Zydus Lifesciences has been granted ‘Orphan Drug Designation’ (ODD) by the United States Food and Drug Administration (USFDA) for ZYIL1, for treatment of patients with Cryopyrin Associated Periodic Syndrome (CAPS). The FDA's Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States.
ZYIL1 is a novel oral small molecule NLRP3 inhibitor which has demonstrated CSF penetration in non-human primates. In September 2022, Zydus had announced positive Phase 2 proof-of-concept (POC) study in CAPS patients, and publication of Phase 1 study results in Clinical Pharmacology in Drug Development, supporting the advancement of ZYIL1 into pivotal clinical trials in CAPS patients.
Zydus Lifesciences (formerly known as Cadila Healthcare) is an India-based pharmaceutical company.
