• The FDA has granted fast track designation to treprostinil inhalation powder in SSc-associated Raynaud’s phenomenon.
• The RE-WARM trial assessing the drug is set to begin in October.

The FDA has granted fast track designation to Yutrepia for the treatment of Raynaud’s phenomenon associated with systemic sclerosis, according to a press release from the manufacturer.

Yutrepia (Liquidia) is an inhalable dry-powder formulation of treprostinil designed to be administered via a “a convenient, low-effort, palm-sized device,” the release said. The drug is currently approved for the treatment of pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease.

“As someone who has cared for people with systemic sclerosis for many years, I view the FDA’s fast track designation as an important recognition by the regulator of the seriousness of Raynaud’s phenomenon in this disease, and the substantial unmet need that remains,” Dinesh Khanna, MD, MSc, director of the University of Michigan Scleroderma Program, told Healio.

Khanna is the lead investigator of the phase 2a RE-WARM trial, which will evaluate the safety and pharmacodynamics of Yutrepia in approximately 75 U.S. adults with SSc-associated Raynaud’s phenomenon across 30 sites. The study is expected to begin in October.

“Raynaud’s phenomenon affects approximately 95% of patients with systemic sclerosis,” he said. “For many patients, Raynaud’s is not simply an inconvenience caused by cold weather. Recurrent reductions in blood flow can produce significant pain, loss of hand function, digital ulcers, infection and, in the most severe cases, irreversible tissue damage including gangrene and amputations.”

These patients often go untreated, according to Khanna.

“Our treatment options remain limited, are frequently used off-label, and may not provide adequate benefit or may cause systemic side effects,” he said.

The FDA’s fast track process is designed to expedite the review of therapies intended to treat serious conditions with unmet needs. Companies whose drugs receive the designation are eligible for more frequent interactions with the FDA during clinical development.

“Fast track designation will facilitate more frequent communication with the FDA and a more efficient development and review process, and provide an opportunity to move a potentially meaningful therapy forward with greater regulatory collaboration and urgency,” Khanna said.

The impact of the designation is likely to be felt in clinics across the country, he added.

“For rheumatologists, this designation is significant because it acknowledges a clinical problem that we encounter routinely but still struggle to treat effectively,” Khanna said. “Despite the major impact of SSc-associated Raynaud’s phenomenon on pain, hand function, digital ulcers and quality of life, clinicians often have to construct treatment strategies from medications that were not specifically developed or rigorously evaluated for this condition,” he told Healio.

“Ultimately, our goal is to provide rheumatologists with something they urgently need: high-quality evidence and, if the trial is successful and the therapy is approved, an additional treatment option developed specifically for patients with SSc-associated Raynaud’s phenomenon.”

Dinesh Khanna, MD, MSc, can be reached at khannad@med.umich.edu.