WASHINGTON, June 15 (Reuters) - A new pill for a debilitating and often life-threatening lung disease won approval from U.S. health officials on Friday.
Letairis from Gilead Sciences Inc. <GILD> received Food and Drug Administration clearance for treating pulmonary arterial hypertension, or PAH.
Industry analysts estimate sales of the drug could top $1 billion a year. The generic name is ambrisentan.
PAH results from high blood pressure in the arteries around the lungs. Early symptoms of fatigue can progress to the point that minor exertion is extremely difficult.
About 100,000 people in the United States have PAH, the FDA said. The disease can occur as a complication of illnesses such as scleroderma and lupus, or for other unknown reasons.
In company studies of 393 patients, people who took Gilead's once-a-day pill were able to walk further on a standard six-minute walk test that measures endurance. Letairis also delayed the worsening of PAH, the FDA said.
The new drug is part of a family called endothelin receptor antagonists (ERAs), which block the action of a substance that narrows blood vessels and elevates blood pressure.
Swiss biotechnology company Actelion sells a drug in the same class called Tracleer, a twice-a-day pill. Actelion receives nearly 95 percent of its revenue from Tracleer sales.
"Letairis is similar to an existing drug, but offers the potential for fewer drug interactions," Dr. John Jenkins, director of the FDA's Office of New Drugs, said in a statement.
Pfizer also sells a version of Viagra for PAH under the name Revatio.
On Friday, Encysive Pharmaceuticals Inc. <ENCY> said the FDA again declined to approve its proposed PAH drug called Thelin.
The most common side effects from Letairis included leg and ankle swelling, nasal congestion, facial flushing and sinusitis, the FDA said.
Patients should have monthly blood tests to check for potential liver injury, the FDA said. The drug should not be used by pregnant women or women who may become pregnant because it may cause birth defects. Tracleer carries similar warnings.
A one-month supply will cost $3,940, said Kevin Young, Gilead's executive vice president of commercial operations. The drug will be available in the United States early next week.
Because of the risks of birth defects and liver injury, Letairis will be available only through prescribers, pharmacies and patients who sign up for a restricted distribution program, Gilead said.
Gilead acquired Letairis by buying its developer, Myogen Inc. <<
Thelin seems dead in the US. In addition to my thinking they won't be able to recruit patients for another trial some posters on iVillage pointed out several other reasons including: lack of funds/would be a couple years behind Letairis. The only chance I'd see for it if it is co-formulated with another class of drugs for PAH (the most likely candidate would be a longer acting PDE5 but Thelin is daily and Revatio and Cialis seem to have different dosing regimins).
From Gilead's PR, conference call yesterday and more so their Goldman Sachs call a couple days ago they seem all set for a launch and head-to-head competition with Tracleer/Actelion. I found it interesting they note the percent of patients not needing additional therapy after 1 year. Actelion has Ventavis (Inhaled Prostacylin) and was also studying both with PDE5's. I think it is more marketing/hype as most PAH opinion leaders think combination therapy is where treatment paradigms are heading.
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FDA Approves New Orphan Drug for Treatment of Pulmonary Arterial Hypertension
The U.S. Food and Drug Administration (FDA) today approved Letairis (ambrisentan) for the treatment of pulmonary arterial hypertension, a rare, life-threatening condition characterized by continuous high blood pressure within the arteries of the lungs.
"Letairis represents a valuable addition to the treatment alternatives for this orphan disease," said John Jenkins, M.D., director of FDA's Office of New Drugs. "Letairis is similar to an existing drug, but offers the potential for fewer drug interactions."
In pulmonary arterial hypertension, the small arteries in the lungs become narrowed or blocked, and the heart must work harder to pump the blood through them. Over time, the overworked heart muscle may become weak and lose its ability to pump enough blood through the lungs. Symptoms include shortness of breath, fatigue, chest pain, dizzy spells and fainting. About 100,000 people in the United States have pulmonary arterial hypertension.
Letairis, a new drug not previously approved in the United States, was granted a priority review by FDA. A priority review designation is intended for those products that address unmet medical needs. For priority drug applications, FDA sets a target date of six months after the date of receipt for the agency to complete all aspects of a review and to take action.
The safety and effectiveness of Letairis were demonstrated in two international clinical trials involving 393 patients. Letairis significantly improved physical activity capacity compared with a placebo, as shown by a six-minute walk, a standard test. Letairis also delayed the worsening of the pulmonary hypertension.
The most common side effects in patients using Letairis included swelling of legs and ankles, nasal congestion, sinusitis, and getting red in the face (flushing).
Letairis should not be used by women who are pregnant or may become pregnant because the drug may cause birth defects. Patients taking Letairis must have monthly blood tests to check for potential liver injury.
Letairis will be available in five-milligram and 10-milligram once-daily tablets.
Letairis was granted orphan drug status by FDA because it treats a rare disease and meets other criteria. Orphan designation qualifies the drug's sponsor for a tax credit and marketing incentives.
Letairis is manufactured by Gilead Sciences, Inc., Foster City, Calif. Gilead acquired the U.S. rights to ambrisentan when it acquired Myogen, Inc. in 2006. GlaxoSmithKline holds rights to ambrisentan outside of the United States.
For more information:
The Orphan Drug Act www.fda.gov/orphan/
National Heart Lung and Blood Institute – What is Pulmonary Arterial Hypertension? www.nhlbi.nih.gov/health/dci/Diseases/pah/pah_what.html
ENCY/GILD - How does the FDA approve a drug with significant safety issues, but fails to approve another drug for the same indication for failure to demonstrate effectiveness when three regulatory agencies have not questioned the efficacy data? The agency even admits that the data indicates an improved exercise tolerance. Somethings wrong with this picture.
evidently there was something in Encysive's data as far as safety that the FDA didn't like. I have no idea what it was and Given claimed he wasn't going to tell for compeititive reasons. That made sense to me. The fact the Gilead was approved and doesn't seem to have the same issue leads me to believe it wasn't a class effect but something different about thelin in particular and he was lying.
Again I don't know what the problem is and I don't know if it will ever come out. I can't see European doctors prescribing Thelin without knowing what the issue is.
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