Policy & Regulation
FDA Grants Orphan Drug Designation to Ayala's AL101 for Potential Treatment of Adenoid Cystic Carcinoma
17 May 2019 - - Israeli clinical-stage company Ayala Pharmaceuticals has been granted Orphan Drug Designation from the US Food and Drug Administration's Office of Orphan Products Development for AL101, a potent and selective inhibitor of gamma secretase-mediated Notch signalling, for the treatment of ACC, the company said.

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.

ACC is a rare form of cancer. In the US, 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 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 2 for adenoid cystic carcinoma patients with tumor bearing Notch activating mutations (ACCURACY).

Ayala Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company dedicated to developing targeted cancer therapies for people living with genetically defined cancers.

Ayala is broadly developing its product candidates, AL101 and AL102, best-in-class gamma secretase inhibitors, with clinical and preclinical studies underway in both solid tumors (AL101) and hematologic malignancies (AL102).

As a precision oncology company, Ayala was founded in November 2017 with an experienced global management team and a strong investor base.
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