Fortress Biotech Faces FDA Setback on Menkes Disease Treatment: CUTX-101's Approval Hinges on Addressing Manufacturing Deficiencies

Fortress Biotech (FBIO) received a Complete Response Letter (CRL) from the FDA for its drug CUTX-101, intended to treat Menkes disease. The CRL highlighted manufacturing practice deficiencies, but no concerns were raised about the drug's efficacy or safety. Cyprium, the drug's developer, will receive significant financial benefits from Sentynl Therapeutics upon approval, including a Rare Pediatric Disease Priority Review Voucher and potential milestone payments.

Fortress Biotech, Inc. (Nasdaq: FBIO) and its subsidiary Cyprium Therapeutics received a Complete Response Letter (CRL) from the FDA for CUTX-101, a drug intended to treat Menkes disease. The CRL highlighted manufacturing practice deficiencies but did not raise concerns about the drug's efficacy or safety Fortress Biotech and Cyprium Therapeutics Receive Complete Response Letter from FDA for CUTX-101 NDA for Menkes Disease Treatment^[1].

Menkes disease is a rare pediatric condition characterized by severe neurological symptoms and caused by mutations in the copper transporter gene ATP7A. The FDA's CRL noted cGMP (current Good Manufacturing Practice) deficiencies at the manufacturing facility where CUTX-101 is produced. Sentynl Therapeutics, which assumed development and commercialization of CUTX-101 from Cyprium in December 2023, is responsible for addressing these issues and resubmitting the NDA Fortress Biotech and Cyprium Therapeutics Receive Complete Response Letter from FDA for CUTX-101 NDA for Menkes Disease Treatment^[1].

Sentynl expects to receive a Rare Pediatric Disease Priority Review Voucher from the FDA upon approval, which can provide significant future commercial opportunities for Cyprium. Additionally, Cyprium will benefit from potential royalty income and milestone payments from Sentynl, enhancing its revenue prospects. The total potential financial benefit to Cyprium is up to $129 million Fortress Biotech and Cyprium Therapeutics Receive Complete Response Letter from FDA for CUTX-101 NDA for Menkes Disease Treatment^[1].

The issuance of a CRL indicates significant regulatory issues with the NDA, potentially delaying the drug's market entry and affecting investor confidence. The CRL highlighted cGMP deficiencies, raising concerns about the quality control processes and reliability of the product. This negative impact on Cyprium Therapeutics could adversely affect Fortress Biotech, given Cyprium's majority ownership by Fortress Fortress Biotech and Cyprium Therapeutics Receive Complete Response Letter from FDA for CUTX-101 NDA for Menkes Disease Treatment^[1].