Bristol drug for fat disorder may work, but risky -FDA staff

Reuters

By Ransdell Pierson

Dec 9 (Reuters) - An experimental Bristol-Myers drug totreat rare and potentially fatal disorders involving loss ofbody fat appears effective for some patients although withconsiderable safety risks, staff members of the U.S. Food andDrug Administration said on Monday.

The staff observations were posted on the FDA Website aheadof a planned meeting on Wednesday by a panel of medical experts,who will advise the FDA on the drug, called metreleptin.

The briefing document, meant to flag potential safetyconcerns about the drug, noted it has been tested since 2000 bythe U.S. National Institutes of Health.

Bristol-Myers, which has commercial rights to themedicine, is awaiting U.S. approval for metreleptin, to treatdiabetes and/or lipodystrophy, a group of rare acquired orinherited disorders associated with loss of body fat.

Only a few thousand people worldwide are believed to havethe disorders, in which fat builds up in the blood and organssuch as liver and muscle, and can lead to diabetes, pancreatitisand fatty liver disease. There are no approved drugs to treatunderlying causes of the disease, including deficiencies of thehuman hormone leptin that occur with loss of fat tissue underthe skin.

Metreleptin is a form of leptin meant to reduce accumulationof fat in organs, and thereby better control blood sugar andhigh levels of triglyercides - a type of fat in the bloodstream.Bristol-Myers is developing it in partnership with AstraZenecaPlc.

The FDA briefing document noted that all patients in the NIHtrial received metreleptin, and there was no separate groupreceiving placebos.

Without a placebo group, it is "challenging to attributebeneficial changes to metreleptin" versus improvements in dietor improved compliance in taking other medicines, the documentsaid.

"Nevertheless, a subgroup of patients appears to haveachieved benefits from metreleptin that would be unlikely tohave been achieved spontaneously," the report said, includingnormalization of metabolic control. Benefits were most apparentin patients who had uncontrolled diabetes.

Safety of the drug is hard to gauge, the report said, giventhe array of medical problems that patients with the disordersalready have and the possibility that metreleptin may worsencertain conditions.

It cited concerns about potentially higher risks oflymphoma, immune-related reactions, hypoglycemia, pancreatitisand liver abnormalities.

"It is important to ensure that the appropriate patientpopulation is targeted, given the serious risks that may beassociated with the drug," the briefing report said.

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