Mallinckrodt blames manufacturing issue for FDA rejection of its rare kidney disorder med

Efforts by three companies to get terlipressin across the FDA finish line for a rare kidney condition have spanned more than a decade. And, just when it appeared Ireland-based Mallinckrodt might finally be poised to get it done, terlipressin has been hit with yet another setback.

Tuesday, the FDA issued a complete response letter for Mallinckrodt’s new drug application for the drug’s use on those with hepatorenal syndrome (HRS), a life-threatening disorder which features a rapid loss of kidney function and has no approved treatment.

This glitch, however, might be solvable without too much stress.

Over the last two weeks, Mallinckrodt determined it would be necessary to identify a new manufacturing facility for the packaging and labeling of the drug, the company said. With the change, it became clear that the inspection of the new site couldn’t be accomplished by the agency's target approval date.

“We are working with the new facility and have it ready for inspection by the FDA,” Steven Romano, M.D., chief scientific officer of Mallinckrodt, said. “We believe there is a path to approval in 2022.”  

There were no efficacy or safety issues with the drug, Mallinckrodt noted.

RELATED: FDA rejects Mallinckrodt's kidney drug over risk-benefit doubts

Indicated for a variety of conditions, terlipressin has yet to make the grade for HRS. It hasn’t been for lack of trying.

In September of 2020, Mallinckrodt appeared on the verge of winning approval when an advisory committee voted 8-7 in favor of recommending a green light. A phase 3 trial had shown the benefit of the drug but the FDA ultimately rejected terlipressin because of uncertainty of benefit over risk.

That CRL was met with disappointment from Romano, who said Mallinckrodt would pursue “all available options,” to get the drug approved.

More than 10 years earlier, Orphan Therapeutics was the first to try to get the drug indicated for HRS but came up short in a phase 3 trial. After Ikaria picked up the North American rights to terlipressin, it too flunked a late-stage study.

After Mallinckrodt bought Ikaria in 2016—primarily to acquire its nitric oxide drug Inomax—the FDA helped convince Mallinckrodt to take another swing at HRS.

RELATED: Crippled by opioid-crisis litigation, Mallinckrodt settlement brings company closer to reorganization

The CRL comes along with another bit of news about the company as a Delaware bankruptcy court approved a $67.75 million settlement between Mallinckrodt and investors who claimed that the company concealed its reliance on federal reimbursements for its lucrative Acthar gel product, Law360 reports.

Last year the troubled company agreed to a $1.7 billion settlement to resolve opioid claims while also undergoing a Chapter 11 restructuring.