FDA Rejects CUTX-101 for Rare Copper Deficiency Due to Manufacturing Issues

The FDA has rejected the New Drug Application (NDA) for CUTX-101, a treatment for Menkes disease, due to manufacturing-related issues at the facility producing the drug135.

Menkes disease is a rare, often fatal, pediatric disorder caused by mutations in the ATP7A gene, typically resulting in death by age 2 to 313.

No concerns with the drug's safety or efficacy data were cited by the FDA; the rejection was solely based on deficiencies at the manufacturing facility detected during a September inspection135.

CUTX-101 is a subcutaneous injectable form of copper histidine designed to restore copper homeostasis in patients1.

Sentynl, Cyprium, and Fortress plan to request a meeting with the FDA to discuss resubmission and corrective actions regarding manufacturing15.

Manufacturing-related FDA rejections have become a recurring issue affecting other rare disease drug submissions recently3.

Sources:

1. https://www.fiercebiotech.com/biotech/fda-snubs-fortress-cypriums-zydus-partnered-menkes-disease-treatment-hopes-manufacturing

3. https://www.biospace.com/fda/fortress-sentynl-fail-to-secure-fda-nod-in-rare-pediatric-disease

5. https://www.fortressbiotech.com/news-media/press-releases/detail/719/fortress-biotech-and-cyprium-therapeuticsannounce-an