Renibus Therapeutics Receives Orphan Drug Designation from FDA for Veverimer in Anti-GBM Disease

Renibus Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to veverimer for the treatment of anti-glomerular basement membrane disease (anti-GBM), a rare life-threatening condition characterized by acute renal failure. This designation is intended to encourage the development of products for diseases that affect fewer than 200,000 individuals in the United States, making Renibus eligible for certain incentives such as tax credits for clinical trials and exemption from user fees.

Key Takeaways:

• The FDA has granted Orphan Drug Designation to veverimer for the treatment of anti-GBM disease, a rare condition affecting fewer than 200,000 individuals in the United States.
• Veverimer is an oral, non-absorbed hydrochloric acid binder that has been shown to suppress ammoniagenisis and associated activation of the complement cascade, key mediators of anti-GBM disease and other complement mediated renal diseases.
• Renibus is also evaluating veverimer's potential use for the treatment of other renal diseases.
• Renibus has a lead first-in-class combination drug therapy, RBT-1 (stannic protoporfin / iron sucrose), which has completed enrollment in a Phase 3 pivotal trial (PROTECT) measuring its primary objective to reduce the risk of post-operative complications and improve outcomes following cardiac surgery.
• RBT-1 has been granted FDA Breakthrough and Fast Track Designations by the Division of Cardiology and Nephrology at the FDA.

Statistics:

• Veverimer has been granted Orphan Drug Designation for the treatment of anti-GBM disease, a condition affecting fewer than 200,000 individuals in the United States.
• Renibus has completed enrollment in a Phase 3 pivotal trial (PROTECT) for RBT-1, with plans to evaluate its potential use in treating other renal diseases.
• RBT-1 has been granted FDA Breakthrough and Fast Track Designations by the Division of Cardiology and Nephrology at the FDA.
• Veverimer is an oral, non-absorbed hydrochloric acid binder that has been shown to suppress ammoniagenisis and associated activation of the complement cascade.

Sources:

• PR Newswire, "Renibus Therapeutics Announces FDA Orphan Designation for Veverimer in Anti-Glomerular Basement Membrane Disease"
• Renibus Therapeutics website, "Renibus Therapeutics Receives Orphan Drug Designation for Veverimer in Anti-GBM Disease"
• Renibus Therapeutics website, "About Renibus Therapeutics"