- Home
- Medical news & Guidelines
- Anesthesiology
- Cardiology and CTVS
- Critical Care
- Dentistry
- Dermatology
- Diabetes and Endocrinology
- ENT
- Gastroenterology
- Medicine
- Nephrology
- Neurology
- Obstretics-Gynaecology
- Oncology
- Ophthalmology
- Orthopaedics
- Pediatrics-Neonatology
- Psychiatry
- Pulmonology
- Radiology
- Surgery
- Urology
- Laboratory Medicine
- Diet
- Nursing
- Paramedical
- Physiotherapy
- Health news
- Fact Check
- Bone Health Fact Check
- Brain Health Fact Check
- Cancer Related Fact Check
- Child Care Fact Check
- Dental and oral health fact check
- Diabetes and metabolic health fact check
- Diet and Nutrition Fact Check
- Eye and ENT Care Fact Check
- Fitness fact check
- Gut health fact check
- Heart health fact check
- Kidney health fact check
- Medical education fact check
- Men's health fact check
- Respiratory fact check
- Skin and hair care fact check
- Vaccine and Immunization fact check
- Women's health fact check
- AYUSH
- State News
- Andaman and Nicobar Islands
- Andhra Pradesh
- Arunachal Pradesh
- Assam
- Bihar
- Chandigarh
- Chattisgarh
- Dadra and Nagar Haveli
- Daman and Diu
- Delhi
- Goa
- Gujarat
- Haryana
- Himachal Pradesh
- Jammu & Kashmir
- Jharkhand
- Karnataka
- Kerala
- Ladakh
- Lakshadweep
- Madhya Pradesh
- Maharashtra
- Manipur
- Meghalaya
- Mizoram
- Nagaland
- Odisha
- Puducherry
- Punjab
- Rajasthan
- Sikkim
- Tamil Nadu
- Telangana
- Tripura
- Uttar Pradesh
- Uttrakhand
- West Bengal
- Medical Education
- Industry
USFDA grants Orphan Drug Designation to Zydus's ZYIL1 for treatment of Cryopyrin Associated Periodic Syndrome
ZYIL1 is a novel oral small molecule NLRP3 inhibitor which has demonstrated CSF penetration in non-human primates.
Ahmedabad: Zydus Lifesciences Ltd., a discovery-driven, global life sciences company today 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 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 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.
Mr. Pankaj R. Patel, Chairman, Zydus Lifesciences Ltd., 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.
Ruchika Sharma joined Medical Dialogue as an Correspondent for the Business Section in 2019. She covers all the updates in the Pharmaceutical field, Policy, Insurance, Business Healthcare, Medical News, Health News, Pharma News, Healthcare and Investment. She has completed her B.Com from Delhi University and then pursued postgraduation in M.Com. She can be contacted at editorial@medicaldialogues.in Contact no. 011-43720751