FDA Rejects Fortress and Sentynl's Drug for Menkes Disease Due to Manufacturing Issues

What's Happening?

The FDA has rejected the investigational treatment for Menkes disease developed by Fortress Biotech and Sentynl Therapeutics. Menkes disease is a rare genetic disorder that typically results in death by the age of three. The rejection was based on manufacturing issues at the facility where the drug is produced, as noted in the FDA's complete response letter. Fortress Biotech stated that the FDA did not find any deficiencies related to the drug's efficacy or safety. The drug, CUTX-101, is a subcutaneous formulation of copper histidinate, which has shown promising results in reducing the risk of death by 80% in treated patients compared to untreated historical controls. Despite the rejection, Fortress plans to seek a meeting with the FDA to discuss resubmission.

Why It's Important?

The rejection of CUTX-101 highlights ongoing challenges in drug manufacturing that can impact regulatory approval, even when a drug shows potential efficacy and safety. Menkes disease affects approximately 1 in 100,000 infants, and the lack of approved treatments underscores the need for effective therapies. The FDA's decision reflects a broader trend of manufacturing-related rejections, as seen with other companies like Biogen and Scholar Rock. Approval of CUTX-101 could have significant implications for patients and the rare disease community, offering a potential treatment option and possibly earning a priority review voucher for Fortress Biotech.

What's Next?

Fortress Biotech and Sentynl Therapeutics plan to engage with the FDA to address the manufacturing issues and discuss the possibility of resubmitting their application for CUTX-101. If approved, the drug could provide a much-needed treatment for Menkes disease and potentially earn Fortress up to $129 million in development and sales milestones, along with royalties on net sales. The rare disease space remains active, with the FDA open to expedited approvals, although companies continue to face unexpected rejections.

Beyond the Headlines

The rejection of CUTX-101 due to manufacturing issues raises questions about the regulatory process and the balance between ensuring drug safety and efficacy versus addressing logistical challenges in production. This situation may prompt discussions on how to streamline manufacturing practices to prevent similar setbacks in the future, especially for drugs targeting rare diseases where treatment options are limited.