Elevar Therapeutics, Inc. (“Elevar”), a fully integrated biopharmaceutical company built on the promise of elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options, today announced that the U.S. Food and Drug Administration (FDA) has granted rivoceranib (apatinib) with orphan drug designation for the treatment of adenoid cystic carcinoma (ACC), a rare form of cancer that most commonly develops in the salivary glands or other regions of the head and neck. Approximately 1,200 new cases of ACC are diagnosed each year in the United States. 

The FDA’s Office of Orphan Drug Products grants orphan status to medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S.

“We are pleased by the FDA’s recognition of the critical need to develop treatments for adenoid cystic carcinoma (ACC) given the progressive nature of the disease,’ said Alex Kim, chief executive officer of Elevar. “The orphan drug designation provides further momentum for the rivoceranib development program, which we are committed to advancing as quickly as possible for patients in need.”

ACC is considered a slow growing but relentless cancer that is characterized by nerve invasion and metastases. Although good local control is usually achieved by resection of the primary tumor and adjuvant radiation therapy, more than half of patients eventually have recurrent and/or metastatic disease. Currently, no curative treatments are available for these patients which underscores the need for effective new therapies.

“With no approved treatments for adenoid cystic carcinoma (ACC), a significant unmet need remains for therapies that slow or stop this relentless disease,” said Mark Gelder, M.D., chief medical officer of Elevar. “The orphan drug designation from the FDA reinforces the urgency of Elevar’s work with rivoceranib in ACC and our commitment to improving treatment experiences and outcomes for patients who have limited or inadequate therapeutic options.”

In November 2020, Elevar announced that the Company met its initial 55 patient enrollment target for its Phase 2, open-label, multicenter, clinical trial of rivoceranib in patients with recurrent or metastatic ACC one year ahead of schedule. The Company also announced increasing the target size of the trial to approximately 72 patients to further explore the potential clinical benefit of rivoceranib in this patient population.