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Zydus gets Orphan Drug Designation from USFDA for ZYIL1 to treat Cryopyrin Associated Periodic Syndrome

Orphan drug designation by USFDA for ZYIL1 provides eligibility for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval

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Zydus Lifesciences announced that the United States Food and Drug Administration (USFDA) has granted ‘Orphan Drug Designation’ (ODD) to ZYIL1, for the treatment of patients with Cryopyrin Associated Periodic Syndrome (CAPS). The FDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the US.

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 the 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.”

CAPS is a rare, life-long auto-inflammatory condition, caused by NLRP3 activating mutations and is classified under orphan diseases. The chronic inflammation due to IL-1beta release in CAPS patients leads to urticaria-like rash, fever, arthralgia, and increased risk of amyloidosis. CAPS patients also experience multiple neurological complications like sensorineural hearing loss, migraine, headache, aseptic meningitis and myalgia. Bone deformities and neurological impairment have been reported in Neonatal Onset Multisystem Inflammatory Disease (NOMID), the most severe form of CAPS.

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