The treatment cannot be granted full approval due to ongoing litigation.
The US Food and Drug Administration (FDA) has granted tentative approval for treprostinil (YUTREPIA) inhalation powder for the treatment of pulmonary arterial hypertension (PAH).
The treatment, developed by Liquidia Corporation, is specifically earmarked to improvement the exercise ability of adult patients with New York Heart Association (NYHA) Functional Class II-III symptoms.
The medication includes an ease-of-use, convenient, direct lung delivery and higher dosage range platform that makes treprostinil a preference to nebulized therapy and an alternative to oral treatments.
The tentative approval means the company has met all the regulatory standards for quality, safety, and efficacy required for full approval in the US. It’s also possible the treatment is an option to delay the use of parenteral therapies in patients with PAH.
The approval was based on the results of the Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil (INSPIRE) study, an open-label, multi-center phase 3 clinical trial in which patients with PAH who are naïve to inhaled Treprostinil or who are transitioning from nebulized treprostinil (Tyvaso).
"The tentative approval for YUTREPIA is another step toward providing an important option for patients with PAH in the US,” Tushar Shah, MD, Chief Medical Officer of Liquidia, said in a statement. “We believe YUTREPIA can improve the limitations of current nebulized therapies by allowing the administration of an expanded dose range of inhaled treprostinil using a proven, convenient, palm-sized device.”
The treatment cannot yet be marketed in the US because of a regulatory stay pursuant to the Drug Price Competition and Patient Term Restoration Act in which United Therapeutics filed a lawsuit against Liquidia for alleged infringement of three patents related to Tyvaso. Because of this, the FDA is unable to grant final approval until the expiration of the regulatory stay on October 27,2022, unless there is earlier resolution or settlement of the ongoing litigation.