Ayala Pharmaceuticals, a clinical-stage company developing medicines for cancers that are genetically defined, announced that it has been granted Orphan Drug Designation from the U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) for AL101, a potent and selective inhibitor of gamma secretase-mediated Notch signaling, for the treatment of ACC.
Orphan Drug Designation is granted to drug therapies intended to treat diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation by the FDA entitles Ayala to seven years of market exclusivity for the use of AL101 for the treatment of ACC, if approved, plus significant development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.
“Receiving Orphan Drug Designation from the FDA signifies our continued progress and commitment to develop AL101 as a potential treatment for people with ACC,” said Roni Mamluk, Ph.D., Chief Executive Officer at Ayala Pharmaceuticals. “Given the proof of concept demonstrating that AL101 has a significant inhibitory effect on tumor growth in a mouse model using implanted ACC patient-derived xenograft tumors coupled with the significant unmet need in ACC, we look forward to accelerating AL101 development for patients 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.
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