EMA Grants Bersiporocin Orphan Drug Designation

Daewoong Pharmaceuticals

The European Medicines Agency (EMA) granted its orphan drug designation to bersiporocin, a medication developed by Daewoong Pharmaceuticals. The drug earned the title for its treatment of idiopathic pulmonary fibrosis (IPF).

Daewoong Pharmaceuticals describes the drug as the world’s first PRS inhibitor for IPF.¹

In a press release, Daewoong Pharmaceutical CEO Seng-ho Jeon said, “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."

IPF is a rare disease and only has about 13 cases per 100,000 people across the entire planet. It is a serious condition and the 5-year survival rate is about 40%. Patients with the condition suffer from a reduction of lung function due to a build-up of collagen in the lungs.

Bersiporocin inhibits prolyl-tRNA synthetase, which reduces the excess collagen production caused by IPF.

The EMA grants the orphan drug designation to drugs and treatments that have the potential treat conditions that effect fewer than five in 10,000 people in the EU. Once drugs receive the designation, they are granted multiple benefits, including guidance on clinical trials, reduced authorization fees, and 10-year market exclusivity (depending upon the drug’s approval).

The EMA is the latest agency to grant the drug orphan drug designation. In May, 2023, Daewoong Pharmaceuticals announced that FDA had granted the designation for the drug’s use in the US. FDA also granted it fast track designation for IPF.

The announcement came alongside the publication of the results of a study that revealed the molecular mechanism of the drug. The results were published in EMBO Molecular Medicine that same month.²

In a press release issued at the time, Daewoong Pharmaceutical’s chief of drug discovery center Joon Seok Park said, “this study will support the unique mode of action behind Bersiporocin as a first-in-class antifibrotic, and encourage the ongoing IPF Phase II clinical studies. We believe Bersiporocin will become the next therapeutic option to patients who are still suffering for this devastating disease."

The corresponding author of the publication, Prof. Sunghoon Kim also said, “Investigating novel target, novel mechanism is crucial in precision medicine and global drug discovery. This study sets an important milestone that essential enzymes like PARS1 can be targeted for drug discovery.”

As part of the latest news regarding Bersiporocin, Daewoong Pharmaceuticals noted that its currents statements on the drug and its capabilities are forward-looking and based on its current understanding of the drug and its efficacy. The drug is still going through testing and the regulatory process for government approvals in various regions. While the drug has completed Phase I trials (conducted in Austria and Korea), Phase II trials are still ongoing and are being held in Korea and the United States.

Source

1. Daewoong Pharmaceutical's Bersiporocin Receives 'Orphan Drug Designation' in Europe to Treat Idiopathic Pulmonary Fibrosis. Daewoong Pharmaceuticals. January 29, 2024. https://www.prnewswire.com/news-releases/daewoong-pharmaceuticals-bersiporocin-receives-orphan-drug-designation-in-europe-to-treat-idiopathic-pulmonary-fibrosis-302046419.html
2. Daewoong Pharmaceutical Publishes Molecular Mechanism of Bersiporocin as an Antifibrotic in EMBO Molecular Medicine. Daewoong Pharmaceuticals. May 25, 2024. Accessed on January 29, 2024. https://www.prnewswire.com/news-releases/daewoong-pharmaceutical-publishes-molecular-mechanism-of-bersiporocin-as-an-antifibrotic-in-embo-molecular-medicine-301834515.html