FDA demands 2-year phase 3 from Aradigm in hard-hitting approval rejection

FDA
The FDA wants the trial to show the inhaled antibiotic has a significant effect on clinically meaningful endpoints. (FDA)

The FDA has told Aradigm it will need to run a two-year phase 3 trial before refiling for approval of its inhaled formulation of ciprofloxacin. That recommendation leaves Aradigm potentially needing to raise a sizable sum of money at a time when its stock is plumbing new depths.

Aradigm received multiple recommendations from the FDA in the CRL for Linhaliq in non-cystic fibrosis bronchiectasis patients with chronic lung infections. The request for data from a phase 3 trial that runs for two years or more dents hopes that Aradigm can quickly and cheaply address the FDA’s concerns and refile for approval. 

The FDA wants the trial to show the inhaled antibiotic has a significant effect on clinically meaningful endpoints, such as the co-primary endpoints of frequency and severity of exacerbations. Officials at the FDA think the trial will need to run for two years or more to show the durability of the efficacy of Linhaliq.

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Aradigm will need to run the trial while working to address other recommendations made by the FDA in the CRL. The agency thinks Aradigm should run a human factors study into the effectiveness of the product packaging and instructions, and get an independent third party to go over the source data from Aradigm’s previous phase 3 program to verify the results. 

The CRL also asked Aradigm to provide, “among other things,” more microbiology-related product quality information and a new in vitro drug release method development report. That gives Aradigm a sizable to-do list, the full extent of which is only known by the company and the FDA.

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Aradigm’s ability to check off all the items on the to-do list rests on its ability to raise additional money. The biotech’s bank balance currently falls short of what it is likely to need to keep the lights on and run a phase 3 trial, and its ability to raise the extra cash is hindered by its rock-bottom stock price, which took another 36% hit in premarket trading following news of the CRL. The drop followed prior double-digit hits in the wake of the FDA’s briefing documents and later advisory committee meeting. 

Management at Aradigm is remaining publicly upbeat in the face of the difficulties.

"We remain confident in the efficacy, safety and quality of Linhaliq and will request a meeting with FDA to discuss the topics covered in the CRL with the view to developing plans to move towards resubmission of the Linhaliq NDA as soon as possible,” Aradigm CEO Igor Gonda, Ph.D. said in a statement. “Our focus is also on the submission in the near future for marketing approval in the European Union.”