FDA Grants Orphan Drug Status to Zydus Lifesciences' ZYIL1 for Treating Cryopyrin-Associated Periodic Syndrome

Zydus Lifesciences has announced that its drug, ZYIL1, has received orphan drug designation (ODD) from the United States Food and Drug Administration (US FDA) for the treatment of Cryopyrin Associated Periodic Syndrome (CAPS) patients. The FDA’s Office of Orphan Drug Products grants ODD to encourage the development of medications for rare diseases that impact fewer than 200,000 individuals in the US.

Publish Date: 21-03-2023 Source: Zydus Lifesciences

Multiple myeloma (MM) is a complex hematologic malignancy characterized by the uncontrolled proliferation of clonal plasma cells in the bone marrow that secrete large amounts of immunoglobulins and other non-functional proteins. Despite decades of progress and several landmark therapeutic advancements, MM remains incurable in most cases. These cells are hyperproliferative differentiated B-lymphocytes capable of secreting a variety of immunoglobulins (Ig), with the most common being IgG, IgA, and, to a lesser extent, IgD. The abnormal PCs typically grow in the bone marrow, with only a small fraction of patients presenting with the extramedullary disease at the time of diagnosis. Most patients experience a relapse during treatment, progress to refractory, and stop responding to treatment within 60 days of the last treatment, characterized by poor prognosis and a high risk of relapse. With each additional relapse, patients experience a shorter PFS. PFS in first-line patients were 18 months, but by the 4th line of therapy, PFS was only 4.7 months. The primary goal of treatment for relapsed MM is to alleviate disease symptoms and prevent the development of CRAB symptoms (Calcium > 11.5 mg/dL; creatinine > 2 mg/dL; haemoglobin < 10 g/dL or >2 points below the lower normal limit; new bone lesions). The treatment of RRMM is a complex and standard combination of proteasome inhibitors, immunomodulatory drugs and monoclonal antibodies, with the addition of histone deacetylase inhibitors and alkylating agents.

In the USA, the incidence of Multiple myeloma ranges from 5 to 9 cases per 100,000 population per year with 45% of 5-year survival.

However, the current Cryopyrin-Associated Periodic Syndrome (CAPS) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (DFV890, ZYIL1, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Cryopyrin-Associated Periodic Syndrome treatment. The key companies in the advanced development stage are Novartis Pharmaceuticals, Zydus Lifesciences Limited, etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Cryopyrin-Associated Periodic Syndrome (CAPS) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2022 to 2032.

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