Step Pharma receives US FDA orphan drug designation for dencatistat in T-Cell lymphoma

On May 22, 2025 Step Pharma ("the Company"), the global leader in CTPS1 inhibition for targeted cancer treatment, reported that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its lead candidate, dencatistat, for the treatment of T cell lymphoma, encompassing all subtypes including peripheral T cell lymphoma and cutaneous T cell lymphoma (Press release, Step Pharma, MAY 22, 2025, View Source [SID1234653318]).

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The designation highlights the significant unmet medical need in T cell lymphoma, a rare and aggressive group of blood cancers. It also recognises the potential of dencatistat as a novel, selective therapeutic option. Orphan drug designation provides incentives designed to support the development of drugs for rare diseases, including market exclusivity upon approval, tax credits for clinical trial costs, and waiver of certain FDA fees.

Andrew Parker, Chief Executive Officer of Step Pharma, commented:

"Receiving orphan drug designation from the FDA, which may confer seven years of market exclusivity for dencatistat, marks an important milestone in our mission to deliver highly selective cancer therapies for patients with difficult-to-treat cancers. T cell lymphoma remains a challenging disease with limited treatment options, and we believe CTPS1 inhibition represents a new therapeutic approach with the potential to meaningfully improve outcomes."

Dencatistat is being evaluated in a phase 1/2 clinical trial for adult patients with relapsed/refractory T or B cell lymphoma. The Company is also evaluating dencatistat in a phase 1 trial in solid tumour patients, with expansion cohorts in CTPS2-null ovarian and lung cancer planned. Additionally, a phase 1b trial in essential thrombocythaemia is expected to commence in the first half of 2025, further supporting dencatistat’s potential as a targeted therapy across multiple cancer types.