Decrying 'arbitrary and capricious' action, RegenxBio sues FDA over clinical holds on gene therapy
When RegenxBio disclosed that the FDA had placed a partial clinical hold on one of its lead gene therapies, execs outlined several customary next steps: continuing assessment and monitoring, delaying a related IND filing, and working with the FDA to address the matter.
As it turned out, they were planning something much less mundane. Two days after announcing the hold in its Q3 update, RegenxBio filed a lawsuit seeking to set it aside, the FDA Law Blog noted.
The lawsuit shed light on the interactions between the biotech and regulators, revealing that there was actually a full clinical hold on the diabetic retinopathy trial in addition to the partial hold on wet age-related macular degeneration and RegenxBio withdrew the IND.
According to the complaint, the company had discussed results from its Phase I/IIa trial with the FDA and was on track to begin the next phase before the end of 2019.
But on October 18, 2019, without notice or explanation, FDA placed RGX-314 on a clinical hold, effectively halting REGENXBIO’s development of this potentially life-altering treatment for retinal diseases that are leading causes of adult blindness. Since issuing the clinical hold order, FDA has rebuffed REGENXBIO’s repeated attempts to obtain an explanation of the basis for the clinical hold.
Failing to provide advance warning or explanation for the hold, RegenxBio claims, is in violation of the FDA’s own regulations. And that led to an “arbitrary and capricious” final decision, they wrote.
But that’s not it.
Since they didn’t get a chance to review or rebut the reasons for the hold, which harmed their reputation and property interest in RGX-314, RegenxBio believes the FDA violated the Fifth Amendment’s Due Process Clause. Furthermore, they charged a particular section of the Food, Drug, and Cosmetic Act represented an unconstitutional vesting of legislative power in the Secretary of Health and Human Services.
Along with the agency, the federal government, HHS Secretary Alex Azar, acting FDA commissioner Brett Giroir (who has taken over from Ned Sharpless while Stephen Hahn goes through the confirmation process), and FDA regulatory project manager Edward Thompson were also named as defendants. Thompson allegedly first notified RegenxBio of the holds citing “issues associated with [RGX-314’s] delivery systems.”
Following multiple exchanges, the FDA apparently told RegenxBio that it would provide a written explanation of the basis for the hold by this Friday, November 15.
Why risk the ire of regulators when an update is due so soon? As a procedural matter to preserve their rights, the company said.
“This action was taken on the recommendation from counsel as we continue to work with the FDA to address this matter, and we hope this step will help ensure the FDA will provide RegenxBio with their specific concerns about the unspecified device,” it wrote in an email to Endpoints News.
Attorney Deborah Livornese offered this take on the FDA Law Blog:
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.
The biotech added that the plan is still to start the wet AMD trial and file an IND for diabetic retinopathy in Q2 2020, “as we believe that there are readily available and suitable alternatives for all of the devices used in our studies.”