Monday, May 13, 2019
Orphan Drug Designation Granted for Adenoid Cystic Carcinoma Treatment
The FDA has granted Orphan Drug Designation for AL101, a potent and selective inhibitor of gamma secretase-mediated Notch signalling, for the treatment of adenoid cystic carcinoma (ACC).
Orphan Drug Designation is granted to drug therapies intended to treat diseases or conditions that affect fewer than 200,000 people in the U.S.
ACC is a rare form of cancer. In the U.S., there are approximately 566,000 people diagnosed with cancer each year, and only about 1,224 of them are diagnosed with ACC. According to the Adenoid Cystic Carcinoma Organization International, there are approximately 14,873 Americans alive today living with this disease. Current treatment options include surgery, chemotherapy and/or radiation therapy; however, there is no approved drug for the treatment of ACC.
AL101 is a gamma secretase inhibitor developed as a Notch inhibitor for oncology indications. Notch signaling pathway plays an important role in tumorigenesis in several solid and hematological malignancies. Upon ligand binding of the Notch receptor, an important step in the activation of Notch receptors is cleavage by gamma secretase, which frees the Notch intracellular signaling domain.
AL101 is currently in phase II for adenoid cystic carcinoma patients with tumor bearing Notch activating mutations (ACCURACY).