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Serostim receives full FDA approval for treatment of AIDS wasting

ROCKLAND, MASS., August 29, 2003 -- Serono, Inc. announced that the FDA has granted full approval for Serostim [somatropin (rDNA origin) for injection] which is indicated for the treatment of HIV patients with wasting or cachexia to increase lean body mass and body weight, and improve physical endurance. (1)

In 1996, Serostim received accelerated approval, a special regulatory status granted by the FDA for approval of a drug that is used to treat patients with serious or life-threatening illnesses, and provides meaningful therapeutic benefit over any existing treatments. Under the terms of the accelerated approval, Serono conducted a multi-center, confirmatory placebo- controlled study with Serostim. Data from this trial substantiate previous study findings of increased lean body mass and improvement in physical endurance with Serostim. In addition, patients in this study perceived an improvement in their wasting symptoms with Serostim treatment.

"We are very pleased that the FDA has granted full approval for Serostim," said James Sapirstein, Executive Vice President, Metabolic Endocrinology, Serono, Inc. "Wasting continues to be a major concern in the management of HIV and AIDS treatment, even with the benefits of highly active antiretroviral therapy. Serostim makes an important and positive difference in the physical endurance of people experiencing HIV wasting."

HIV-associated wasting is a chronically debilitating and potentially life- threatening condition. It is a metabolic disorder that causes the body to use vital muscle and organ tissue, which is critical for survival, for energy instead of primarily using the body's stored fat. Loss of lean body mass, which consists of muscle tissue, important body organ tissue and blood cells, can lead to increased risk of opportunistic infections, illness, and extreme fatigue and can profoundly diminish a person's quality of life. Serostim was granted orphan drug designation by the FDA in 1991 for this condition.

The full approval of Serostim is based upon a randomized, double-blind, dose-ranging study that confirmed the clinical efficacy of Serostim in the treatment of HIV wasting. The study treated 757 patients with HIV-associated wasting at US, European and other international trial sites. Participants were treated with placebo, Serostim on alternate days (0.1 mg/kg) or Serostim on a daily basis (0.1mg/kg).

The result on the study's primary endpoint of cycle work output was statistically significant for the Serostim daily dose group as compared to the placebo group (p<0.0001). Positive results were also seen for the secondary endpoint of change in lean body mass for the Serostim daily dose group as compared to the Serostim alternate day group (p=0.017) and as compared to the placebo group (p<0.0001). In addition, patient perception of the impact of treatment on their wasting symptoms was positive with both doses of Serostim.

All study participants completing the 12-week placebo-controlled phase of the trial (n=646) continued treatment into an extension phase; 548 of these patients completed an additional 12 weeks of active treatment. In these patients, positive results in cycle work output and lean body mass either improved further or were maintained with continued treatment with Serostim.

Serostim [somatropin (rDNAorigin) for injection] is the only growth hormone approved by the US Food and Drug Administration for the treatment of HIV wasting or cachexia. The recommended dose is 0.1 mg/kg daily (6 mg/day for patients > 55 kg). Serostim 0.1 mg/kg every other day should be considered as a starting dose in patients thought to be at risk of certain adverse effects, i.e., glucose intolerance.

Serostim is generally well tolerated. The most common adverse events associated with Serostim therapy are mild to moderate muscle and joint pain and swelling/edema, which occur in a dose-related manner and often subside with continued treatment or dose reduction. Cases of new onset impaired glucose intolerance, new onset type 2 diabetes mellitus and exacerbation of preexisting diabetes mellitus have been reported in patients receiving Serostim. Some patients develop diabetic ketacidosis and diabetic coma. In some patients, therapy with Serostim necessitated initiation or adjustment of anti-diabetic treatment. Patients with a history of hyperglycemia or other risk factors for glucose intolerance should be monitored closely during treatment with Serostim. Transient increases in glucose levels occur early in treatment and should be monitored.

Use of growth hormone is contraindicated in treatment of patients in intensive care units due to complications following open-heart surgery or abdominal surgery, multiple accidental trauma or acute respiratory failure; patients with active neoplasia; and patients with known hypersensitivity to growth hormone. Serostim must be used in conjunction with antiretroviral therapy.

Posted: August 2003