Investigational drugs developed by Korean pharmaceutical companies are increasingly winning the U.S. Food and Drug Administration’s orphan drug status.
Daewoong Pharmaceutical said on Thursday the FDA granted an orphan drug designation (ODD) to DWN12088, a treatment candidate for idiopathic pulmonary fibrosis (IPF). IPF is an interstitial lung disease, causing the lungs to harden and lose their function gradually. The intractable disease has a five-year survival rate of less than 40 percent.
Four Korean experimental agents have earned FDA’s ODD in the first eight months of 2019.
In January, Bridge Biotherapeutics became the first Korean firm to win FDA’s ODD for its IPF treatment candidate BBT-877 this year.
Genexine’s idiopathic lymphocyte syndrome treatment GX-17 was the next to obtain the status in April, and Hanmi Pharmaceutical’s short bowel syndrome drug HM15912, in May.
FDA’s ODD provides a variety of benefits in approval and sales. The benefits include tax exemptions for development cost, exemption of license application fees, the right to apply for Priority Review to shorten the period of the FDA review, and seven-year monopoly after the market release.
As the drug authorities are encouraging orphan drug development worldwide, the orphan drug market is expected to grow rapidly, observers said.
According to a report on the orphan drug market and R&D status released by Korea Health Industry Development Institute, the global market of orphan drugs is expected to grow 11.3 percent a year from $125 billion in 2017 to $262 billion in 2024.
The growth rate will be more than twice that of the prescription drug market at 5.3 percent, and orphan drugs will account for one-fifth of all prescription medicines in 2024, the report said.
Developers of orphan drugs are gaining investors’ much attention, too. Last year, most of the multinational pharmaceutical firms’ M&As targeted companies specializing in orphan drug development.
Bioverative, developer of hemophilia and rare blood disease treatments, Juno Therapeutics, developer of Chimeric antigen receptor T cell (CAR-T) therapy, and Avesis, a clinical-stage gene therapy firm, have all signed M&A deals with multinational drugmakers, respectively.
Anticipation for Korean orphan drugs has increased, with the rising number of local drugs obtaining FDA’s ODD.
The number of Korean treatment candidates winning FDA’s ODD surged from two in 2015 and three in 2016 to 16 in 2018.
In total, 41 Korean drugs gained FDA’s ODD as of Aug. 14, 2019.
Bridge Biotherapeutics was able to reach a massive 1 trillion won ($825 million) licensing-out deal with Boehringer Ingelheim in July thanks to BBT-877’s ODD obtained in January.
“ODD does not guarantee a new drug’s success, but it provides many benefits in license approval because there is no treatment for rare diseases,” an industry executive said. “ODD gives favor in a licensing deal, so there will be more companies trying to develop an orphan drug.”