Liquidia gains FDA drug approval, makes first shipment of lung therapy

Morrisville-based Liquidia Corp., a biopharmaceutical company spun out of the University of North Carolina at Chapel Hill in 2004, is launching its first commercial therapy.

In May, the U.S. Food and Drug Administration approved Yutrepia, the company’s inhalation powder form of treprostinil, for the treatment of two types of cardiopulmonary disease: pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD).

“Today we celebrate the patients and physicians who will now have access to a potential best-in-class dry-powder form of treprostinil with exceptional portability, tolerability, titratability and durability,” said Roger Jeffs, chief executive officer of Liquidia. “With today’s milestone, our commercial team is prepared to launch Yutrepia and bring meaningful change to the lives of patients in need.”

Only five days after FDA approval of Yutrepia, Liquidia announced its first commercial shipment of the therapy, making prescriptions available to patients at specialty pharmacies. 

“We have moved with exceptional speed to provide a new and differentiated therapeutic alternative to the marketplace,” Jeffs said. 

Drug addresses rare diseases

PAH and PH-ILD affect about 105,000 patients in the United States.

“These patient communities and the physicians who serve them need therapies that can lead to the improvement of quality of life,” said Matt Granato, president and chief executive officer of the Pulmonary Hypertension Association. “We are always glad to see industry research leading to development of drugs that expand options for the patient community.”

PAH is a rare, chronic and progressive disease caused by narrowing, thickening or stiffening of the pulmonary arteries. It can lead to right heart failure and eventually death.

An estimated 45,000 patients are treated for PAH in the United States. There is no cure, so treatment goals are to alleviate symptoms, maintain or improve lung function, delay disease progression and improve quality of life.

PH-ILD includes up to 200 different pulmonary diseases that affect more than 60,000 patients in the U.S. 

Deep-lung delivery

Yutrepia was designed using Liquidia’s proprietary PRINT technology, which can produce drug particles that are precise and uniform in size, shape and composition, and that are engineered for enhanced deep-lung delivery. Yutrepia is inhaled using a “convenient, low-effort, palm-sized device,” according to the company.

Liquidia is also developing an investigational sustained-release formulation of treprostinil administered twice daily with a next-generation nebulizer.

Treprostinil is a prostacyclin analog that is a pulmonary and systemic vasodilator, opening blood vessels.

Yutrepia was approved by the FDA based on the outcomes of a Phase 3 clinical trial that showed the drug to be safe and well tolerated regardless of a patient’s previous exposure to treprostinil.

The commercial launch of Yutrepia coincided with a federal court ruling denying an attempt by United Therapeutics Corp. to prevent the launch. 

United, which has co-headquarters in Research Triangle Park and markets its own PAH drug, Tyvaso, had alleged patent infringement by Liquidia. The U.S. District Court for the Middle District of North Carolina denied United’s request for a preliminary injunction and a temporary restraining order that were intended to stop Liquidia from selling Yutrepia.

New facility leased, funding received

With the launch of Yutrepia, Liquidia has leased additional lab and manufacturing space in Morrisville. The company will expand into nearly 70,000 square feet at Pathway Triangle, a new advanced manufacturing campus built by Boston-based King Street Properties.

Liquidia also announced it had received an additional $50 million from a major royalty-based investor, HealthCare Royalty (HCR), under its sixth amendment of a funding agreement with HCR.

The funding will support the Yutrepia launch, advance Liquidia’s clinical product pipeline and support the build-out of the company’s newly leased manufacturing facility at Pathway Triangle.

Under its terms with HCR, Liquidia has now received $175 million of the $200 million in total potential funding. An additional $25 million is available if Liquidia meets sales milestones for Yutrepia.

Liquidia is a publicly held company. In the company’s early start-up years, the North Carolina Biotechnology Center helped Liquidia’s product-commercialization activities by funding postdoctoral industrial fellowships totaling more than $83,000.