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

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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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.

A Conversation With Rare Disease Advocates