FDA acordă denumirea de medicament orfan pentru tratamentul limfomului cu celule T

Dialectic Therapeutics, Inc., a Texas-based clinical stage biotechnology company focused on creating innovative new technologies to treat cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to DT2216 for the treatment of T-cell lymphoma.  DT2216 is Dialectic’s first generation compound built using its proprietary and novel Antiapoptotic Protein Targeted Degradation (APTaD™) technology platform.

“This is an important milestone in the development of DT2216, our lead APTaD™ compound. The FDA’s decision to grant orphan drug designation underscores our belief that DT2216 could be a promising therapeutic for T-cell lymphoma patients” said Dr. David Genecov, Dialectic’s President and Chief Executive Officer. “There is a critical unmet need for people diagnosed with this rare cancer, in which current approved therapies have relatively low response rates”.

Normal T-cells require BCL-XL expression to survive thymic selection during their development. After thymic selection BCL-XL normal T-cells no longer express BCL-XL. However, many T-cell lymphomas re-express BCL-XL as a mechanism of their neoplastic transformation and permits their continued survival as a malignancy. Studies have demonstrated the importance of BCL-XL in T-cell lymphoma survival. Dialectic has shown that DT2216 is an effective treatment for T-cell lymphoma in preclinical studies.

The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.