WHA

Forzinity (elamipretide)

Indications for Prior Authorization

Forzinity (elamipretide)
  • For diagnosis of Barth Syndrome
    Indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg.

Criteria

Forzinity

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

  • Diagnosis of Barth syndrome
    • AND
  • Disease is confirmed by the presence of a mutation in the Tafazzin gene, as detected by an FDA-approved test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA) [A, 5]
    • AND
  • Patient weight is greater than or equal to 30kg
    • AND
  • Prescribed by or in consultation with one of the following: [B, 5]
    • geneticist
    • cardiologist
    • neurologist
    • specialist focused on the treatment of metabolic disorders
Forzinity

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

  • Patient demonstrates positive clinical response to therapy (e.g., improvement in knee extensor muscle strength or 6 minute walking test, quality of life)

  • Guideline ID
    GL-448288

  • Formulary
    premium

  • Effective date
    Jan 1, 2026

  • P&T approval date
    Dec 17, 2025

P & T Revisions

1970-01-01

  1. Forzinity Prescribing Information. Stealth BioTherapeutics Inc. Needham, MA. September 2025.
  2. Barth Syndrome. Available at https://rarediseases.org/rare-diseases/barth-syndrome/. Accessed November 11, 2025.
  3. ClinicalTrials.gov. A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome (TAZPOWER). Available at: https://www.clinicaltrials.gov/study/NCT03098797. Accessed November 11, 2025.
  4. Stealth BioTherapeutics Announces FDA Accelerated Approval of FORZINITY™ (elamipretide HCl), the First Therapy for Progressive and Life-limiting Ultra-rare Genetic Disease Barth Syndrome. Available at: https://www.prnewswire.com/news-releases/stealth-biotherapeutics-announces-fda-accelerated-approval-of-forzinity-elamipretide-hcl-the-first-therapy-for-progressive-and-life-limiting-ultra-rare-genetic-disease-barth-syndrome-302562058.html. Accessed November 11, 2025.
  5. Clinical Consult with pediatric cardiologist 11/24/2025.
  1. An elevated MLCL:CL (monolysocardiolipin to cardiolipin) ratio not appropriate as it's sent internationally [Amsterdam] and can take about 1 month to get test results, significantly delaying patient treatment. [5]
  2. The specialist prescriber requirement of geneticist, cardiologist, neurologist, and specialist focused on the treatment of metabolic disorders appropriate. A hematologist would most likely only play a minor role in blood test for low neutrophil count and risk of infection. [5]