MBX Biosciences’ Hypoparathyroidism Drug Candidate Gains Orphan Drug Status

MBX Biosciences’ investigational drug MBX 2109 received Orphan Drug designation from the FDA for treatment of hypoparathyroidism.

The company’s lead candidate, MBX 2109 is an investigational long-acting parathyroid hormone prodrug designed to restore parathyroid hormone physiology and correct the mineral imbalances to alleviate disorder-related symptoms. Prodrugs are medications that are metabolized into a pharmacologically active drug.

The therapy is currently being evaluated in a phase 1 clinical trial to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple ascending doses.

The Orphan Drug designation provides FDA support for the development and evaluation of new treatments for rare diseases.