases
4. Terefe, H. The Push for Momentum, Not Mistakes in Orphan Drug Development. PharmTech.com. Oct. 15, 2025. https://www.pharmtech.com/view/the-push-for-momentum-not-mistakes-in-orphan-drug-development