FDA triple play roundup: An approval, a denial and a response

Genentech gets good news for Actemra, Lilly gets bad news on liprotamase and GSK/Valeant press forward on retigabine

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SILVER SPRING, Md.—As the United States "celebrates" the deadline for filing of 2010 tax  forms today, the U.S. Food and Drug Administration (FDA) figures prominently in Monday's morning news. It dealt a blow to Eli Lilly and Co. with regard to liprotamase, a non-porcine pancreatic enzyme replacement therapy (PERT), under investigation for the treatment of exocrine pancreatic insufficiency (EPI) and gave a thumbs-up to Roche subsidiary Genentech for Actemra, for the treatment of a rare form of juvenile arthritis.

Looking to the good news first, Genentech got approval for Actemra (tocilizumab), whether given alone or in combination with methotrexate, for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in children ages 2 years and older.

SJIA, or Still's disease, is a rare, potentially life-threatening disorder in children that causes severe inflammation throughout the body. SJIA is distinguished from other forms of juvenile idiopathic arthritis (JIA) by the prominence of systemic and inflammatory features, including spiking fevers; rash; swelling and inflammation of lymph nodes, liver, and spleen; and high white blood cell and platelet counts. The prevalence of JIA is an estimated one to two per 1,000 children, and SJIA affects about 10 percent of all JIA patients.

Actemra is an interleukin-6 receptor blocker that had previously been approved by the FDA in January for treatment of adults with moderately to severely active rheumatoid arthritis who have had an inadequate response to other approved therapies.

"This new indication of Actemra provides the first approved therapy for children with this rare disease," says Dr. Badrul Chowdhury, M.D., Ph.D., director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA's Center for Drug Evaluation and Research.

In the bad news for the day, Eli Lilly reported that it had received a complete response letter from the FDA for its New Drug Application (NDA) for liprotamase to treat EPI, noting that the letter "communicated the need for Lilly to conduct an additional clinical trial prior to a re-submission."

"Lilly is looking forward to further discussion with the FDA to address the items outlined in the letter and provide the requested information as quickly as possible," says Dr. Eiry Roberts, vice president of autoimmune, bone-muscle-joint and liprotamase product development at Lilly. "We are committed to working with the agency and the cystic fibrosis community to make this important treatment option available to patients."

People with EPI have very low levels or none of the key digestive enzymes made by the pancreas—lipase, amylase and protease—in their small intestine, resulting in improper digestion and absorption of fat, carbohydrates and proteins, often requiring treatment with PERTs. EPI occurs very frequently in patients with cystic fibrosis and in other diseases including chronic pancreatitis and pancreatectomy.

In other news of the day, the FDA reported that GlaxoSmithKline (GSK) and Valeant Pharmaceuticals International Inc. had submitted a response to its complete response letter of Nov. 30 for an NDA for retigabine (known as ezogabine outside the United States). The drug is an investigational anti-epileptic drug being studied for the adjunctive treatment of adults with partial-onset seizures.

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