FDA grants Orphan Drug Designation to Ariceum cancer therapy

Designation follows promising preclinical data and FDA clearance

Ariceum Therapeutics, a private biotech company based in Berlin, has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its proprietary radiopharmaceutical, 225Ac-satoreotide, for treating patients with Small Cell Lung Cancer (SCLC).

SCLC is a severe condition with a poor prognosis and a 5-10% overall five-year survival rate. The orphan drug designation recognises the potential of 225Ac-satoreotide as a treatment option for SCLC patients. According to Ariceum, phase I/II human clinical trials will begin in Q1 2025 under the trial name, SANTANA-225.

Manfred Rüdiger, Chief Executive Officer at Ariceum Therapeutics, said: “Receiving ODD for 225Ac-satoreotide is a recognition of its potential as a treatment option for patients with SCLC. The FDA’s ODD will support our objective to accelerate the development of 225Ac-satoreotide through human trials.”

The FDA provides ODD to drugs that show potential for diagnosing and treating rare diseases. The designation offers several development and commercial incentives, including seven years of market exclusivity in the US following product approval and FDA assistance in clinical trial design.

In October 2024, Ariceum released preclinical data highlighting the potential of 225Ac-satoreotide. The radiopharmaceutical showed a high frequency of complete durable responses and 100% survival, supporting its advanced clinical development in SCLC, MCC and other aggressive cancers.

The companion patient selection tracer, 68Ga-SSO120, is being developed as part of a ‘theranostic pair’ for combined diagnosis and targeted radionuclide treatment.

Ariceum aims to provide a potentially life-saving therapy to patients with limited alternatives.