FDA advisory committee recommends tesamorelin acetate to reduce HIV-associated lipodystrophy

The FDA Endocrinologic and Metabolic Drugs Advisory Committee voted unanimously — 16-0 — in favor of the approval of tesamorelin acetate, a growth hormone-releasing factor, for the treatment of excess abdominal fat in HIV patients with lipodystrophy.

The drug is being developed by the Montreal-based Theratechnologies under the name Egrifta. It is an analogue of growth hormone-releasing hormone designed to induce and maintain a reduction of excess visceral abdominal fat with HIV-associated lipodystrophy. The drug is a sterile lyophilized powder for injection.

Committee member Allison B. Goldfine, MD, said Theratechnologies laid out a development plan and met the goals.

“There is no other treatment available [for HIV-associated lipodystrophy] and there is equipoise for risks. I don't take lightly that things were revealed that have potential risks, but there is also potential benefit,” said Goldfine, assistant director of clinical research at Joslin Diabetes Center.

According to an FDA Advisory Committee briefing document, clinical trial evidence supports the following potential benefits of tesamorelin acetate:

Reduction of visceral adipose tissue
Preservation of abdominal subcutaneous adipose tissue and limb fat
Improvement of “belly appearance distress” and “belly profile”
Reduction in waist circumference and trunk fat
Improvement in lean body mass and other body composition measures
Improvement in lipid parameters (triglycerides, total cholesterol, total cholesterol/HDL ratio, non-HDL)

The committee expressed some concern about the following potential risks associated with tesamorelin:

An increase in insulin-like growth factor I levels associated with a theoretical risk of cancer
Small number of patients with worsening of glucose homeostasis
Hypersensitivity reactions
Presence of antibodies

“I think that the risk–benefit ratio is greatly in favor of benefit. The company did a very nice job of presenting their data, but I think that they did a terrible job of giving any type of firm recommendations to physicians on how to use this medication,” David M. Capuzzi, MD, PhD, professor of medicine and biochemistry at Thomas Jefferson University and Lankenau Institute for Medical Research, Philadelphia, said during the meeting.

Capuzzi said a professional society should produce guidelines on how to best use this drug, with focus on the following three points: the course of action for a patient who has high IGF-I levels; the approach for patients with diabetes, particularly if treatment does not show deterioration of glucose control; and creation of treatment intervals and monitoring recommendations for patients who do not respond to the drug.

While the FDA is not required to follow the recommendations of the advisory committee, it usually does. The FDA has indicated that the target date for the agency to complete its review tesamorelin will be July 27.

For more information, please visit the FDA website.