Redx Pharma’s pioneering treatment of Idiopathic Pulmonary Fibrosis (IPF), Zelasudil, has been granted orphan drug status by the US regulator, the Food and Drug Administration (FDA).
Zelasudil, currently in Phase 2a clinical study for IPF with topline data expected in the first quarter of 2024, targets ROCK2, an enzyme central to fibrosis or lung-scarring
Blocking the enzyme is crucial to avoid systemic hypotension (high blood pressure), said Redx, a side effect of existing treatments that systemically inhibit both ROCK2 and ROCK1, another enzyme linked to scarring.
Zelasudil can potentially treat several fibrotic diseases and has demonstrated robust anti-fibrotic effects in a range of industry-standard in vivo preclinical models, the AIM-listed firm added.
Jane Robertson, Redx Pharma's chief medical officer, commented: "We are delighted that the FDA has recognised the potential of zelasudil.
"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.
IPF is a severe and life-threatening chronic lung condition with limited treatment options.
Around 170,000 people suffer from the disease in the US, EU and Japan and around 53,000 new cases are diagnosed every year.
Patients diagnosed with IPF have an estimated life expectancy of 3 to 5 years. There is no known cure and current treatment only slows the progression of the disease.
The US grants orphan drug designation for potential treatments for diseases or condition that affects fewer than 200,000 in the US and gives commercial protections to encourage drug development.