The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to 4SC’s resminostat (Kinselby) for cutaneous T-cell lymphoma (CTCL).
Resminostat is an orally administered class I, IIb and IV histone deacetylase (HDAC) inhibitor that potentially offers an approach to treating different kinds of cancer.
The company demonstrated that resminostat is well tolerated and can inhibit tumour growth and proliferation, cause tumour regression, and strengthen the body’s immune response to cancer in the RESMAIN study.
The drug is currently being investigated in a pivotal study in cutaneous T-cell lymphoma (CTCL) as maintenance treatment by 4SC in Europe and by Yakult Honsha in Japan. Maintenance treatment – a unique innovative treatment approach in CTCL – is intended to prolong the period patients are stable and not progressing.
Jason Loveridge, CEO of 4SC, commented: “Receiving orphan drug designation for resminostat provides us with a number of important benefits, most crucially seven years’ market exclusivity in the US, a key foundation of our efforts to commercialise Kinselby. We are currently preparing a marketing authorisation application for Kinselby in the EU, which remains on track for submission in Q1 2024.”
CTCL is a rare disease with approximately 5,000 patients being newly diagnosed in Europe each year. Currently, CTCL is incurable and treatment options for advanced-stage CTCL are limited.
Although patients respond to the available treatment options, the duration of response is often short-lived and declines as the severity of the disease increases. The key therapeutic challenge in advanced-stage CTCL is therefore to make remissions more durable by halting disease progression and improving patient’s quality of life.