SteadyMed, Ltd., a company that develops products to treat orphan and high-value conditions with unmet parenteral delivery needs, recently announced that its lead product candidate, Trevyent® has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of pulmonary arterial hypertension (PAH).
Trevyent is a combination of the company’s PatchPump technology and the vasodilator treprostinil. Treprostinil is the only parenteral treatment for PAH that is approved by the FDA for both intravenous (IV) and subcutaneous (SC) administration. Trevyent is a small, discreet, single-use (2 day) PatchPump filled with treprostinil and programmed at the correct delivery rate. The PatchPump has sensors that provide patients visual and audible feedback on successful delivery and end of dosing.
The Orphan Drug Designation suggests that Trevyent may have clinical superiority over currently available treprostinil-based PAH treatments, such as Remodulin, the market leading parenteral prostacyclin sold by United Therapeutics.
“I am very pleased that Trevyent has been granted Orphan Drug Designation by the FDA for the treatment of PAH,” said Jonathan Rigby, president and chief executive officer of SteadyMed, in a press release. “Both parenteral and inhaled treprostinil have previously been granted Orphan Drug Designation, so I believe that this achievement supports our expectation that Trevyent, if approved, may offer a meaningful contribution to patient care compared to other oral, inhaled and parenteral prostacyclin treatments for PAH.”
PAH is a life-threatening, progressive disease caused by narrowing or tightening (constriction) of the pulmonary arteries that connect the right side of the heart to the lungs. There is currently no cure for the condition.
The Orphan Drug Act provides a special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. The Orphan Drug Designation qualifies the sponsor for various development incentives. This means that Trevyent may be eligible for tax credits for qualified clinical testing, and a waiver of the Prescription Drug User Fee Act filing fees. Upon approval, Trevyent may also be eligible to receive U.S. marketing exclusivity for seven years, possibly block other treprostinil-based products from coming to market.