After finding an intraventricular tumor in one clinical trial participant, the FDA placed a clinical hold on an experimental gene therapy developed by Regenxbio, a Rockville, Md.-based biotech company.
Regenxbio said in a Jan. 28 news release the patient is one of 10 participants in a phase 1/2 study for a Hurler syndrome gene therapy. Hurler syndrome is a severe condition with a life expectancy of about 10 years, according to the Cleveland Clinic.
Intraventricular tumors are typically benign tumors inside the brain’s ventricles. The tumor was detected during a brain MRI of a 5-year-old who received Regenxbio’s experimental gene therapy RGX-111 four years ago, according to the company.
The FDA placed a clinical hold on RGX-111 as well as on RGX-121, an experimental gene therapy for Hunter Syndrome.
“We are surprised by FDA’s decision to place our RGX-121 program on hold while the investigation of this single, inconclusive incident in RGX-111 continues,” Curran Simpson, president and CEO of Regenxbio, said in the release. “These are separate therapies, and the positive safety profile of RGX-121 in more than 30 patients treated, including those dosed nearly seven years ago, remains unchanged.”
The company said it is awaiting the full clinical hold letter from the FDA and that causality of the brain tumor has not been established.
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