In a 16-0 vote yesterday, a U.S. Food and Drug Administration (FDA) advisory committee recommended that the drug tesamorelin be approved for the treatment of HIV-associated lipodystrophy.

Although the FDA is not required to follow the recommendations of its advisory committees, it usually does.

Tesamorelin is being developed by Montreal-based Theratechnologies Inc. If approved, the drug would be marketed in the United States by EMD Serono, a unit of the German pharmaceutical company Merck KGaA. The proposed brand name for tesamorelin is Egrifta.

“I think that it’s a good day for us and I think that it should be a good day for the patients, as well,” said Yves Rosconi, CEO of Theratechnologies, in an interview with The AIDS Beacon.

“Some of the patients have said that they have waited 15 years to be able to treat this disease.”

HIV-associated lipodystrophy is a condition in which antiretroviral drugs cause abnormal fat redistribution, including excess fat deposits in the abdomen, breasts, and neck, and loss of fat from the arms, legs and face.

The FDA estimates that 200,000 to 800,000 people display symptoms of HIV-associated lipodystrophy. There are currently no approved treatments for the condition.

The committee recommended approval for tesamorelin due to its ability to reduce excess abdominal fat in people with HIV-associated lipodystrophy. The drug is injected daily into the abdominal region.

Since the fat loss typically disappears when tesamorelin treatment is stopped, most patients will have to take the drug on a long-term basis to sustain its effects.

Patients did not seem deterred by this prospect. “If given the opportunity, I would stay on tesamorelin for life,” said Dr. Lisa Hamilton, a participant in one of the tesamorelin clinical trials, during yesterday’s committee meeting. “This improved my quality of life so much.”

While stating that Theratechnologies had not demonstrated that the reduction in fat would lead to better health outcomes, the committee nonetheless felt the psychological benefits to patients, in terms of improved body image, outweighed the uncertainty of its medical benefit.

Jeff Berry, a representative from the AIDS Treatment Activists Coalition’s Drug Development Committee, urged the advisory committee not to underestimate the effects of improved body image on patient morale.

“This is no different than breast reconstruction following mastectomy,” he said during yesterday’s meeting . “We firmly believe that [tesamorelin] should be approved.”

The advisory committee also hopes that the drug will lead to higher adherence to antiretroviral regimens. Clinicians have been concerned that people with HIV may be halting or delaying treatment due to fears of lipodystrophy.

The advisory panel did raise some significant safety concerns. Clinical trials showed a slightly increased incidence of diabetes in patients receiving tesamorelin; additionally, tesamorelin raised levels of a protein called insulin-like growth factor 1 (IGF-1), which has been associated with increased cancer risk.

Since people with HIV are already at higher risk for cardiovascular problems and several forms of cancer, these issues are a concern. Dr. Victoria Cargill, a member of the advisory committee, was particularly worried about the increased risk of diabetes.

Many people with HIV are African-American or Hispanic, populations with increased risk of diabetes and cardiovascular problems. Since tesamorelin’s clinical trial population was mostly Caucasian, Dr. Cargill argued, the diabetes risk in these populations may be underestimated by the clinical trial results.

Additionally, the committee was concerned about the long-term effects of elevated IGF-1 levels. Since the beneficial impact of tesamorelin usually disappears once the treatment is stopped, patients are likely to take the drug indefinitely to maintain abdominal fat loss. This may mean living with permanently elevated levels of IGF-1, which could have health ramifications, particularly with respect to cancer rates.

The clinical trial data, which covered only a one-year time span, do not give enough information on long-term effects in patients, the committee found. The panel strongly recommended additional studies to assess these risks.

Nonetheless, Dr. Hamilton and Deborah Sergi-Laws, both participants in the tesamorelin clinical trials, were excited about the approval recommendation.

“I just hope not to wait too much longer,” said Ms. Sergi-Laws. “I’ve been hoping for [approval] since I went off it last. So definitely one of my first questions is, when can I get a hold of it?”

Dr. Hamilton agreed. “I would strongly suggest it. At least to try it. You know, some people are afraid of injections, but I’d at least recommend they try it. It’s that worth it.”

Theratechnologies representatives told the Beacon yesterday that they expect an FDA approval decision about tesamorelin by July 27. Before then, Theratechnologies plans to work with the FDA to find ways to address concerns about tesamorelin’s safety while still letting the drug be approved. Potential options include post-approval clinical trials or observational studies focused on tesamorelin’s safety.

For more information on the FDA advisory committee meeting, please see the AIDS Beacon liveblog of the event. Additional information about tesamorelin is available at the Theratechnologies website.