Congenital generalized lipodystrophy — a disorder that significantly reduces all fat tissue and affects a few thousand people worldwide — is typically treated with daily injections of metreleptin.
Although metreleptin is administered subcutaneously, the lack of subcutaneous fat in these patients makes “each injection painful,” according to Svetlana Ten, MD, the study’s first author, a pediatrics professor at Rutgers University and a physician at West Orange, N.J.-based RWJBarnabas Health.
Daily metreleptin injections also cost thousands of dollars annually, Rutgers University noted in a news release.
Dr. Ten and other researchers examined whether tirzepatide, a Type 2 diabetes drug that improves insulin resistance and costs less than metreleptin, could provide a better treatment option for congenital generalized lipodystrophy patients. In the study, a 23-year-old man’s blood glucose levels nearly normalized, while a 64-year-old woman achieved normal blood glucose levels.
A larger trial is needed to validate these results and confirm the long-term efficacy and safety of tirzepatide for this use, according to the release. However, recruiting enough patients may be challenging given the disorder’s rarity.
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