Fortress Bio and Zydus' Rare Pediatric Treatment Denied FDA Approval

The FDA has declined to approve Fortress Bio and Zydus' treatment for a rare pediatric disease. Fortress Biotech is a biopharmaceutical company focused on acquiring and advancing assets, with seven marketed prescription pharmaceutical products and over 25 programs in development. The company's products span six market areas, including oncology, rare diseases, and gene therapy.

The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding Fortress Biotech's (Nasdaq: FBIO) New Drug Application (NDA) for CUTX-101, a treatment for Menkes disease in pediatric patients. This decision comes after the FDA noted cGMP deficiencies at the manufacturing facility, despite positive clinical efficacy results for CUTX-101.

Menkes disease is a rare, X-linked recessive pediatric disorder caused by mutations in the copper transporter ATP7A. The condition is characterized by distinctive clinical features such as sparse and depigmented hair, connective tissue problems, and severe neurological symptoms. The FDA's CRL did not cite any other approvability concerns or deficiencies in CUTX-101's efficacy and safety data, but it did highlight the need for manufacturing facility improvements.

In December 2023, Sentynl Therapeutics, Inc., a wholly-owned subsidiary of Zydus Lifesciences, Ltd., assumed responsibility for the development and commercialization of CUTX-101. Sentynl expects to address the FDA's concerns and pursue resubmission promptly. If approved, Sentynl will transfer a Rare Pediatric Disease Priority Review Voucher (PRV) to Cyprium Therapeutics, Inc., Fortress Biotech's majority-owned subsidiary, and Cyprium will also be eligible for royalties on net sales of CUTX-101 and up to $129 million in aggregate development and sales milestones.

Fortress Biotech, an innovative biopharmaceutical company, focuses on acquiring and advancing assets to enhance long-term value for shareholders. The company has eight marketed prescription pharmaceutical products and multiple programs in development across various therapeutic areas, including oncology, dermatology, and rare diseases. The company has established partnerships with leading academic research institutions and biopharmaceutical companies to maximize opportunities.

The FDA's decision to decline approval for CUTX-101 may impact Fortress Biotech's financial outlook and shareholder expectations. The company's ability to successfully address the FDA's concerns and secure approval for CUTX-101 will be crucial for its future growth and profitability.