Brand Name: FORZINITY™
Chemical Name: Elamipretide
Generic Available: No

Forzinity is a therapy administered via subcutaneous injection, typically given once daily.  It is a mitochondria-targeting peptide designed to improve the function of mitochondria – the energy-producing structures within cells.  

Forzinity received accelerated approval for treating Barth syndrome, an ultra-rare form of mitochondrial disease caused by mutations in the TAZ gene associated with heart problems, muscle weakness, delayed growth, chronic fatigue, and low levels of white blood cells that fight off infection. It is thought to work by selectively targeting and stabilizing a key mitochondrial lipid called cardiolipin, which plays a crucial role in maintaining mitochondrial structure and function. 

The U.S. Food and Drug Administration (FDA) has granted accelerated approval to  Forzinity to improve muscle strength in adult and pediatric patients with genetically confirmed Barth syndrome weighing at least 30 kilograms (approximately 66 pounds).

UMDF is proud to partner with Barth Syndrome Foundation to support patients diagnosed with Barth syndrome. If you have other forms of mitochondrial disease — or are still on your diagnostic journey and suspect mito, we encourage you to reach out to UMDF’s support team at (888) 900-6486 or support@umdf.org. 

What kind of diagnostic test is required before starting ?

A prescription for Forzinity will typically require a confirmed diagnosis of Barth syndrome. This is typically done through genetic testing to identify mutations in the TAZ gene, which is responsible for the condition. Additional evaluations may include clinical assessment, metabolic testing, and cardiac imaging to understand the full impact of the disease. 

There is no single required diagnostic tool beyond confirming the genetic mutation, but your doctor may also order blood tests, echocardiograms, or other assessments to guide treatment and monitor progress. 

How do I receive this treatment?  

Forzinity is administered through a subcutaneous injection once per day (vial and syringe). The injection is given just under the skin and can be done at home by the patient, a caregiver, or a trained professional. Your healthcare provider will train you on proper injection technique and discuss the best time of day to administer the medication. 

What are the side effects?

In clinical trials, Forzinity was generally well tolerated. The most commonly reported side effect was injection site reactions such as redness, pain, or swelling. This is typically mild to moderate and tends to improve over time. Contact your healthcare provider if any side effects persist or become bothersome. 

Can I take Forzinity with my other medications or supplements?

Before starting Forzinity, talk to your healthcare provider about all medications, vitamins, and supplements you take. While no major drug interactions have been reported, your doctor will consider your full medical history to ensure Forzinity is safe and effective for you. 

 How is Forzinity different from other treatments for Barth syndrome?

Forzinity is the first FDA-approved therapy specifically for Barth syndrome. It works by targeting mitochondria and stabilizing a lipid called cardiolipin that’s essential for energy production. Unlike supportive treatments like cardiac medications or nutritional supplements, Forzinity directly addresses the underlying mitochondrial dysfunction in Barth syndrome. 

 How much does Forzinity cost?

The list price of Forzinity is set by the manufacturer and may vary depending on insurance coverage, treatment duration, and patient assistance programs.  If you have concerns around coverage and pricing, we suggest you reach out to Stealth’s Mito Assist program, which can be found two questions below. 

 Will insurance cover Forzinity?

Since Forzinity is FDA-approved for Barth syndrome, many private insurance plans and Medicaid/Medicare programs are expected to cover it for those with a confirmed diagnosis of Barth syndrome who meet the prescribing criteria. Coverage will vary depending on your plan. Ask your healthcare provider or specialty pharmacy team to help you navigate prior authorizations and cost support. 

If you receive a denial for Forzinity when you believe you meet all the necessary requirements, please know you don’t have to face the appeals process alone. UMDF is here to support you with tools, resources, and guidance to help navigate insurance appeals. Our goal is to ensure that patients and families have the best chance of accessing the treatments they need. Reach out to UMDF Support for assistance at (888) 900-6486 or support@umdf.org. We are committed to standing alongside you every step of the way. 

 Is there financial assistance available?

Stealth has announced a support program called “Mito Assist,” which will offer personalized patient support services to help affected individuals and caregivers understand coverage options, manage insurance submissions, and access financial assistance resources. This includes:

• Co-pay assistance for eligible commercially insured patients.
• Patient assistance programs for uninsured or underinsured individuals.
• Support for at-home subcutaneous injection training.
• Dedicated case management through AnovoRx to provide ongoing communication with patients, caregivers, and healthcare providers.

You can start the process by completing the Patient Enrollment Form on the AnovoRx site: Forzinity Enrollment Form

 When will it be available at my doctor’s office or pharmacy?

Stealth has selected AnovoRx Specialty Pharmacy as its exclusive commercial distribution partner for Forzinity in the United States. As of December 4, 2025, the product is now officially commercially available. 

 What should I talk to my doctor about before starting treatment? 

Discuss your medical history, current medications, and goals for treatment. Be sure to mention any past side effects to injectable medications or any concerns about administering the drug. Your care team will work with you to develop a personalized treatment plan and address questions about monitoring, logistics, and long-term management. 

 What if a patient has Barth syndrome, but doesn’t meet the weight requirements (30kg)? 

While the manufacturer is working with the FDA to provide data needed to expand the label to include all ages, they have agreed to provide a critical stop gap in care and continue providing expanded access to children weighing less than 30 kg who are currently receiving treatment or require emergency access.  

Are patients with any other forms of mitochondrial disease eligible for the drug?

At this time, Forzinity is FDA-approved only for the treatment of Barth syndrome. This means insurance coverage and access are currently limited to adult and pediatric patients with a confirmed diagnosis of Barth syndrome weighing at least 30 kilograms (kg). Forzinity has been studied in other mitochondrial diseases including primary mitochondrial myopathy, with clinical research ongoing. You can see the latest news on the NuPower study on our UMDF Therapeutic Pipeline.  

 Can a doctor write me a prescription for Forzinity even if I do not have Barth syndrome?

Doctors are able to write a prescription for indications other than those approved by the FDA if they feel it is medically warranted (off-label use).  While off-label use of medication is allowed in the USA, insurance coverage could be problematic. While we don’t yet have any details, we anticipate this will be an expensive medication that insurance – both private and public – will only approve for Barth syndrome patients who are at least 30kg.  If you have any questions about the potential role of Forzinity  in your medical care, please discuss this with your healthcare professional. You may also reach out to a clinician at any Mitochondrial Care Center, who have specific mitochondrial disease expertise. 

 I was in the TAZPOWER clinical trial / Expanded Access Program and meet the new prescription criteria. What are the next steps for me to remain on the medicine?

If you’re a patient currently receiving elamipretide through Stealth’s Expanded Access Program who meets the weight requirement of 30kg, you shouldl be contacted directly by your healthcare provider and AnovoRx case managers to facilitate a seamless transition to commercial therapy once Forzinity becomes available.