The rejection comes a week after the FDA placed clinical holds on RGX-121 and a separate therapy for a different rare disease, Hurler syndrome, when a treated study participant with the latter condition developed a brain tumor. The FDA extended the hold to both products because of certain similarities, as well as a “shared risk” between the two studies. Regenxbio has countered that no causal link was established and no similar issues were identified in all of the 32 trial ... The rejection comes a week after the FDA placed clinical holds on RGX-121 and a separate therapy for a different rare disease, Hurler syndrome, when a treated study participant with the latter condition developed a brain tumor. The FDA extended the hold to both products because of certain similarities, as well as a “shared risk” between the two studies. Regenxbio has countered that no causal link was established and no similar issues were identified in all of the 32 trial participants who have received RGX-121. The decision comes two weeks after the agency halted testing due to safety concerns and represents the latest regulatory setback for a gene therapy maker. However, the FDA ultimately decided that, while it agreed “in principle” to the design of the clinical trial Regenxbio built its application around, multiple issues with its results persisted. The regulator wasn’t convinced by the surrogate measure Regenxbio used to assess the treatment’s effectiveness, and was skeptical of the study’s comparisons between historical data and the group receiving the therapy. The Food and Drug Administration rejected a gene therapy Regenxbio has been developing for a rare disease called Hunter syndrome, in a decision that marks the latest blow for a field that has seen multiple recent regulatory setbacks in the U.S.

Original title: “FDA rejects Regenxbio treatment in another blow to gene therapy | BioPharma Dive”

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”

The cancer case was reported in ... for the rare enzyme deficiency Hurler syndrome, Regenxbio announced on Jan. 28. The inherited disease results in the buildup of compounds in tissue, leading to organ dysfunction and central nervous system problems. Enzyme replacement therapy is a standard treatment, but engineered enzymes do not cross the blood brain barrier. RGX-111 delivers a gene to central nervous system cells to bring ... The cancer case was reported in a Phase 1/2 test of RGX-111, a potential treatment for the rare enzyme deficiency Hurler syndrome, Regenxbio announced on Jan. 28. The inherited disease results in the buildup of compounds in tissue, leading to organ dysfunction and central nervous system problems. Enzyme replacement therapy is a standard treatment, but engineered enzymes do not cross the blood brain barrier. RGX-111 delivers a gene to central nervous system cells to bring production of the deficient enzyme to the CNS. Tests of two Regenxbio gene therapies have been placed under an FDA clinical hold after a patient in one of the studies developed cancer. Our recap of recent regulatory news also includes one clinical hold removed, several complete response letters, and drug approvals in the U.S. and Europe. —The ultra-rare inherited neurodegenerative disorder Menkes disease has its first FDA-approved therapy, a drug developed by Sentynl Therapeutics. In Menkes, a genetic defect impairs the ability to absorb and transport copper, which is essential for certain bodily processes. —Intellia Therapeutics has FDA permission to resume Phase 3 testing of the gene therapy nexiguran ziclumeran (nex-z) as a potential treatment for polyneuropathy associated with the rare disease hereditary transthyretin amyloidosis (ATTR). Last October, the agency placed a clinical hold on this study and a separate Phase 3 test in ATTR cardiomyopathy after a patient in the cardiomyopathy study developed liver complications.

Original title: “Clinical Trial Holds Cast Doubt on Approval Chances of Regenxbio Gene Therapy for Rare Disease - MedCity News”

Regenxbio said the abnormal growth ... for the treatment of severe mucopolysaccharidosis type I (MPS I). Also known as Hurler syndrome, the rare disease can cause developmental delays and shorten life span. RGX-111 is designed to improve outcomes by using an AAV9 vector to deliver the IDUA gene to the CNS. While the adverse event occurred in a recipient of RGX-111, the FDA extended the clinical hold to cover RGX-121 because of similarities between the therapies, trial populations ... Regenxbio said the abnormal growth was found in a recipient of RGX-111, a gene therapy the company is developing for the treatment of severe mucopolysaccharidosis type I (MPS I). Also known as Hurler syndrome, the rare disease can cause developmental delays and shorten life span. RGX-111 is designed to improve outcomes by using an AAV9 vector to deliver the IDUA gene to the CNS. While the adverse event occurred in a recipient of RGX-111, the FDA extended the clinical hold to cover RGX-121 because of similarities between the therapies, trial populations and shared risks of the studies. The FDA put clinical holds on two Regenxbio gene therapies, including a candidate that is less than two weeks away from an approval decision. A central nervous system (CNS) tumor has prompted the FDA to REGENXBIO clinical hold FDA Adverse Events tumor Cell & Gene Therapy Biotech

Original title: “FDA puts clinical hold on Regenxbio gene therapies weeks before approval ruling”