FDA Rejects Regenxbio's Gene Therapy for Rare Disease MPS II
The U.S. Food and Drug Administration has declined to approve Regenxbio's gene therapy designed to treat mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. The rare genetic disorder causes progressive damage to multiple organ systems and primarily affects boys, with limited existing treatment options available to patients.
The FDA's rejection represents a major blow to Regenxbio, which had positioned the therapy as a potentially transformative one-time treatment for MPS II patients. The specific reasons for the rejection likely involve concerns about the therapy's efficacy data, safety profile, or manufacturing processes, though the details of the FDA's complete response letter will inform the company's next steps.
The decision highlights the continued challenges facing gene therapy developers as they work to bring advanced treatments for rare diseases through the rigorous regulatory approval process. For the small community of MPS II patients and their families, the rejection delays access to what was hoped to be a breakthrough treatment, though Regenxbio may pursue additional studies or resubmission to address the FDA's concerns.
The FDA's rejection represents a major blow to Regenxbio, which had positioned the therapy as a potentially transformative one-time treatment for MPS II patients. The specific reasons for the rejection likely involve concerns about the therapy's efficacy data, safety profile, or manufacturing processes, though the details of the FDA's complete response letter will inform the company's next steps.
The decision highlights the continued challenges facing gene therapy developers as they work to bring advanced treatments for rare diseases through the rigorous regulatory approval process. For the small community of MPS II patients and their families, the rejection delays access to what was hoped to be a breakthrough treatment, though Regenxbio may pursue additional studies or resubmission to address the FDA's concerns.