Heidelberg Pharma Obtains FDA Orphan Drug Designation for HDP-101 in Multiple Myeloma Treatment

Heidelberg Pharma AG has achieved a significant milestone as the US Food and Drug Administration (FDA) granted Orphan Drug Designation for HDP-101, its proprietary ATAC candidate for multiple myeloma treatment. This designation marks a notable advancement in developing treatments for this rare and severe form of cancer. HDP-101, currently under clinical Phase I/IIa study, aims to provide a new therapeutic option for relapsed/refractory multiple myeloma (RRMM) patients.

The innovative approach of HDP-101, combining an anti-BCMA antibody with the Amanitin toxin through a specific linker, targets BCMA, a protein highly expressed on multiple myeloma cells. This targeted mechanism of action underpins its potential as a vital therapy for multiple myeloma, addressing unmet medical needs within this patient population.

Orphan Drug Designation entitles Heidelberg Pharma to various incentives, including tax benefits, fee waivers, and potentially seven years of market exclusivity post FDA approval. This status is aimed at encouraging the development of medications for rare diseases, affecting fewer than 200,000 people in the US. The designation underscores the therapeutic potential of HDP-101 and Heidelberg Pharma's commitment to addressing critical gaps in cancer treatment.

The company plans to present early safety and efficacy data at the American Association for Cancer Research Annual Meeting, further highlighting its ongoing efforts to combat multiple myeloma. However, HDP-101 remains an investigational product, with its safety and efficacy continuing to be evaluated.