Sun Pharma Research Arm- SPARC gets USFDA orphan drug designation for novel kinase inhibitor

Mumbai: Sun Pharma Advanced Research Company (SPARC) on Monday said the US health regulator has given orphan drug designation to its novel kinase inhibitor, SCO-088, for the treatment of chronic myeloid leukaemia (CML).

SCO-088 is a novel and highly selective Bcr – Abl kinase inhibitor intended for the treatment of resistant Chronic Myelogenous Leukemia. SCO-088 is a potent inhibitor of Bcr-Abl and its mutant forms.

The Orphan Drug Act provides economic incentives to encourage the development of drugs for diseases affecting fewer than 2 lakh people in the US, Sun Pharma Advanced Research Company (SPARC) said in a filing to BSE.

"This is an important milestone in SPARC's journey to provide a solution for patients who are not responding to the existing treatments available in the market. SCO-088 will fill the gap in the treatment of resistant CML," SPARC CEO Anil Raghavan said.

Orphan drug designation will grant seven years of US market exclusivity upon approval of SCO-088 for the treatment of patients with CML, SPARC said.

CML is a myeloproliferative neoplasm that accounts for approximately 15% of newly diagnosed cases of leukaemia in adults. Central to the pathogenesis of CML is the fusion of the Abelson murine leukaemia (ABL1) gene on chromosome 9 with the Breakpoint Cluster Region (BCR) gene on chromosome 22. This rearrangement is known as the Philadelphia chromosome1. The American Cancer Society estimated that in the United States in 2018 there were about 8,430 new cases of CML (4,980 in men and 3,450 in women) and about 1,090 deaths due to CML (620 men and 470 women)2.

Incentives include a waiver of user fee by United States Food and Drug Administration (USFDA) and assistance in clinical trial design by the regulator, it added.

Shares of SPARC were trading at Rs 113.80 per scrip on BSE, down 2.94 per cent from the previous close.

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