4SC receives Orphan Drug Designation for Resminostat in the European Union

4SC AG, a biotech company improving the lives of patients suffering with advanced-stage CTCL, received notification from the European Medicines Agency (EMA) that it has granted Orphan Drug Designation (ODD) to resminostat for the treatment of cutaneous T-cell lymphoma (CTCL).

ODD provides privileged status to drugs that show promise for the treatment of rare diseases in the European Union. ODD qualifies 4SC for benefits including protocol assistance, market exclusivity and fee reductions.

Today’s announcement follows the announcement on 27 September 2023 that the US FDA had granted resminostat (Kinselby) orphan drug designation, which gives a number of benefits, most importantly seven years’ market exclusivity in the US.

CTCL is a rare disease with approximately 5,000 patients being newly diagnosed in Europe each year. The disease arises from malignant transformation of T-cells, a specialised subgroup of immune cells, and primarily affects the skin, but may ultimately involve lymph nodes, blood and visceral organs.   

4SC recently announced data from its RESMAIN study, one of the largest randomised, controlled clinical trials in advanced CTCL, demonstrating for the first time the benefit of maintenance therapy in patients with advanced CTCL. In this study, resminostat (Kinselby) showed a statistically significant improvement in progression free survival of 97.6% compared to placebo, with a risk reduction of 38% in recently announced headline trial results (median Progression Free Survival (”PFS"): 8.3 months versus 4.2 months; p=0.015; HR: 0.623 (95%CI: 0.424, 0.916).  

Furthermore, resminostat (Kinselby)’s median time to next treatment versus placebo showed a significant improvement of 8.8 months compared to 4.2 months; p= 0.002; HR: 0.594 (95% CI: 0.424, 0.916). The side effects of resminostat were mainly mild to moderate, manageable and reversible. 

Jason Loveridge, Ph.D., CEO of 4SC, commented: “CTCL is currently an incurable disease and patients are in great need of better therapies. Our recent RESMAIN trial has demonstrated strong efficacy data for resminostat (Kinselby). Receiving orphan drug designation provides us with a number of important benefits for resminostat (Kinselby), most crucially 10 years’ market exclusivity in the European Union, and, alongside our Orphan Drug Designation in the US, provides us with a solid foundation for our efforts to commercialise Kinselby in these major markets.”

Additional analyses established that those treated showed a clinically meaningful improvement in median “total” PFS (defined from start of last prior therapy to disease progression) of 24.3 months, compared to 14.9 months for those in the placebo group.