Pharma, BioPharma

United Therapeutics notches another FDA approval for lung drug

The FDA decision for Tyvaso as a treatment for pulmonary arterial hypertension in patients who have interstitial lung disease is the second approval for the United Therapeutics drug. The additional indication gives the company another way to grow revenue for the drug, which faces generic competition.

 

United Therapeutics has received FDA approval for its drug, treprostinil, as a treatment for high blood pressure affecting the lungs in patients who have a group of rare respiratory disorders called interstitial lung diseases.

The regulatory nod is the second for the inhaled version of treprostinil, which was initially approved in 2009 as a treatment for pulmonary arterial hypertension—high blood pressure in the arteries that carry blood from the heart to the lungs. Silver Spring, Maryland-based United Therapeutics also sells the drug in injectable and oral formulations. Together, the three versions of treprostinil generated nearly $1.3 billion in sales last year.

The inhaled formulation of treprostinil, marketed as Tyvaso, accounted for $483.3 million in 2020 sales. However, treprostinil has lost patent protection, so approval in a new indication gives United Therapeutics a way to grow revenue as the company faces the prospect of generic competition.

Interstitial lung disease (ILD) is a group of diseases characterized by scarring in the lungs that causes breathing problems and makes it difficult for oxygen to reach the bloodstream. Symptoms include shortness of breath while active, labored breathing, and fatigue. Exposure to hazardous materials and autoimmune diseases can lead to ILD, though in many cases the exact cause is unknown, according to the Mayo Clinic. The American Lung Association notes that lung damage from the disorder is often irreversible and worsens over time.

ILD can be complicated by pulmonary hypertension—high blood pressure in the lungs. According to United Therapeutics, an estimated 15% of patients (about 30,000) with early-stage ILD have pulmonary hypertension. In more severe ILD, pulmonary hypertension may affect up to 86% of patients, the company said. Prior to Thursday’s approval of Tyvaso, there were no FDA-approved therapies for ILD patients who have pulmonary hypertension.

The FDA based its decision on data from a double-blind study that enrolled 326 patients randomly assigned to receive Tyvaso or a placebo for 16 weeks. United Therapeutics reported that the study met the main goal of demonstrating statistically significant improvement as measured by distance in a six-minute walk test. The study also showed improvements in forced vital capacity, which is a measure of how much air can be exhaled from the lungs, as well as fewer flare-ups of symptoms that restrict breathing.

United Therapeutics reported that its drug, given four times daily in up to 12 breaths per session, was well tolerated with a safety profile consistent with previous Tyvaso studies. The most common problems reported in those clinical trials included cough, headache, nausea, and dizziness. Results of the latest clinical trial were published in January in the New England Journal of Medicine.

United Therapeutics said it will make Tyvaso available to ILD patients immediately. With the approval for Tyvaso in a second indication, the company expects the number of patients treated with the drug will double by the end of next year, barring any delays related to Covid-19, Michael Benkowitz, president and chief operating officer, said in a prepared statement.

Remodulin, the injectable version of treprostinil, is United Therapeutics’ top-selling version, accounting for $516.7 million in 2020 sales. In its annual report, United Therapeutics said that generic competition has so far caused “minimal erosion” of Remodulin sales.

One way United Therapeutics has been trying to maintain sales for its top drug is by acquiring companies developing competing versions. In 2018, the company reached a $216 million deal to acquire SteadyMed, a San Ramon, California-based company developing a system called Treyvant that administered treprostinil via a skin patch and disposable pump.

The FDA rejected Treyvant last year. Last month, United Therapeutics disclosed that the regulator asked for a redesign of the product and another clinical trial to test the new design. The company concluded that continued development of Treyvant was “no longer commercially reasonable” and it stopped work on the product. But by then, another United Therapeutics product had already made Treyvant moot. In February, the company launched Remunity Pump, a pump system that uses disposable cartridges filled with treprostinil, which is administered subcutaneously. That product was developed under a partnership with Deka Research and Development Corp.

Photo: ThitareeSarmkasat, Getty Images

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