FDA Announcement on Regulatory Flexibility for Cell and Gene Therapies Brings Hope to the MPS Community
The National MPS Society welcomes the FDA’s recent announcement, Flexible Requirements for Cell and Gene Therapies to Advance Innovation, which outlines a more adaptable regulatory approach to supporting the development of cell and gene therapies. This initiative by the FDA recognizes the unique scientific and manufacturing challenges inherent to these transformative treatments and aims to help expedite development while maintaining safety and quality standards.
“For the MPS community, progress in policy is not abstract—it directly impacts the lives of children and families who cannot afford to wait,” said Terri Klein, President and CEO at the National MPS Society. “We welcome the FDA’s focus on flexibility and innovation and have emphasized in our letter the urgent and critical needs of our community as we continue to advocate for timely access to promising therapies.”
As part of its continued advocacy efforts, the Society has submitted a letter to FDA Commissioner, Martin A. Makary, M.D., M.P.H., emphasizing the critical needs of our community, including the importance of regulatory flexibility that accelerates access to safe and effective therapies for people living with MPS. You can click here to read the full letter.
