FDA Grants Orphan Drug Status to 4SC’s Resminostat for CTCL, Advancing Commercialization Efforts
4SC AG, a clinical-stage biopharmaceutical company based in Germany, has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for its drug candidate resminostat (Kinselby) in the treatment of cutaneous T cell lymphoma (CTCL). This designation grants 4SC seven years of market exclusivity in the United States, providing a significant boost to the company’s efforts to commercialize Kinselby.
Cutaneous T cell lymphoma (CTCL) is a rare disease that affects the skin and can eventually involve lymph nodes, blood, and other organs. Currently, CTCL is incurable and treatment options for advanced-stage CTCL are limited. 4SC’s resminostat, an orally administered histone deacetylase (HDAC) inhibitor, offers a promising approach to treating this challenging disease. It has demonstrated tolerability, inhibition of tumor growth, tumor regression, and enhancement of the immune response against cancer.
The FDA’s Orphan Drug Designation recognizes the potential of resminostat to address an unmet medical need in a rare disease. It provides several important benefits to 4SC, including seven years of market exclusivity in the US. This exclusivity will give 4SC a competitive advantage and enable the company to focus on advancing the commercialization of Kinselby.
In addition to the Orphan Drug Designation in the US, 4SC is also preparing a marketing authorization application for Kinselby in the European Union. The submission is on track for the first quarter of 2024. This global effort reflects 4SC’s commitment to bringing this potentially life-changing therapy to patients worldwide.
4SC’s current pipeline is protected by a comprehensive portfolio of patents and includes resminostat as the lead drug candidate in clinical development. The company aims to generate future growth and enhance its value by forming strategic partnerships with pharmaceutical and biotech companies and by potentially marketing and selling approved drugs in select territories.
Based in Planegg-Martinsried near Munich, Germany, 4SC is listed on the Prime Standard of the Frankfurt Stock Exchange and had 16 employees as of June 30, 2023. The company is driven by its mission to develop innovative small-molecule drugs that target key indications in cancer with high unmet medical needs.
The FDA’s Orphan Drug Designation for resminostat represents a significant milestone for 4SC and highlights the potential of this novel therapy in addressing the urgent medical need in patients with CTCL. With market exclusivity in the US and a promising clinical development program, 4SC is poised to make a positive impact in the field of cancer treatment.
