Regenxbio Sues FDA Over Unexplained Clinical Trial Hold
Biotech company Regenexbio sued the US Food and Drug Administration (FDA) last week for failing to provide an explanation for placing a hold on a clinical trial for an experimental gene therapy to treat wet age-related macular degeneration.FDA’s decision effectively halted the development of the potential one-time subretinal treatment without a clear basis, the complaint said.
“By failing to provide advance notice of, or any reasoned basis for, the clinical hold, FDA violated the FDCA and its own regulations. FDA’s clinical hold on RGX-314 is therefore contrary to law and arbitrary and capricious under the Administrative Procedure Act,” the company said.
Timeline
Last month, the company said it was notified via voicemail and email from Edward Thompson, an FDA regulatory project manager, that two of the company’s INDs testing RGX-314 were on full clinical hold.
Thompson pointed to “issues associated with [the investigational drug’s] delivery systems” and “device concerns,” but the agency did not offer further detail until a week later, when Rachael Anatol, the deputy director of FDA’s Office of Tissues and Advanced Therapies, sent Regenxbio an email clarifying that one of the INDs was on a partial clinical hold, meaning current patients could continue to be treated, while the other IND was on a full hold.
The company then pulled the IND on a full hold, which was meant to test the therapy as a treatment of diabetic retinopathy.
Deborah Livornese, counsel at Hyman, Phelps & McNamara, wrote Monday: “While it seems unlikely that the merits of the clinical hold will be resolved through the judiciary process in a helpful timeframe, the complaint has likely increased the chances that FDA will respond with a thorough explanation of its reason for the hold when it does provide the written basis,” which is due on Friday.