Liquidia Technologies announced that the US Food and Drug Administration (FDA) has accepted its new drug application (NDA) for LIQ-861.

LIQ-861 is a dry powder inhaled formulation of treprostinil that is under development for the treatment of pulmonary arterial hypertension (PAH), based on the company’s novel PRINT technology, which enhances deep-lung drug delivery. Despite LIQ-861’s mechanism of action not being novel, GlobalData believes that its clinical development is significant because of its less cumbersome dosing administration than the currently available PAH treatment options.

If its development is successful, LIQ-861 will have an edge over other already established marketed treprostinil drugs, namely United Therapeutics’ Orenitram, Remodulin, and Tyvaso. LIQ-861 is also likely to be favoured by patients and physicians as it will likely increase patient compliance.

LIQ-861 may be a more convenient treatment as it is formulated to deliver the maximum dose with one to two breaths from a small, palm-sized inhaler, in comparison to nine breaths from Tyvaso’s nebulized treprostinil. The process of inhaling Remodulin is cumbersome, as the process takes five to ten minutes and includes both assembling and cleaning the device.

Because Orenitram (an oral formulation of Remodulin) is an oral tablet taken two to three times per day, patients can experience increased severe prostacyclin-like side effects such as gastrointestinal issues. Key opinion leaders interviewed by GlobalData have emphasized that current therapies have not shown dramatic benefits in patients and survival rates remain disappointing. Therefore, LIQ-861 represents a more convenient, low-toxicity option for PAH treatment.

Treprostinil acts as prostacyclin receptor agonist. The drug candidate assists in the direct vasodilation of the pulmonary and systemic arterial vascular beds and helps the inhibition of platelet aggregation. Activation of prostacyclin receptors results in the vasodilatation of human pulmonary arteries, inducing a beneficial pulmonary vasorelaxant effect. The prostacyclin analogues, GlaxoSmithKline’s Flolan (epoprostenol), Actelion’s Veletri (epoprostenol), and Actelion’s Ventavis (iloprost), are approved to the target prostacyclin pathway in PAH patients. The only prostaglandin prostacyclin receptor agonist approved to target the prostacyclin pathway is Actelion’s Uptravi (selexipag).

As the company’s leading product, LIQ-861’s NDA application was supported by positive clinical data from multiple clinical trials, including the open-label, Phase III INSPIRE clinical study, which assessed the safety, tolerability, and pharmacokinetic characteristics of the drug. Results showed that LIQ-861 can be effectively titrated to therapeutic levels through a comprehensive range of inhaled doses and was well tolerated. The FDA is expected to make a decision on whether LIQ-861 will receive approval for PAH in November 2020.