Shire gets FDA boost – but suffers trial setback
pharmafile | April 10, 2015 | News story | Research and Development, Sales and Marketing | FDA, Shire, algs, lifitegrast, shp625
Shire has achieved mixed results in its attempts to get regulatory approval for two new treatments – bagging a priority review for one but failing to show benefits in a mid-stage trial of another.
A Phase II study of SHP625, an investigational treatment for a genetic disorder called Alagille syndrome (ALGS), failed to meet either its primary or secondary endpoints in a 13-week trial in 20 children.
ALGS is a rare and life-threatening genetic disorder that causes accumulation of bile acids in the liver and severe itching. SHP625 blocks bile acids from being absorbed back into the digestive system, and so reduces bile acids in the liver.
The primary endpoint in its IMAGO study was the change from baseline in blood serum bile acid levels compared to placebo, while the secondary endpoint was a reduction in itching.
Average serum bile acid levels and pruritus were lower in both SHP625 and placebo treated groups at the end of the study. But a post-hoc analysis showed a positive correlation between the changes in serum bile acid levels and itchy symptoms in the SHP625 treated group, which could not be statistically explained due to the small size of the placebo group.
“We have gained important insights from these first results from one of several Phase II studies in the SHP625 development programme,” says Philip Vickers, head of R&D at Shire. “We remain committed to continuing the ongoing studies of SHP625 in ALGS and other indications.”
The company is still conducting two larger Phase II studies of SHP625, and the treatment is also being looked at in patients with progressive familial intrahepatic cholestasis, primary biliary cirrhosis and primary sclerosing cholangitis.
In the US however, the FDA has accepted another investigational Shire offering, lifitegrast, for a priority regulatory review as a treatment for dry eye disease in adults. If approved, it could be the first drug with an indication to treat both signs and symptoms of the disease. Under the terms of an FDA priority review, the timeline is cut from 12 to eight months – giving a provisional decision date of 25 October this year.
“Our commitment to moving lifitegrast forward reflects our intent to grow in the ophthalmics therapeutic category in areas of unmet patient need. We look forward to working closely with the FDA throughout the review process,” adds Vickers.
“The symptoms of dry eye are one of the most common complaints from patients, yet there remains a tremendous unmet need,” says Professor Stephen Pflugfelder, from Baylor College of Medicine in Houston. “It’s encouraging to see Shire moving the program for lifitegrast forward.”
Lilian Anekwe
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