IPF therapy candidate bersiporocin granted orphan drug status in EU
Oral medication now being tested in Phase 2 clinical trial in US, Korea
Bersiporocin, an oral therapy for idiopathic pulmonary fibrosis (IPF) now being tested in a Phase 2 clinical trial, has been granted orphan drug status in Europe by the European Medicines Agency (EMA).
The EMA gives this designation to treatments that have the potential to improve care for people with rare diseases, defined as conditions affecting fewer than 5 of every 10,000 people in the European Union. Orphan drug status is meant to incentivize companies to develop treatments for rare diseases: Here, it confers multiple benefits to bersiporocin’s developer, Daewoong Pharmaceutical, including a guarantee of 10 years of market exclusivity if the therapy is ultimately approved in the EU.
The U.S. Food and Drug Administration (FDA) had granted bersiporocin orphan drug status in 2019. In 2022, the FDA also awarded the experimental therapy fast track designation, which aims to speed the development of treatments with the potential to improve care for serious conditions.
The news was cheered by Daewoong, which stated in a company press release that the drug’s safety was demonstrated in a Phase 1 study.
“With bersiporocin’s global recognition and its clinical advancements, we are more committed than ever to delivering this promising treatment to IPF patients, potentially transforming the landscape of this challenging disease,” said Seng-ho Jeon, Daewoong’s CEO.
Bersiporocin already awarded orphan drug status in US
IPF is characterized by fibrosis, or scarring, in the lungs, compromising respiratory function. Scar tissue is mainly made up of a protein called collagen. Bersiporocin, previously known as DWN12088, is designed to reduce the production of collagen, thereby lessening scarring.
Daewoong has already completed Phase 1 clinical trials that evaluated the safety profile and pharmacological properties of bersiporocin in more than 160 healthy people. Those trials took place in Korea and Australia, according to the release.
The company now is sponsoring a Phase 2 trial (NCT05389215) to further test the therapy.
Launched in 2022, that trial is expected to wrap up later this year.
Underway at about 30 study sites in Korea and the U.S., it has an estimated enrollment of 102 adults with IPF, all ages 40 and older. The first participant was dosed in early 2023.
Participants in the trial are randomly assigned to take a tablet of bersiporocin or a placebo twice daily for about six months. The study’s main goals are to assess the safety of bersiporocin in people with IPF and to evaluate how the therapy affects the decline in forced vital capacity, a common lung function measure that indicates how much air a person can forcefully exhale after taking a deep breath.