What Is Forzinity, and Why Does It Matter?
Forzinity (elamipretide) is the first medicine to receive accelerated approval from the FDA for improving muscle strength in people with Barth syndrome who weigh at least 66 pounds (30 kilograms).
Barth syndrome is an extremely rare genetic disorder affecting mostly boys, with about 1 in 1 million male births, or roughly 150 people in the U.S. It often appears in infancy with severe heart problems, with most early deaths happening before age 5. Teens and adults with Barth syndrome have constant tiredness, muscle weakness, infections, and poor stamina for physical activities, which affects their daily lives significantly.
Barth syndrome is linked to problems in the mitochondria, which are the tiny powerhouses in your cells that produce energy. Forzinity works by directly targeting the faulty mitochondria, helping to improve their structure and function.
Why Was It Approved?
The FDA’s accelerated approval was based on signs that Forzinity may improve the strength of the leg muscle used to straighten the knee, which could help with everyday activities like standing up more easily or walking longer distances. Accelerated approval allows earlier clearance of medicines for serious conditions based on early signs showing that they might be helpful in providing relief, even if their full benefit hasn’t been confirmed at the time.
In studies, people who were given this medicine had better muscle strength, especially during the longer-term part of the study when everyone received the medicine.
The most common side effects reported in the study were mild-to-moderate reactions where the needle went in, such as redness, pain, bruising, or itching. Some people had serious allergic reactions such as a rash, bumps, and eczema-like irritation.
For continued approval, the FDA will require a follow-up study to confirm that the improvements in muscle strength lead to real-life benefits like better movement or less fatigue.
What Do I Need to Know?
Forzinity is given once a day as an injection under the skin, usually in the belly (not too close to the belly button) or outer thigh. It comes in a vial meant for one person only. After getting trained by your health care provider, you or your caregiver can give the shot at home. You should rotate the injection site daily and avoid injecting in places where the skin is tender, hard, red, bruised, scarred, or has stretch marks.
This medicine is not approved for newborns or children who weigh less than 66 pounds because of safety concerns related to a preservative in the formula. You should not take this medicine if you are allergic to any of its ingredients. Be sure to tell your health care provider about your health issues and all the medicines you take, including over-the-counter drugs, vitamins, and herbal supplements.
Reactions to the injection can often be managed with creams or allergy medicine. If you notice signs of a serious allergic reaction, such as a rash, swelling, or trouble breathing, stop using the medicine and get medical help right away. No data is available about how this medicine affects pregnant or breastfeeding women. Inform your health care provider if you are breastfeeding, pregnant, or planning to get pregnant.
Forzinity is expected to be available in the U.S. through specialty pharmacies by the end of the year. Your health care provider can help you decide if this treatment could be a good option for you or your child.