RARE Daily

United Therapeutics Acquires Rare Disease Priority Review Voucher

December 28, 2020

Rare Daily Staff

United Therapeutics said it has entered into an agreement to acquire a Rare Pediatric Disease Priority Review Voucher from Y-mAbs for $105 million, which it plans to use with a forthcoming New Drug Application with the U.S. Food and Drug Administration.

The voucher entitles the holder to designate a new drug application with the FDA for priority review and provides for an expedited review that is four months shorter than the standard review period. Following the close of the transaction, United Therapeutics intends to apply the voucher to its application for Tyvaso DPI, a drug-device combo that uses inhaled dry powder for delivery for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). The company plans to begin the NDA filing in the first half of 2021.

“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.  

Pulmonary arterial hypertension (PAH) is a rapidly progressing disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation. PAH results in significant strain on the heart, often leading to limited physical activity, heart failure, and reduced life expectancy. The five-year survival rate for patients with PAH is approximately 57 percent. Available therapies generally act by promoting the dilation of pulmonary vessels without addressing the underlying cause of the disease.

Tyvaso DPI is an inhaler version of United Therapeutics’ hypertension medicine Tyvaso, a prostacyclin vasodilator approved in 2014 for the treatment of pulmonary arterial hypertension to improve exercise ability. The company is currently seeking a supplemental approval for Tyvaso to expand its labeling to include PH-ILD.

Photo: Michael Benkowitz, president and chief operating officer of United Therapeutics  


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