The FDA has placed clinical holds on RegenXBio's investigational gene therapies RGX-111 and another for rare neurodevelopmental disorders, including mucopolysaccharidosis type I (Hurler syndrome). This decision follows the discovery of a neoplasm in a trial participant, impacting ongoing studies.
The FDA has placed clinical holds on a pair of investigational gene therapies developed to address two rare neurodevelopmental disorders in children, according to the manufacturer. In a press release, RegenXBio Inc. said that a study of RGX-111 for the treatment of mucopolysaccharidosis type ... The FDA has placed clinical holds on a pair of investigational gene therapies developed to address two rare neurodevelopmental disorders in children, according to the manufacturer. In a press release, RegenXBio Inc. said that a study of RGX-111 for the treatment of mucopolysaccharidosis type I (MPS I), also known as Hurler syndrome, has been halted due to a single case of neoplasm (intraventricular CNS tumor) discovered in a participant treated in a phase 1/2 clinical trial. “As independent researchers, clinicians and patient advocates advancing novel approaches for rare diseases, we share in the field’s deep sadness that a trial participant receiving an experimental gene therapy experienced a serious adverse event,” Jeff Auletta, MD, senior vice president at the National Marrow Donor Program and co-scientific director of the Center for International Blood and Marrow Transplant Research, told Healio. The FDA put a similar hold on a study investigating RGX-121 to treat mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, due to similarities between the products and the clinical trials. “The FDA is wrong on several fronts,” Scott Loiler, PhD, chief scientific officer of the National MPS Society, told Healio. “Current standard of care for newly diagnosed MPS in children is hematopoietic stem cell transplantation, which has a mortality rate of greater than 10%, or enzyme replacement therapy, which does not cross the blood-brain barrier, so kids have neurological decline over time. “The FDA does not have a clear scientific rationale to hold RGX-121 other than they are not prepared to decide on their [biologics license application] and this is just another mechanism to delay these life-changing therapies from reaching the patients in desperate need,” Loiler told Healio. Jeff Auletta, MD, and Scott Loiler, PhD, can be reached at [email protected]. ... Disclosures: Loiler is employed by the National MPS Society. Auletta is employed by the National Marrow Donor Program. Ask a clinical question and tap into Healio AI's knowledge base.
Original title: “FDA places clinical holds on gene therapies for rare neurodevelopmental conditions”