MDT-001

MDT-001 is a code name associated with multiple distinct drug candidates in development by different pharmaceutical companies. The most prominent candidate is a first-in-class, oral small molecule developed by **Medetia Pharmaceuticals** for the treatment of **Nephronophthisis (NPHP)**, a rare genetic ciliopathy that leads to end-stage renal disease. This MDT-001 was identified via a phenotypic screening platform and is designed to restore the function of the primary cilium, which is defective in NPHP and other ciliopathies. By targeting the underlying ciliary dysfunction, MDT-001 aims to slow or prevent the progression of renal fibrosis and cyst formation. The drug received Orphan Drug Designation from the FDA in 2021. Another candidate, also designated **MDT-001**, is being developed by **Modulate Therapeutics** as a subtype-selective **GABA-A receptor positive allosteric modulator (PAM)** for the treatment of **Dravet Syndrome** and other refractory epilepsies. Additionally, the code MDT-001 is used by **MDT Pharma** in Japan for a veterinary formulation of **medetomidine hydrochloride** used as a sedative in aquaculture.

Other names

MDT-01MDT01MDT 01