USFDA grants Orphan Drug Designation for Zydus's ZYIL1

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For treatment of patients with Cryopyrin Associated Periodic Syndrome

Zydus Lifesciences announced that the United States Food and Drug Administration (USFDA) has granted 'Orphan Drug Designation' (ODD) to 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.

Pankaj R. Patel, Chairman, Zydus Lifesciences, said, This Orphan Drug Designation from the USFDA underlines the urgent need to develop ZYIL1 to address this rare and critical chronic inflammatory condition CAPS, a high unmet medical need. ZYIL1 demonstrated Phase 2 proof-of-concept in CAPS patients showing rapid clinical improvement as early as day 3 which sustained till the end of the treatment, in addition to the improvement in inflammatory markers like Serum Amyloid A (SAA), CRP, IL-6. There were no Serious Adverse Events (SAE's) observed.

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