Orphan Drug Designation (ODD) is intended to promote the development of drugs that address rare diseases. In the United States, the Food and Drug Administration (FDA) grants this status to drugs or biological products designed to treat diseases affecting fewer than 200,000 people in the country. During the development of the drug candidate, this designation provides certain advantages, such as tax credits for clinical trials conducted in the U.S. At a later stage, ODD offers the opportunity to waive fees associated with applications for marketing approval, as well as up to seven years of market exclusivity.

Elicera Therapeutics is developing ELC-100 as a new potential treatment for neuroendocrine tumors (NET). The drug candidate is based on an oncolytic virus and exerts its therapeutic effect by selectively infecting and killing neuroendocrine cancer cells. In the fall of 2024, the company announced that the final patient had been recruited for the first part of an ongoing Phase I/II clinical trial aimed at identifying the maximum tolerated dose (MTD) and evaluating the treatment's safety in patients. The company is expected to report the final data from the first part of the study around mid-2025.

"Neuroendocrine tumors represent a highly heterogeneous indication and in our ongoing clinical study with severely ill patients that can be divided into several subgroups, including based on treatment history. This diversity highlights the need for new therapeutic solutions to be developed with a broad understanding of the specific needs of different patient groups. We are very pleased that ELC-100 has been granted Orphan Drug Designation in the U.S. The decision by the FDA is a significant milestone in our efforts to develop a new form of treatment for patients with neuroendocrine tumors",says Jamal El-Mosleh, CEO of Elicera Therapeutics.