Humira for Ulcerative Colitis May Not Be Effective: FDA Staff

An FDA advisory panel is scheduled to meet today to review whether to recommend the agency expanded the approved uses for Humira to include treatment of ulcerative colitis. However, a staff report suggests that the popular medication may not be effective for treatment of the bowel condition and may require additional studies.

Humira (adalimumab) is one of the top selling drugs in the United States, generating sales of about $9 million a year.

The manufacturer, Abbott Laboratories, is seeking approval for additional uses for the medication, which is currently authorized by the FDA for treatment of rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, moderate to severe chronic psoriasis and juvenile idiopathic arthritis.

In documents prepared by FDA staff (PDF) in advance of the advisory panel meetings, questions were raised about whether a less than 10% difference in effectiveness between a placebo and Humira for ulcertive colitis treatment was “meaningful” enough to widen use of the drug for this new indication.

Ulcerative colitis is an inflammatory bowel disease that affects nearly 700,000 Americans, impacting the lining of the intestines and colon. Symptoms of the disease include abdominal pain, blood in the stool, diarrhea, fever, fatigue and weight loss and is typically treated with corticosteroids, immunomodulators or Remicade if patients do not respond to other treatments.

The FDA rejected Abbott’s initial application for approval to use Humira to treat ulcerative colitis last year, citing the drug ineffectiveness at relieving symptoms such as diarrhea, rectal bleeding, abdominal pain, fever and fatigue.

Humira is part of a class of medications known as tumor necrosis factor blockers. Johnson & Johnson’s Remicade (infliximab) is another medication in this class, which is currently approved to treat both rheumatoid arthritis and ulcerative colitis, but it must be administered intravenously by a physician. Humira, on the other hand, is a patient administered injection.

Abbott proposes that patients would gain the benefit of convenience with Humira over Remicade, however the FDA staff indicated that “moving patients from an effective treatment to potentially less effective treatment, while still exposing them to the comparable serious safety risks, is a concern.”

Potential side effects of Humira may include a risk for serious infections such as tuberculosis, malignancies, Hepatitis B virus reactivation, neurological reactions and heart failure. The FDA has also indicated that Humira may be linked to Hepatosplenic T-Cell Lymphoma, a rare cancer of white blood cells.

Abbott has suggested that the new use of Humira for ulcerative colitis could potentially add more than $1 billion in sales. Abbott is also testing Humira in other conditions, such as pediatric Crohn’s disease.