LIQ861, Inhaled Treprostinil for PAH, Back Up for FDA Approval

An updated application has been filed with the U.S. Food and Drug Administration (FDA), again asking that LIQ861 be approved as an inhalation treatment of pulmonary arterial hypertension (PAH), its developer Liquidia announced.

The company first made this request in an application to the FDA submitted in April 2020. But the agency responded in November with a request for more data on the therapy and its use, in the form of complete response letter. FDA and Liquidia officials then met in January to discuss specific requirements of a second approval request for LIQ861.

Filed on May 7, the updated application includes more information and clarification of the therapy’s chemistry, manufacturing, and controls, Liquidia announced in a press release. More data was also given regarding the biocompatibility — essentially, further evidence of its safety — for the portable device used to administer LIQ86. Additional clinical trial data, or information related to the medication’s toxicology or clinical pharmacology were not required.

“The team has worked hard to provide a comprehensive and rapid response … We look forward to working with the FDA through the review process during the coming months,” said Damian deGoa, Liquidia’s CEO.

If the FDA accepts the new application, Liquidia anticipates an approval decision being announced within six months of its resubmission or by Nov. 7.

On-site inspections of  manufacturing facilities for LIQ861 are also required for approval, and the FDA noted these inspections were impossible throughout much of 2020 due to travel limitations under the pandemic. Liquidia “remains prepared to conduct these inspections at any time,” it stated in its release.

LIQ861 is a dry powder formulation of treprostinil, administered using a portable inhaler. Treprostinil is a vasodilator — a compound that causes blood vessels to relax and widen, lowering blood pressure. LIQ861 is based on Liquidia’s proprietary PRINT technology, reported to allow for the production of highly uniform drug particles that are optimized for delivery into the lungs.

Liquidia’s initial FDA application was supported by findings from a Phase 3 clinical trial, INSPIRE (NCT03399604), which demonstrated that LIQ861 was safe and well-tolerated, and improved exercise capacity and quality of life in adults with PAH.