The U.S. Food and Drug Administration rejected SteadyMed’s new drug application (NDA) for Trevyent (treprostinil), a treatment for pulmonary arterial hypertension (PAH). According to the FDA’s Refusal to File letter, the application requires more information on device specifications and performance testing.
Additional design verification and validation testing were also requested by the FDA to allow the NDA validation and review.
Trevyent combines SteadyMed’s PatchPump delivery device with treprostinil, a prostacyclin-based therapy known for its action as a strong vasodilator. This investigative drug with delivery device was designed to overcome the limitations and complexity of existing treprostinil delivery systems. The inhaled form of treprostrinil was approved by the FDA in 2009; it can also be taken as an infusion or orally.
Small and discreet, Trevyent is a single-use system that can deliver the vasodilator drug subcutaneously or intravenously for two days. It is pre-filled and pre-programmed to deliver treprostinil at the prescribed therapeutic delivery rate. The system also has sensors to alert patients with audible and visual signals of successful delivery and end of dosing.
SteadyMed submitted the NDA for Trevyent in June 2017 based on positive results of safety and tolerability profiles from a study of 60 healthy volunteers. Trevyent was granted FDA orphan drug status for the treatment of PAH in January 2016.
SteadyMed will now request a meeting with the FDA within 30 days for further clarification on the requirements needed to achieve a successful resubmission of its NDA.
“We believe that the issues raised in the letter from FDA can be sufficiently addressed. Our next step is to work with the FDA to address the open issues and identify a path to a successful resubmission and acceptance of our application,” Jonathan M. N. Rigby, president and CEO of SteadyMed, said in a press release.
“We believe that Trevyent holds the potential to significantly improve the lives of patients suffering from PAH compared to the current standard of care, and remain committed to bringing the product to patients in need,” Rigby added.
While SteadyMed follows all the requirements to achieve FDA approval for commercializing Trevyent in the U.S., its partner Cardiome Pharma will pursue marketing authorization for the therapy in Europe, Canada, and the Middle East.