FDA approves inhaled treprostinil for pulmonary hypertension associated with ILD

The FDA approved inhaled treprostinil for the treatment of patients with pulmonary hypertension associated with interstitial lung disease, according to a press release from United Therapeutics.

This approval was based on data from the INCREASE study, a phase 3, multicenter, randomized, double-blind, placebo-controlled study that enrolled 326 patients with WHO group 3 pulmonary hypertension associated with ILD. Patients were randomly assigned to inhaled treprostinil (Tyvaso, United Therapeutics; n = 163) or placebo (n = 163) four times daily.

After 16 weeks, inhaled treprostinil significantly improved 6-minute walk distance vs. placebo, and showed benefit across several subgroups, including etiology of pulmonary hypertension-associated ILD, disease severity, age, sex, baseline hemodynamics and dose. The researchers also observed improvement in secondary endpoints including NT-proBNP, time to first clinical worsening event, change in peak 6-minute walk distance at 12 weeks, change in trough 6-minute walk distance at 15 weeks, placebo-corrected improvements in forced vital capacity and fewer exacerbations of underlying lung disease. Treatment with inhaled treprostinil of up to 12 breaths per session four times daily was well tolerated and safe, according to the release.

“Adults living with both interstitial lung disease and pulmonary hypertension typically have a poor quality of life because of increased shortness of breath, poor exercise tolerance and increased mortality,” Aaron Waxman, MD, PhD, director of the pulmonary vascular disease program at Brigham and Women’s Hospital and chair of the INCREASE Study Steering Committee, said in the release. “Until now, clinicians treating these patients did not have any approved treatment options. The regulatory approval of Tyvaso, an inhaled treatment, is exciting news both for patients with [pulmonary hypertension-associated ILD] and the physicians who treat adults living with this serious, life-threatening disease. This will change the way we manage these patients.”

Tyvaso was first approved in 2009 for the treatment of pulmonary arterial hypertension to improve exercise ability.

“The FDA approval of Tyvaso for patients with pulmonary hypertension associated with ILD is a landmark treatment advancement for this vulnerable patient population,” Martine Rothblatt, PhD, chairperson and CEO of United Therapeutics, said in the release. “It also underscores our commitment to driving innovation in the field of pulmonary hypertension and expanding the number of patients who can achieve a clinical benefit from Tyvaso. We plan to tap into our experience and expanded infrastructure to bring this safe and effective inhaled therapy to the many patients living with pulmonary hypertension associated with ILD in the United States.”