FDA grants orphan drug designation to zelasudil for IPF

The FDA granted orphan drug designation to zelasudil for treating patients with idiopathic pulmonary fibrosis, according to a press release from the drug’s manufacturer.

Zelasudil (RXC007, Redx Pharma) is an oral, selective inhibitor that targets rho-associated coiled-coil containing protein kinase 2 (ROCK2). Along with treating IPF, zelasudil may also be a treatment option for those with other interstitial lung diseases and cancer-associated fibrosis, according to the press release.

The release notes that this orphan drug designation comes after two presentations of in vivo preclinical models in 2022 that demonstrated the “robust anti-fibrotic effects” of the drug.

To evaluate the safety, tolerability and efficacy of zelasudil in patients with IPF, the manufacturer is currently conducting a 12-week phase 2a multicohort, randomized, double-blind, placebo-controlled dose-ranging study and hopes to present topline data in the first quarter of 2024, according to an earlier press release.

The release also details planned assessments of target engagement and fibrosis modification over 28 days of treatment.

“We are delighted that the FDA has recognized the potential of zelasudil for the treatment of IPF and granted orphan drug designation,” Jane Robertson, MD, MBBS, chief medical officer of Redx Pharma, said in the release. “Selectively targeting ROCK2 is an exciting, novel approach which could provide a new treatment option for patients with IPF, and with potential applications in other interstitial lung diseases and cancer-associated fibrosis. We are encouraged by both the strength of our preclinical package as well as the clinical results to date and we look forward to reporting phase 2a topline data in Q1 2024.”

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

Reference:

• Redx provides progress update on RXC007 clinical programme. https://www.redxpharma.com/wp-content/uploads/2023/02/Redx-Pharma-Redx-Provides-Progress-Update-on-RXC007-Clinical-Programme.pdf. Published Feb. 9, 2023. Accessed Aug. 28, 2023.