FDA Priority Review Likely for Tyvaso DPI, Inhaled Treprostinil for PAH

United Therapeutics is planning to ask the U.S. Food and Drug Administration (FDA) for an accelerated review of Tyvaso DPI, an investigational therapy-inhaler device treatment for two forms of pulmonary hypertension, in an approval request it expects to file by midyear.

The request, in the form of a new drug application to the agency, will make use of a rare pediatric disease priority review voucher that United Therapeutics paid $105 million to acquire under a recent definitive purchase agreement, the company announced in a press release.

Priority review shortens the FDA’s consideration of a new drug’s approval application to eight months, rather than the standard one year.

“This acquisition affirms our commitment to bring a new generation of treatments to patients with pulmonary hypertension as quickly as possible,” said Michael Benkowitz, president and chief operating officer of United Therapeutics.

Tyvaso DPI combines a dry powder inhaled formulation of treprostinil — a vasodilator agent that can widen blood vessels in the lungs, lowering blood pressure — and the Dreamboat dry powder inhalation device. The device was approved by the FDA in 2014 for use alongside Afrezza, an inhaled formulation of insulin, to treat type 1 and 2 diabetes; both the treatment and its inhalation device were developed by MannKind.

Formerly known as Treprostinil Technosphere, Tyvaso DPI is being co-developed by United and MannKind to treat people with pulmonary arterial hypertension (PAH), and those with pulmonary hypertension associated with interstitial lung disease (PH-ILD).

If approved, Tyvaso DPI will give these patients access to an alternative — and potentially more convenient, according to United — treatment option to Tyvaso, United’s nebulized formulation of treprostinil that was first approved by the FDA in 2009 for use with a hand-held nebulizer.

“Once approved, Tyvaso DPI with the innovative Dreamboat device is expected to be a major advancement in the delivery of inhaled treprostinil therapy, offering substantial convenience compared to the existing Tyvaso nebulizer,” Benkowitz said. 

Two other trepostinil-based therapies marketed by United are approved to treat PAH in the U.S. They are Remodulin, a formulation given as a continuous infusion or injected under the skin or into the bloodstream, and an oral extended-release formulation called Orenitram.