Silver Spring’s United Therapeutics just cleared the next hurdle in its effort to bring another product across the regulatory finish line.
The company said Wednesday that the Food and Drug Administration accepted for priority review its new drug application for Tyvaso DPI, an inhaler that dispenses a dry powder form of pulmonary arterial hypertension (PAH) drug treprostinil. That means the agency will determine whether the product should receive the green light to go to market as a treatment for PAH and PAH associated with interstitial lung disease.
Two months after it submitted its application to the agency, United Therapeutics said it expects the review to be finished in October, and that the FDA “indicated that they have not identified any potential review issues at this time.” The Maryland drugmaker sought the go-ahead to take Tyvaso DPI to market after buying a priority review voucher to speed up the regulatory process. That positioned UT to receive a decision by the FDA by December at the latest.
An approval of the application depends on an FDA inspection of the Tyvaso DPI manufacturing facility in Danbury, Connecticut. That site is run by Westlake Village, California-based MannKind Corp. (NASDAQ: MNKD), UT’s development partner for Tyvaso DPI following a 2018 license agreement. The inspection, according to United Therapeutics, is slated to happen in the third quarter.
That the FDA accepted UT’s application for review “represents an important regulatory step toward offering this meaningful new product," chairperson and CEO Martine Rothblatt said in a statement Wednesday.
Tyvaso DPI would serve an addressable U.S. market of at least 75,000 patients. That includes 45,000 PAH patients, plus roughly 30,000 additional patients with interstitial lung disease — which causes scarring of the lungs and hinders breathing — who currently have no treatment options. Tyvaso DPI consists of a small inhaler that fits in a pocket, and doesn’t require batteries or an external power source, making it a more convenient option than existing nebulized Tyavso therapy, the company said.
If it receives approval, the product “will represent yet another path to help us achieve our goal of serving 25,000 patients by the end of 2025,” Rothblatt said in a statement. It would also further company’s goal of doubling the number of patients using Tyvaso therapies by the end of 2022, Michael Benkowitz, UT's president and chief operating officer, said in a statement in April. That’s critical to the biotech’s income, as sales of its existing Tyvaso formulation increased 16% year-over-year to $483.3 million in 2020.
That UT is advancing Tyvaso DPI also reflects its need to innovate in other areas, as its top-selling drug, Remodulin, saw sales wane 12% to $516.7 million in 2020, due largely to generic competition and Covid-19, according to the company.
Tyvaso has faced similar threats, and is the subject of a lawsuit that continues to play out. United Therapeutics filed the suit in June 2020 against Morrisville, North Carolina-based Liquidia Technologies Inc. for patent infringement, and said June 7 it’s now pursuing additional claims for trade secret misappropriation against Liquidia and a former United Therapeutics employee.
The employee was “central to Tyvaso’s development” and then became an executive with that business and allegedly brought over confidential UT documents relating to Tyvaso, UT said earlier this month. The Silver Spring company filed its amended complaint June 4, and the defendants have until July 2 to respond, according to court filings. Liquidia declined to comment for this story, citing active litigation. The case is scheduled for trial in March 2022.
Despite the litigation and competition, the company’s products that use treprostinil — Remodulin, Tyvaso and Orenitram — accounted for nearly $1.3 billion in sales in 2020, according to SEC filings. United Therapeutics closed the year with $1.48 billion in net revenue, as its patient base using treprostinil therapies hit an all-time high in the fourth quarter.
The company is now studying treprostinil's inhaled form for other uses, while also expanding beyond its core PAH business and into the pulmonary fibrosis space. United Therapeutics (NASDAQ: UTHR) saw its share price climb to a high Wednesday of $184.78, up 3.6%, in the early afternoon.
