FDA approves first treatment for children with Menkes disease

The US Food and Drug Administration yesterday approved the Zycubo (copper histidinate) injection as the first treatment for Menkes disease in pediatric patients. The FDA approved Zycubo, previously known as CUTX-101, for Sentynl Therapeutic, a part India’s Zydus Lifesciences (BOM: 532321) which filed the regulatory filing and is partnered with Fortress Biotech (Nasdaq: FBIO).

“With today’s action, children with this devastating, degenerative disease will have an FDA-approved treatment option and the potential to live longer,” said Dr Christine Nguyen, deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research (CDER), adding: “The FDA will continue to work with the rare disease community to advance drug development for patients with Menkes disease and other rare conditions.”

“This approval marks an unprecedented advance for children with Menkes disease,” said Dr Tracy Beth Hoeg, acting director of CDER. “The company demonstrated a large improvement in overall survival compared with untreated patients, using an innovative trial design that addressed the challenges of studying an ultra-rare disease,” she noted.”