RARE Daily

FDA Grants United Therapeutics Priority Review for PAH Drug

June 18, 2021

The U.S. Food and Drug Administration granted United Therapeutics priority review the New Drug Application for Tyvaso DPI for the treatment of pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease.

Photo: Martine Rothblatt, chairperson and CEO of United Therapeutics

United Therapeutics expects the agency’s review to be complete in October 2021. The agency also indicated that they have not identified any potential review issues at this time.

Pulmonary arterial hypertension (PAH) is life-threatening high blood pressure in the arteries of the lungs, affecting the ability of the heart and lungs to work properly in afflicted patients. PAH is a serious, progressive disease for which there is no cure.

Interstitial lung disease (ILD) is a group of lung diseases that are characterized by significant scarring or fibrosis of the bronchioles and alveolar sacs within the lungs. Increased fibrotic tissue in ILD prevents oxygenation and free gas exchange between the pulmonary capillaries and alveolar sacs, and the condition can present with a wide range of symptoms, including shortness of breath with activity, labored breathing and fatigue. Pulmonary hypertension (PH) frequently complicates the course of patients with interstitial lung disease and is associated with worse functional status measured by exercise capacity, greater supplemental oxygen needs, decreased quality of life, and worse outcomes. An estimated 30,000 patients in the United States may suffer from PH-ILD, which is included within Group 3 of the World Health Organization (WHO) classification of PH. Only Tyvaso Inhalation Solution is approved to treat patients with this disease.

Tyvaso DPI is an investigational drug-device combination therapy comprised of a dry powder formulation of treprostinil and a small, portable, dry powder inhaler. If approved, Tyvaso DPI is expected to provide a more convenient method of administration compared with traditional nebulized Tyvaso therapy. United Therapeutics is developing Tyvaso DPI under a collaboration and license agreement with MannKind. United Therapeutics and MannKind are also developing BluHale, a Bluetooth-connected accessory for the Tyvaso DPI inhaler with a companion mobile application intended to help the patient track information about inhaler use.

“The acceptance of the Tyvaso DPI NDA for review represents an important regulatory step toward offering this meaningful new product to both PAH and PH-ILD patients,” said Martine Rothblatt, chairperson and CEO of United Therapeutics. “If approved, Tyvaso DPI will represent yet another path to help us achieve our goal of serving 25,000 patients by the end of 2025.”

Author: Rare Daily Staff

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