The U.S. Food and Drug Administration (US FDA) has sought more time to review the additional data submitted by United Therapeutics Corp on inhalalable treprostinil (Tyvosa) to treat pulmonary arterial hypertension.
Treprostinil’s injectable and intravenous versions are currently sold in the market by United Therapeutics under the brand name Remodulin.
The Food and Drug Administration will delay its decision for another three months from April 30 to July 30 to review additional study data.
Following this, United Therapeutics said review process for the New Drug Application (NDA) for Tyvaso (treprostinil) will likely extend beyond its originally anticipated action date.
US FDA had raised questions concerning human factors testing and the instructions for use (IFU) of the OptiNeb portable nebulizer that delivers Tyvaso.
“In response to FDA comments related to our originally submitted instructions for the OptiNeb nebulizer, we provided the FDA with a revised IFU. Following our most recent discussion with the FDA, we have agreed to conduct human factors testing to validate that the revised IFU results in the proper use of the device by a naive user,”said Roger Jeffs, Ph.D., United Therapeutics’ president and chief operating officer.
This relatively small study in normal subjects will test the new IFU in its ability to guide patients to accomplish such tasks as proper device assembly, drug administration, disassembly and cleaning, among other things, Mr Rogers explained.
United Therapeutics Corporation is a biotechnology company focused on the development and commercialization of treatments for chronic and life-threatening cardiovascular and infectious diseases and cancer.
United Therapeutics warned of the possibility of delay last month after FDA officials said the company needed to test patients’ ability to follow instructions to use and maintain the portable nebulizer that delivers the inhaled drug. The company turned in data from that human study this month.
Treprostinil is a synthetic analogue of prostacyclin PGI2. Treprostinil effects vasodilation, which in turn lowers the blood pressure. Treprostinil also inhibits platelet aggregation.
Pulmonary hypertension is increased pressure in the pulmonary arteries -the arteries carry blood from the heart to the lungs to pick up oxygen. Pulmonary hypertension is a rare disease with an incidence of about 2-3 per million per year. It has a prevalence of about 15 per million. Adult females are almost three times to have Pulmonary hypertension than adult males.
Only about 1.1% of patients with chronic obsessive pulmonary disease (COPD) develop pulmonary hypertension. Sleep apnea is also associated with very mild pulmonary hypertension, typically below the level of detection.
The USFDA delay is the latest of the series of obstacles United Therapeutics had to face while diversifying its Remodulin franchise. An oral version of the drug failed its clinical trials according to results released last fall. At the time, United Therapeutics attempted to soften the blow with news that it signed a licensing agreement with Eli Lilly & Co. to help develop and market a formulation of the larger pharmaceutical’s Cialis drug for pulmonary hypertension.