South Korea’s Daewoong Pharmaceutical said it received U.S. Food and Drug Administration (FDA) orphan drug designation (ODD) for DWN12088, an investigational drug to treat patients with idiopathic pulmonary fibrosis (IPF).
The FDA ODD certification facilitates the development and approval of drugs for rare or refractory diseases by exempting the manufacturer from license review fees and enabling an option for priority review and seven-year monopoly after marketing authorization.
The oral IPF therapy selectively inhibits Prolyl-tRNA Synthetase (PRF) protein activity and excessive production of collagen, the cause of pulmonary fibrosis.
Preclinical studies demonstrated the efficacy and safety of the drug compared to existing therapies, and the company already submitted a phase 1 trial plan to the Human Research Ethics Committee (HREC) in Australia.
IPF is an interstitial lung disease, where pulmonary tissue becomes thickened and scarred over a period of time without a known cause. This rare disease has a five-year survival rate of less than 40 percent.
DWN12088 is the industry’s first IPF treatment that targets PRS and the ODD will help the company develop the treatment faster for patients suffering from IPF, said Daewoong Pharm chief executive Jeon Seng-ho.
Daewoong Pharm said it will focus R&D resources to better understand the biology of fibrosis which occurs in different organs including the lung, skin, kidney and liver.
In February, the company was named to receive research grants for DWN12088 from the government.
Shares of Daewoong Pharmaceutical closed Tuesday up 0.31 percent at 163,500 won ($134).
By Kim Byung-ho and Minu Kim
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