Stockholm, 15th September 2026
Oxcia AB today announces that the company’s drug candidate OXC-201 has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of idiopathic pulmonary fibrosis (IPF). The announcement follows the earlier positive decision from the European Medicines Agency (EMA) only a few weeks ago.
The FDA’s decision is an important milestone for Oxcia’s OXC-201 program and underscores the significant medical need for new treatment options for patients with IPF, a serious, progressive and life-threatening lung disease where current treatments primarily slow disease progression but do not cure the disease or restore lung function.
The U.S. market currently represents approximately 80% of the global market.
Orphan Drug Designation from the FDA may provide several important benefits during drug development, including regulatory support, fee reductions, tax credits for qualified clinical trial costs and up to seven years of market exclusivity in the United States following potential marketing approval. Together with the previous ODD status from the EMA, this strengthens OXC-201’s regulatory and commercial position ahead of upcoming clinical development.
“That OXC-201 has now also been granted Orphan Drug Designation by the FDA is a very important recognition of the program’s potential. The U.S. market is by far the largest market for IPF. ODD status strengthens our ability to develop OXC-201 efficiently and positions the candidate as a potentially differentiated treatment in an area of significant medical need,” says Ulrika Warpman Berglund, CEO of Oxcia AB.
OXC-201 – a completely new approach to IPF
OXC-201 is a first-in-class oral OGG1 inhibitor being developed for the treatment of IPF. By targeting central mechanisms behind inflammation and fibrosis, for example effects on oxidative stress, OXC-201 has the potential to influence the underlying processes of the disease. Preclinical studies have shown that OXC-201 affects both inflammatory and fibrotic disease markers, produces clear tissue effects and improves lung function in disease models. Early data also indicates the potential to reduce cough, one of the most troublesome symptoms of IPF, as well as a favorable tolerability profile.
Clinical studies of OXC-201 are expected to begin in 2027, with the aim of establishing safety, biomarker response and early clinical signals that can support continued development and future partnerships.
About idiopathic pulmonary fibrosis
Idiopathic pulmonary fibrosis is a rare, chronic and progressive lung disease that leads to scarring of the lung tissue. The disease causes a gradual decline in lung function, shortness of breath and often severe cough. The prognosis is serious, and there remains a significant need for new treatments that can provide better efficacy, improved quality of life and better tolerability.
About Oxcia
Oxcia AB is a clinical-stage biotechnology company developing first-in-class treatments based on the company’s O2-DDR platform, with a focus on oxidative stress, oxidative DNA damage and DNA damage response. Using this platform, Oxcia can address driving factors and root causes behind many diseases and develop innovative treatments.
To date, the platform has generated two first-in-class drug candidates: OXC-101, currently in clinical development for acute myeloid leukemia, and OXC-201, a novel treatment for idiopathic pulmonary fibrosis. Both projects have received Orphan Drug Designation from both the FDA and the EMA. Oxcia’s goal is to develop transformative treatments for diseases with significant medical need and thereby make life less short.
For more information contact:
Ulrika Warpman Berglund, CEO, Oxcia AB (publ)
Telephone: +46 (0) 73 270 9605
ulrika.warpmanberglund@oxcia.com