Drug intelligence / Profile preview

CC8464

Development stage
Unknown
Lead developer
Pelthos Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

CC8464 (also known as ASP1807) is an orally available, peripherally restricted small molecule inhibitor of the voltage-gated sodium channel NaV1.7. It was initially developed by Chromocell and later licensed to Astellas Pharma for further development and commercialization. NaV1.7 is a key ion channel involved in pain transmission, particularly in neuropathic and inflammatory pain conditions. By selectively blocking NaV1.7 in the peripheral nervous system, CC8464 aims to reduce pain without central nervous system side effects such as addiction or psychiatric disorders. The drug has demonstrated efficacy in multiple animal models of human neuropathic and inflammatory pain, including models relevant to erythromelalgia (EM), a rare condition characterized by burning pain symptoms linked to mutations in the NaV1.7 gene[1][3][4][5]. Phase I clinical trials have shown that CC8464 is well tolerated with no significant liver, renal, cardiovascular, or CNS toxicity[5]. The compound has received FDA Fast Track designation for idiopathic small fiber neuropathy-associated neuropathic pain[1].

02

Targets

SCN9A (Sodium channel protein type 9 subunit alpha)

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