Shire ($SHPG) has resubmitted the once-rejected eye treatment lifitegrast for FDA approval, handing in new data the company hopes will get its self-described blockbuster drug onto the market.
The drug, rejected in October, is a small-molecule therapy designed to interrupt the immune process that leads to dry eye disease. In its complete response letter, the FDA asked for more data on lifitegrast's efficacy, unconvinced by a prior Phase III trial in which the treatment succeeded on only one of its two primary endpoints. Shire followed up with exactly that, touting a third late-stage study showing that the drug charted a statistically significant reduction in patient-reported eye dryness compared with placebo after 12 weeks and met secondary goals of improving symptoms from baseline at weeks two and 6.
Now Shire has completed its resubmission, giving the FDA 30 days to accept the second application and trigger what Shire expects to be a 6-month review period for lifitegrast.
"Because we believe that, if approved, lifitegrast has the potential to help the millions of U.S. adults living with symptoms of dry eye disease, we worked diligently to submit our response to the CRL as quickly as possible," Shire R&D chief Philip Vickers said in a statement.
Shire has long said that lifitegrast has the potential to bring in more than $1 billion a year at its peak, and management has often cited the drug as a cornerstone of the company's future.
But Shire's pipeline landscape has shifted over the past year. In April, the liver disease drug SHP625, star of the company's $260 million acquisition of Lumena Pharmaceuticals, failed in a Phase II study. And in October, Shire paid Lumena's shareholders $90 million to nix all future milestone payments as it decides what to do with SHP625 and a second related asset, reassessing some pipeline projects it once said could bring in $2 billion a year.
|Shire CEO Flemming Ornskov|
But Shire CEO Flemming Ornskov remains confident that the company's pending $32 billion merger with Baxalta ($BLXT), expected to close this summer, will transform it into a powerhouse in the rare disease field. Adding Baxalta's sizable presence in hemophilia and pipeline of oncology therapies will help Shire grow its revenue to $20 billion by 2020, Ornskov has said.
- read the statement