Specialty pharmaceutical company SteadyMed Ltd. has filled an application requesting orphan drug designation for its lead medical product, an investigational therapy to treat pulmonary arterial hypertension (PAH) called Trevyent. The application has been acknowledged by the U.S. Food and Drug Administration (FDA) and the company will now wait for a decision from the agency once the review process is complete.
Orphan drug status is granted to therapies and other biological products meant for the treatment of rare conditions, which is determined by the FDA as those affecting fewer than 200,000 people in the United States. Alternatively, the FDA can also grant the designation if the medical product demonstrates clinical superiority when compared to other drugs already approved for the treatment of the same orphan disease.
PAH has been diagnosed in about 30,000 individuals throughout the country, making it a rare but also progressive and life-threatening condition that can cause premature death due to heart failure. If Trevyent is awarded orphan drug designation, the company will receive several incentives including tax credits, a New Drug Application (NDA) submission fees waiver, eligibility for NDA priority review, and seven years of marketing exclusivity following the drug’s approval.
“SteadyMed continues to execute on our strategic goals including our submission of this request for Orphan Drug Designation,” the president and CEO of SteadyMed, Jonathan M. N. Rigby in a press release, who also disclosed some of the future plans of the company for the new therapy. “We look forward to a decision from the FDA as we continue to drive to submission of an NDA for Trevyent in the first quarter of 2016.”
Trevyent is a development-stage drug product that is delivered via SteadyMed’s PatchPump to administer treprostinil, which is a vasodilatory prostacyclin analogue for the treatment of PAH. The PatchPump is described by the company as a novel, compact, pre-filled, preprogrammed, disposable, parenteral drug administration platform, and currently neither Trevyent or the PatchPump are currently FDA-approved.
SteadyMed recently raised $12.2 million in equity financing to invest in the development process of Trevyent. The business was co-led by subsidiaries of Federated Investors Inc. and Deerfield Management Company L.P., as well as existing shareholders from the company, and as a result of this transaction, William Slattery, a partner at Deerfield, will be joining the Board of Directors of SteadyMed.
In addition, the company established last June a development and manufacturing agreement with Bespak Europe, a Consort Medical company and supplier of drug delivery devices for injectable and inhaled products. The two companies will join efforts to produce Trevyent, as they have already collaborated in the development of the novel PatchPump device, which will be used for the parenteral delivery of treprostinil.