Known brand names include Forzinity.
A once-daily injection for people with Barth syndrome, a rare inherited disease of the mitochondria that weakens the heart and muscles. It binds a fat molecule inside mitochondria to help them work better, and it is approved to improve muscle strength in patients who weigh at least 30 kg.
Elamipretide travels into cells and settles on the inner membrane of the mitochondria, where it binds cardiolipin, a fat molecule that holds the energy-making machinery in shape. In Barth syndrome that fat molecule is faulty, so the mitochondria are misshapen and make energy poorly. By binding cardiolipin, the drug improves the structure and function of the mitochondria. Muscles that depend on that energy can then produce more force.
Daily injection (1/7)
Elamipretide is injected under the skin once a day, peaking in the blood within an hour.
After an injection under the skin, nearly all of the dose reaches the blood, and levels peak within about an hour. It spreads through the body's water, binds plasma proteins only loosely, and is broken down into two inactive fragments that leave in the urine within about 48 hours. Because the kidneys clear it, exposure rises as kidney function falls, which is why the dose drops with severe kidney impairment. In the trial, strength gains did not appear during the first 12 weeks but built up over the following months of open-label treatment.
Elamipretide is a once-daily injection for adults and children with Barth syndrome, a rare genetic disorder in boys and men that damages the mitochondria, the energy producers inside cells, and weakens the heart, skeletal muscles, and immune system. It is approved to improve muscle strength in patients who weigh at least 30 kg. The approval is an accelerated one, based on stronger knee muscles rather than on a proven change in how people feel or function. Continued approval depends on a confirmatory trial.
Patients or caregivers inject it under the skin of the stomach area or outer thigh after training, moving the site each day. The dose is lowered in adults whose kidneys work poorly, because the drug leaves the body through the urine. It is sold as Forzinity, and the whole development program involved 12 male patients aged 12 to 35. It has not been studied in patients under 30 kg, and it is not for newborns, because its preservative, benzyl alcohol, can be toxic to them.
Redness, hardness, itching, pain, bruising, or hives where the shot goes in are what nearly everyone notices, and in the trial every treated patient had some injection-site reaction. A rise in eosinophils, a type of white blood cell, is common in blood tests and settles over months without symptoms. The label treats allergic reactions as serious, including rash, eczema-like skin changes, and cough, which can appear minutes to months after starting. It also warns that the benzyl alcohol preservative can cause a fatal gasping syndrome in newborns, who must never receive it.