Regenxbio sues FDA over gene therapy clinical hold

Regenxbio is taking the FDA to court over a clinical hold. The lawsuit alleges the FDA imposed the hold “without notice or explanation” and has since “rebuffed” Regenxbio’s repeated requests for an explanation of the regulatory action.

In the lawsuit, news of which was reported by FDA Law Blog, Regenxbio argues the legislation that gives the FDA the power to issue clinical holds is unconstitutional. Regenxbio wants the court to pass a judgment that the law is unconstitutional and issue a permanent injunction to set aside the partial and full clinical holds affecting its pipeline. 

The dispute began last month when, in Regenxbio’s telling of events, an FDA official told it two INDs were on a full clinical hold, effective immediately, without providing a reason. Regenxbio later learned the clinical hold is “due to issues associated with their delivery systems.”

Regenxbio wants a fuller explanation of the “issues” and “device concerns” that prompted the FDA clinical hold but claims it is being made to wait. The FDA allegedly told Regenxbio it would issue a letter outlining the deficiencies within 30 days, as is required by regulations, and later responded to “multiple communications” from the company by saying it will provide an explanation by Nov. 15.

The lack of warning and explanation are the main operational grievances identified in the lawsuit but the case gets at bigger topics with implications far beyond Regenxbio’s INDs. While some complaints raised in the lawsuit are specific to the FDA’s handling of Regenxbio’s INDs, the case also argues that Congress overstepped when it  empowered the agency to issue a clinical hold “for such other reasons as the [Health and Human Services] Secretary may by regulation establish.” 

Regenxbio wants the court to order the legislation unconstitutional. The company is also seeking “any other relief that this Court deems just and proper,” reflecting its belief it has been harmed by the clinical hold.  

“FDA’s actions have harmed, and continue to harm, Regenxbio. In addition to the reputational harm on Regenxbio caused by the clinical hold, Regenxbio is suffering actual and direct economic and competitive injury from the delay in development of these life-altering blindness treatments,” the lawsuit alleges.

The case is the second time this year that the FDA has been taken to court over a clinical hold. In February, Vanda Pharmaceuticals sought a legal fix to its partial clinical hold, arguing the FDA’s request for 9-month, non-rodent toxicity studies runs counter to scientific evidence. A federal judge returned the matter to the FDA in March. Vanda filed an amended complaint in May and a hearing is set for next month.