Although it must conduct a Phase IV trial, United Therapeutics Corp. received FDA approval to market Remodulin for the treatment of pulmonary arterial hypertension in patients with New York Heart Association Class II through Class IV symptoms to diminish symptoms associated with exercise.
The company received in February an approvable letter for Remodulin (treprostinil sodium) and at that time, UTC said it expected final approval once the label for the product was clearly defined and the design of a Phase IV trial agreed upon. (See BioWorld Today, Feb. 12, 2002.)
"I cannot overexpress the excitement that we all feel at having been able to work together for six years, thinking always of the patients' best interest foremost in our minds, and then come to this point of having an approvable therapy to share with them," UTC CEO Martine Rothblatt said in a conference call. "It's a wonderful sense of accomplishment; it's a wonderful sense of fulfillment."
The Phase IV trial will be designed to verify and describe Remodulin's clinical benefits. Silver Spring, Md.-based UTC will conduct a randomized transition of patients treated with Flolan to either subcutaneous Remodulin or placebo, while measuring clinical deterioration, pulmonary arterial hypertension and exercise performance. Flolan (epoprostenol) is an intravenous therapy marketed by GlaxoSmithKline plc, of London.
"[The trial] will start this year and needs to be completed in the next two years," said Fred Hadeed, chief financial officer at UTC. "One of the things [the FDA] is looking for is some assurance that patients can be transferred from Flolan to Remodulin and be maintained on Remodulin."
That assurance would only spell good news for UTC, considering Flolan is administered intravenously, Remodulin subcutaneously, and there are 2,600 patients taking Flolan in the U.S. And although in the clinical studies of Remodulin, the most common side effect was infusion-site pain (among 85 percent of patients), UTC President and Chief Operating Officer Roger Jeffs said in the conference call that, for "the large part, that side effect can be handled."
"About 85 percent of patients that start on the therapy remain on the therapy without any dropout due to infusion-site pain," he said. "There is a small number of patients that cannot tolerate it long term."
About 50,000 people in North America and Europe are estimated to have NYHA Class II through Class IV forms of pulmonary arterial hypertension, the company said.
Remodulin, formerly UT-15 and also Uniprost, is available now through UTC's U.S. distributors, Priority Healthcare Corp. and Gentiva Health Services. UTC said the recommended price for the drug is $65 per milligram, which works out to a ballpark figure of $50,000 per patient per year, Hadeed told BioWorld Today. The company has submitted marketing applications for Remodulin in France, Switzerland and Canada and other international filings would follow approval in the U.S. and France. Overall, UTC has formed six distribution partnerships for countries or regions outside the U.S.
UTC posted a net loss of about $5.7 million for the first quarter. It had $162.2 million in cash, cash equivalents and marketable securities as of March 31. While not making Remodulin revenue projections at this time, Hadeed said the company has $9 million worth of orders on hand, which suggests the company may be approaching the hallowed ground of the profitable.
UTC now will focus its attention on growing Remodulin. The company has completed a Phase II study in patients with critical limb ischemia and is expected to begin a Phase II/III trial in that population in the next 12 months. Also, last month it acquired most of Waltham, Mass.-based AltaRex Corp.'s cancer portfolio, including OvaRex. UTC expects to start a Phase III trial of OvaRex in ovarian cancer patients in the first quarter of 2003, Hadeed said. (See BioWorld Today, April 23, 2002.)
Lower in the pipeline, UTC is planning to begin Phase I work on UT231B for hepatitis C sometime in the next 12 months.
But for now, UTC can sit back and reflect on past statements made good.
"[The approval] means we have delivered on our promise to our patients, the [clinical investigators] and our shareholders," Hadeed said. "We feel very good about the prospect of treating hundreds, if not thousands, of patients with Remodulin and turning the corner toward profitability."
UTC's stock (NASDAQ:UTHR) rose $1.06 Thursday to close at $14.71.