SS-31 (Elamipretide)
Forzinity · Bendavia · MTP-131 · Szeto-Schiller Peptide
SS-31 — now marketed as Forzinity (elamipretide) — became the first FDA-approved mitochondria-targeted therapeutic on September 19, 2025, when Stealth BioTherapeutics received accelerated approval for an ultra-rare indication: improving muscle strength in adult and pediatric patients with Barth syndrome weighing ≥30 kg. The molecule is a four–amino-acid aromatic-cationic tetrapeptide that concentrates ~1,000-fold in the inner mitochondrial membrane by reversibly binding cardiolipin, stabilizing cristae architecture and restoring ATP synthesis. Despite the landmark Barth approval, Phase 3 trials in two of its three other target indications (primary mitochondrial myopathy and HFrEF) were negative. This page documents both sides of that evidence base honestly.
What is it?
Elamipretide is the clinical name for SS-31, originally developed at Weill Cornell by Hazel Szeto and Peter Schiller in the early 2000s as the lead compound of the 'Szeto-Schiller peptide' series. Its defining feature is selective mitochondrial targeting without a mitochondrial signal sequence: the alternating cationic (D-Arg, Lys) and aromatic (dimethyltyrosine, Phe) residues allow the peptide to cross the plasma membrane and concentrate approximately 1,000-fold at the inner mitochondrial membrane by binding cardiolipin, a unique phospholipid found almost exclusively there. Unlike classical antioxidants, SS-31 does not scavenge free radicals directly — it protects cardiolipin from peroxidation and stabilizes the electron transport chain complexes that cardiolipin organizes into supercomplexes.
After a decade of preclinical work and multiple early-phase clinical trials across heart failure, acute kidney injury, mitochondrial myopathy, and age-related macular degeneration, Stealth BioTherapeutics focused the regulatory effort on Barth syndrome — an ultra-rare X-linked disease (~150 US patients) caused by TAFAZZIN mutations that disrupt cardiolipin remodeling. The FDA granted accelerated approval on September 19, 2025, making Forzinity the first approved treatment for Barth syndrome and the first FDA-approved mitochondria-targeted therapeutic of any class. Continued approval is contingent on confirmatory trials. Phase 3 programs are ongoing in dry AMD (ReNEW/ReGAIN) after ReCLAIM-2 missed its primary endpoints but showed signal on ellipsoid-zone preservation.
Mechanism of Action
SS-31 is a designed aromatic-cationic tetrapeptide that selectively concentrates at the inner mitochondrial membrane without a targeting sequence, binds cardiolipin reversibly, and restores mitochondrial bioenergetics through a structural rather than antioxidant mechanism. The five pathways below represent the current mechanistic consensus.
The binding and protection steps are laboratory and rat work. The human benefit rests on one rare disease; in two other diseases trials found none.
Each step is tagged with where it was shown. A dashed red arrow marks a link the evidence does not establish.
The pathway in words
- Elamipretide. A mitochondria-targeted tetrapeptide; a fluorescent analogue showed it binds cardiolipin, a lipid of the inner mitochondrial membrane. Shown in: lab. Source: PubMed 23813215.
- Protecting mitochondria. Bound to cardiolipin it blocked cytochrome c peroxidase; in rats it protected cristae during kidney ischaemia and sped ATP recovery. Shown in: animals. Source: PubMed 23813215.
- Barth syndrome, randomised. Barth syndrome makes abnormal cardiolipin. In a 12-person crossover trial, neither primary endpoint was met at 12 weeks. Shown in: people. Source: PubMed 33077895.
- Barth syndrome, open-label. Of ten who continued, eight reached week 168; walking distance rose a cumulative 96.1 m, with less fatigue. Shown in: people. Source: PubMed 38602181.
- Approved. Accelerated approval in September 2025 to improve muscle strength in Barth syndrome, for patients of at least 30 kg. Shown in: review. Source: PubMed 41335372.
- tested, no benefit shown. Beyond Barth syndrome. In 218 people with mitochondrial myopathy, walking did not improve; in 71 with heart failure, heart volumes did not change. Shown in: people. Source: PubMed 37268435, 32068002.
Cardiolipin Binding (Primary Mechanism)
Cardiolipin is a unique dimeric phospholipid found almost exclusively on the inner mitochondrial membrane, where its four acyl chains organize electron transport chain complexes (I, III, IV) into respiratory supercomplexes for efficient electron flow. Birk et al. (2013, J Am Soc Nephrol) demonstrated using polarity-sensitive fluorescent analogs that SS-31 binds cardiolipin with high affinity. The SS-31/cardiolipin complex also inhibits the peroxidase activity of cytochrome c, which otherwise catalyzes cardiolipin peroxidation during ischemia. By physically protecting cardiolipin from damage and stabilizing its interaction with cytochrome c, SS-31 preserves the structural integrity the entire respiratory chain depends on.
Cristae Architecture & Supercomplex Assembly
Cardiolipin is required for the tight curvature of mitochondrial cristae, the folded inner-membrane structures that house electron transport. When cardiolipin is damaged (by peroxidation in ischemia) or abnormally remodeled (as in Barth syndrome, where TAFAZZIN mutations produce monolysocardiolipin), cristae flatten, supercomplexes disassemble, and oxidative phosphorylation becomes inefficient. In failing human heart tissue ex vivo, elamipretide treatment restored supercomplex-associated Complex IV activity and improved respiratory control (Chatfield 2019, JACC Basic Transl Sci). The mechanism is structural: stabilizing cardiolipin preserves the membrane geometry that respiratory assembly requires.
Restoration of ATP Synthesis
The downstream consequence of preserved supercomplex function is restored ATP production. In ischemia-reperfusion models, SS-31 accelerates ATP recovery after blood flow is restored. In Barth syndrome, where monolysocardiolipin accumulates and normal cardiolipin is depleted, the cristae phenotype produces measurable exercise intolerance. Forzinity treatment in TAZPOWER improved the 6-minute walk test and patient-reported muscle strength measures — the clinical signal that carried the NDA. The FDA approved the drug on the functional endpoint (muscle strength) rather than on a cardiolipin biomarker.
Reduction of Mitochondrial ROS (Indirect)
SS-31 is not a free-radical scavenger in the conventional sense. It reduces mitochondrial reactive oxygen species (ROS) indirectly, by stabilizing cardiolipin and preventing the cardiolipin-peroxidation/cytochrome-c-detachment cycle that generates electron leak. This distinction matters clinically: decades of ROS-scavenger antioxidant trials (Vitamin E, CoQ10 at most doses) have failed to show meaningful clinical benefit, likely because bulk ROS quenching does not address the structural defects driving pathological ROS generation. SS-31's mechanism targets the source, not the scavenging.
Selective Mitochondrial Concentration
Unlike most mitochondria-targeted compounds (e.g., MitoQ, SkQ1) that require a triphenylphosphonium (TPP+) cation tether to cross the inner membrane, SS-31 concentrates at the inner mitochondrial membrane through its intrinsic structure — alternating cationic and aromatic residues create a molecule that partitions into membranes and is retained at the cardiolipin-rich inner leaflet. This yields a ~1,000-fold concentration gradient versus cytosol with no TPP+ tether, reducing off-target effects and eliminating the mitochondrial membrane depolarization that high-dose TPP+ compounds can cause. The selectivity is a major reason SS-31 advanced to a first-in-class approval while TPP+-based compounds have not.
Published Research
The elamipretide evidence base spans two decades of preclinical work and a dozen clinical trials. The drug succeeded in ultra-rare Barth syndrome (basis for the September 2025 approval) and failed in two larger Phase 3 trials (primary mitochondrial myopathy and HFrEF). Dry AMD showed mixed Phase 2 results with confirmatory Phase 3 trials ongoing. All studies are cited honestly below — including the negative trials, because the full shape of this evidence base matters for understanding where the drug works and where it does not.
Elamipretide: First Approval (canonical post-approval review)
Shirley M, Drugs 2025, PMID 41335372. Systematic review of elamipretide's development program summarizing the regulatory milestones that led to the September 2025 accelerated approval for Barth syndrome. Details the Phase 2/3 TAZPOWER and SPIBA-001 natural-history-control pivotal trials that supported the NDA, the Phase 3 ReNEW/ReGAIN program in dry AMD, and the negative MMPOWER-3 and PROGRESS-HF readouts. This is the field-defining review citation for the molecule post-approval.
TAZPOWER 168-Week Open-Label Extension (Long-Term)
Thompson WR, et al., Genet Med 2024;26(7):101138, PMID 38602181. Long-term extension of TAZPOWER following the 28-week RCT crossover. 10 Barth syndrome patients entered the OLE at 40 mg SC daily; 8 completed the 168-week visit. Elamipretide was well tolerated (injection-site reactions the most common AE) with sustained improvements in 6-minute walk test, muscle strength, and the BTHS-SA Total Fatigue score. Also documented improvements in echocardiographic parameters and reductions in the cardiolipin/monolysocardiolipin biomarker ratio. This long-term safety and durability evidence was critical for the accelerated approval.
ReCLAIM-2 Phase 2 in Dry AMD with Geographic Atrophy
Ehlers JP, et al., Ophthalmol Sci 2024;5(1):100628, PMID 39605874. 48-week randomized placebo-controlled trial of 40 mg SC daily in patients ≥55 with dry AMD and noncentral geographic atrophy. The primary endpoints (low-luminance BCVA and GA area) were NOT met, but a predefined secondary endpoint showed elamipretide slowed ellipsoid zone (EZ) degradation — a surrogate for photoreceptor integrity that precedes visual loss. The EZ signal became the primary endpoint of the ongoing Phase 3 ReNEW and ReGAIN trials. Injection-site reactions led to 17% treatment discontinuation (vs ~9% placebo) — a meaningful tolerability finding for chronic daily dosing in elderly patients.
MMPOWER-3 Phase 3 in Primary Mitochondrial Myopathy (NEGATIVE)
Karaa A, et al., Neurology 2023;101(3):e238-252, PMID 37268435. Pivotal Phase 3 in genetically confirmed primary mitochondrial myopathy (PMM). 218 adults randomized 1:1 to elamipretide 40 mg SC daily or placebo for 24 weeks. Class I evidence that elamipretide does NOT improve the 6-minute walk test or PMM symptom fatigue score at 24 weeks versus placebo. Safety was acceptable. This was the largest elamipretide Phase 3 trial and its failure ended the PMM development program. A post-hoc genotype-specific analysis (Karaa 2024, PMID 39574155) identified a subgroup with mtDNA maintenance defects that may have responded, but this is hypothesis-generating only.
SPIBA-001 Natural History Control Study (Pivotal)
Hornby B, et al., Orphanet J Rare Dis 2022;17(1):336, PMID 36056411. Retrospective propensity-score-matched comparison of 8 TAZPOWER OLE patients against 19 untreated Barth syndrome natural-history controls (12 with serial echo). At week 64, least-squares mean difference in 6-minute walk distance was 79.7 m favoring elamipretide (P=0.0004); at week 76, 91.0 m (P=0.0005). This natural-history comparison — designed under FDA guidance for ultra-rare diseases where conventional randomized control arms are impractical — provided the external efficacy evidence that, combined with TAZPOWER and its OLE, supported the 2025 accelerated approval.
TAZPOWER Phase 2/3 Pivotal RCT in Barth Syndrome
Thompson WR, et al., Genet Med 2021;23(3):471-478, PMID 33077895. Randomized, double-blind, placebo-controlled crossover trial in 12 Barth syndrome patients. Part 1: 12 weeks elamipretide 40 mg SC daily vs placebo, 4-week washout, then crossover. The crossover portion did not meet its primary endpoint, but patient-reported outcomes and the subsequent open-label extension showed clinically meaningful improvements in 6-minute walk test, muscle strength, and BTHS-SA fatigue scores. The pivotal crossover RCT, its OLE (PMID 38602181), and the natural-history comparison (PMID 36056411) together comprised the NDA efficacy package. Notably, the initial NDA (August 2021) was refused-to-file; it took Stealth until January 2024 to resubmit, a May 2025 CRL, and a July 2025 resubmission before the September 19, 2025 accelerated approval.
PROGRESS-HF Phase 2 in HFrEF (NEGATIVE)
Butler J, et al., J Card Fail 2020;26(5):429-437, PMID 32068002. Phase 2 randomized placebo-controlled trial in 71 patients with stable HFrEF (LVEF ≤40%). Three-arm: placebo, elamipretide 4 mg, elamipretide 40 mg SC daily for 28 days. Primary endpoint (change in LV end-systolic volume by cardiac MRI) was NOT different between arms: 4 mg vs placebo P=0.90; 40 mg vs placebo P=0.28. LVEF and safety also unchanged. This negative result effectively ended the general HFrEF development program for elamipretide, though mechanistic work in failing human heart tissue ex vivo continued to show mitochondrial functional improvement (Chatfield 2019, JACC Basic Transl Sci). The disconnect between ex-vivo mitochondrial improvement and in-vivo ventricular remodeling remains unexplained.
Foundational Mechanism — Cardiolipin Binding Identified
The landmark mechanism paper. Using a polarity-sensitive fluorescent analog of SS-31, the authors demonstrated that SS-31 binds cardiolipin with high affinity on the inner mitochondrial membrane, and that the SS-31/cardiolipin complex inhibits cytochrome-c peroxidase activity — the enzyme that catalyzes cardiolipin peroxidation during ischemic injury. This paper established the structural (rather than antioxidant) mechanism that has defined the molecule ever since and separated it conceptually from TPP+-tethered mitochondrial antioxidants. Every subsequent clinical development program — Barth, AMD, heart failure, PMM — traces its rationale to this mechanistic insight.
What people say, and what was measured
Elamipretide, sold in the grey market as SS-31, is the rare case of a mitochondrial peptide that got through to approval — for one rare disease, after failing its endpoints in several others. Both halves of that sentence matter.
It is approved, so the mitochondrial benefits are established.
Heard in: Grey Peptides listing review, October 1, 2026: listings cite approval as proof the peptide repairs mitochondria generally. Sellers are not named.
Approval came for Barth syndrome, a rare genetic disorder, on a trial where the drug improved a six-minute walk distance by 95.9 m (p = 0.024) and a symptom score in its second part, with benefit maintained to 168 weeks. That is one rare disease, not a general mitochondrial claim. PMID: 41335372, 33077895, 38602181
Tested in people; supportedIt restores energy in anyone with mitochondrial dysfunction or fatigue.
Heard in: Grey Peptides listing review, October 1, 2026: listings sell it for energy, ageing and fatigue. Sellers are not named.
Tested, and negative. The MMPOWER-3 trial in primary mitochondrial myopathy provides Class I evidence that elamipretide did not improve the six-minute walk or fatigue at 24 weeks against placebo. A natural-history comparison was run in Barth syndrome separately. PMID: 37268435, 36056411
Tested in people; not shownIt helps the failing heart.
Heard in: Grey Peptides listing review, October 1, 2026: listings include cardiac benefit among its uses. Sellers are not named.
A trial measured exactly this: effects on left ventricular function in heart failure with reduced ejection fraction. The mechanism, re-energising ischaemic mitochondria by interacting with cardiolipin, was established in laboratory work. PMID: 32068002, 23813215
Tested in people; not shownIt treats eye disease linked to mitochondrial decline.
Heard in: Grey Peptides listing review, October 1, 2026: listings mention macular degeneration. Sellers are not named.
ReCLAIM-2, a randomised phase 2 trial in age-related macular degeneration, is the study behind that claim and is the appropriate place to read its result rather than a product page. PMID: 39605874
Tested in people; mixedEach claim is quoted from where it is said, never from a seller, and checked as of October 1, 2026 against the studies listed below.
Dosing Data
Elamipretide is dosed uniformly at 40 mg subcutaneously once daily across virtually every human trial conducted — including the approved Barth regimen and all failed/investigational indications. This is unusual for a drug with an active Phase 3 program; most molecules are still titrating dose by the time they reach pivotal trials. The 40 mg dose emerged from the MMPOWER Phase 1/2 dose-escalation (Karaa 2018, PMID 29500292) and has been consistent since.
Educational information, not medical advice. Each dose above is tagged with its source: an approved label, a published human study, animal data, or amounts reported by users. None is a recommendation for you. An unapproved compound has no established safe or effective human dose, and products sold for “research use only” are not made or tested for people. Talk to a doctor before acting on anything on this site, including before you start, stop or change any medicine or dose. Full medical disclaimer.
→ Blend & Stack Calculator — SS-31 (Elamipretide) is also sold in multi-peptide vials; the calculator shows what each draw delivers of every component; our blends and stacks hub sets out what each mix contains and what has been studied.
Side Effects & Contraindications
Reported Side Effects
Based on the TAZPOWER 168-week OLE (Thompson 2024), MMPOWER-3 (Karaa 2023), ReCLAIM-2 (Ehlers 2024), and the Forzinity prescribing information. The safety profile is dominated by injection-site reactions. Systemic effects are uncommon. Importantly, the 24.8% discontinuation rate in ReCLAIM-2 elderly AMD patients (vs 15.2% placebo) was driven primarily by ISRs and is a meaningful tolerability finding for chronic daily subcutaneous dosing:
Contraindications & Cautions
Forzinity's prescribing information lists specific contraindications and warnings. Additional caution classes are inferred from the mechanism and trial exclusions. This list is not exhaustive — patients on Forzinity should be under the care of a metabolic/genetic specialist who can manage the drug in the context of the patient's full clinical picture:
Legal Status
The legal status of SS-31 / elamipretide is jurisdiction-specific and highly asymmetric as of April 2026: FDA-approved as Forzinity in the United States for Barth syndrome only; investigational or unapproved everywhere else. Compounding is not lawful in the US given exclusivity protection and the absence of Drug Shortage list status. 'Research-chemical' SS-31 continues to be sold by peptide vendors — but this is unregulated material that has not been through cGMP manufacturing and is not bioequivalent to Forzinity.
Frequently Asked Questions
?Is SS-31 the same as Forzinity (elamipretide)?
Yes — SS-31 is the original research designation (from the Szeto-Schiller peptide series developed at Weill Cornell). Elamipretide is the generic name, and Forzinity is the brand name for the FDA-approved formulation marketed by Stealth BioTherapeutics since September 2025. MTP-131 and Bendavia are older development code names that appear in earlier publications. All four terms refer to the identical molecule (H-D-Arg-Dmt-Lys-Phe-NH2).
?What exactly is Forzinity approved for?
Forzinity is approved under FDA accelerated approval (September 19, 2025) for ONE indication: improvement of muscle strength in adult and pediatric patients with Barth syndrome who weigh at least 30 kg. Barth syndrome is an ultra-rare X-linked genetic disease caused by TAFAZZIN mutations — approximately 150 patients in the US. The approval is NOT for heart failure, primary mitochondrial myopathy, macular degeneration, aging, or any other mitochondrial condition. Continued approval is contingent on confirmatory trials.
?Does SS-31 work for anti-aging or general longevity?
There is no clinical evidence supporting SS-31 for anti-aging or general longevity use. The molecule has mechanism-of-action appeal in the aging field (mitochondrial dysfunction is a hallmark of aging, and cardiolipin peroxidation increases with age), and there is preclinical work in aged rodents showing restored muscle function. But the only randomized clinical trial conducted in an age-related disease population — ReCLAIM-2 in dry AMD — did NOT meet its primary endpoints, though it did show a secondary signal on ellipsoid-zone preservation that Phase 3 trials (ReNEW/ReGAIN) are now evaluating. Two other Phase 3 trials (MMPOWER-3 in primary mitochondrial myopathy, PROGRESS-HF in heart failure) failed outright. Until Phase 3 data from ReNEW/ReGAIN reads out, the honest answer is: SS-31 works for Barth syndrome, and we don't know if it works for anything else.
?Why did MMPOWER-3 and PROGRESS-HF fail?
Both were negative Phase 3 trials: MMPOWER-3 (primary mitochondrial myopathy, 218 patients, 24 weeks) showed Class I evidence that elamipretide did NOT improve the 6-minute walk test or fatigue score vs placebo; PROGRESS-HF (HFrEF, 71 patients, 28 days) showed no change in LV end-systolic volume by cardiac MRI. The mechanistic biology held up — the drug does bind cardiolipin, supercomplex function does improve ex-vivo in failing human hearts (Chatfield 2019) — but these cellular effects did not translate into clinical outcomes in these populations. Why Barth worked and PMM/HFrEF didn't remains unclear. One hypothesis: in Barth, cardiolipin itself is defective (the primary molecular lesion) so stabilizing it addresses root cause; in PMM and HFrEF, cardiolipin dysfunction is downstream of other problems and stabilizing it is a partial intervention at best.
?How is Forzinity dosed?
40 mg subcutaneous injection, once daily, for adult and pediatric Barth syndrome patients weighing ≥30 kg. This is a fixed dose — there is no titration schedule. Injection rotation among standard SC sites is recommended to minimize injection-site reactions, which are the most common adverse event. Treatment is chronic; improvements in 6-minute walk test and muscle strength in TAZPOWER were sustained across 168 weeks of continuous dosing. Forzinity is dispensed through specialty pharmacy by Stealth BioTherapeutics.
?Can I buy SS-31 from a peptide research vendor?
Peptide research vendors do sell 'research-use-only' SS-31, but this is unregulated material that has NOT been through FDA cGMP manufacturing. It is not bioequivalent to Forzinity (the approved drug), purity is not guaranteed, and there is no quality assurance on the peptide sequence or formulation. Using research-chemical SS-31 to self-treat Barth syndrome, heart failure, macular degeneration, or primary mitochondrial myopathy is strongly discouraged — particularly for Barth syndrome, where an FDA-approved formulation is available through prescription and specialty pharmacy. For non-Barth indications where no approval exists, the underlying evidence does not yet support self-treatment with any form.
?Is SS-31 eligible for compounding pharmacy dispensing?
No. Compounded SS-31 is not legally available in the United States. Forzinity has orphan drug exclusivity, is not on the FDA Drug Shortage list, and has no reference listed drug that would enable a compounding pathway. This differs materially from the compounded-semaglutide situation of 2022–2024, which relied on an active shortage designation. Any US compounding pharmacy offering 'compounded SS-31' is either operating outside lawful parameters or is not manufacturing the approved elamipretide formulation. For Barth syndrome patients, the approved Forzinity should be accessed through the prescribing specialist and Stealth's specialty pharmacy network.
?How much does Forzinity cost?
Stealth BioTherapeutics has not publicly disclosed Forzinity pricing as of early 2026, but ultra-rare orphan drugs for indications of ~150 US patients typically price at $200,000–$750,000+ per patient per year. Barth Syndrome Foundation and Stealth have communicated intent to use standard specialty-pharmacy and patient-assistance programs to minimize out-of-pocket cost for diagnosed Barth patients, with payer reimbursement (commercial insurance, Medicaid, Medicare for eligible adults) expected to cover the wholesale acquisition cost. Patients with a confirmed TAFAZZIN-based Barth diagnosis should work with their prescribing specialist and Stealth's patient support team rather than trying to navigate access independently.
?Does SS-31 cross the blood-brain barrier?
Preclinical evidence suggests limited but detectable CNS penetration. The molecule's small size (639 Da), aromatic-cationic character, and membrane-partitioning behavior allow some passage, and neuroprotective effects have been shown in preclinical spinal cord injury and traumatic brain injury models. However, no elamipretide clinical trial has been conducted in a primary CNS indication (no Parkinson's, Alzheimer's, ALS, or stroke trials). The drug's development focus has been peripheral (cardiac, skeletal muscle, retina, kidney) and the approved Barth indication works through skeletal muscle. Extrapolating to CNS use requires human CNS-penetration data that doesn't exist.
?What is the future development pipeline for elamipretide?
As of April 2026: (1) Phase 3 ReNEW and ReGAIN trials in dry AMD with geographic atrophy are ongoing, using the ellipsoid-zone preservation endpoint that emerged from ReCLAIM-2's secondary analysis — these are the most consequential upcoming readouts because a positive result would expand elamipretide from an ultra-rare orphan drug to a potentially large commercial indication; (2) confirmatory trials in Barth syndrome are required for continued approval under the accelerated pathway; (3) compassionate-use and expanded-access protocols continue for individual pediatric mitochondrial cases (Maeder 2023, PMID 36636586). The heart failure and primary mitochondrial myopathy programs are effectively closed following the negative Phase 3 readouts, absent new subgroup or biomarker-driven rationale.
References
- [fda-pi] Forzinity (elamipretide) Prescribing Information. Stealth BioTherapeutics. September 2025.
- [clinical-trial] Thompson WR, et al. "A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome (TAZPOWER)." Genet Med, 2021;23:471-478. PMID: 33077895.
- [pubmed] Birk AV, et al. "The mitochondrial-targeted compound SS-31 re-energizes ischemic mitochondria by interacting with cardiolipin." J Am Soc Nephrol, 2013;24:1250-1261. PMID: 23813215.
- [clinical-trial] Butler J, et al. "Effects of elamipretide on left ventricular function in heart failure with reduced ejection fraction: the PROGRESS-HF phase 2 trial." J Card Fail, 2020. PMID: 32068002.
- [pubmed] Shirley M. "Elamipretide: First Approval." Drugs, 2026;86(3):377-383. PMID: 41335372.
- [pubmed] Thompson WR, et al. "Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER." Genet Med, 2024;26(7):101138. PMID: 38602181.
- [pubmed] Ehlers JP, et al. "ReCLAIM-2: A Randomized Phase II Clinical Trial Evaluating Elamipretide in Age-related Macular Degeneration, Geographic Atrophy Growth, Visual Function, and Ellipsoid Zone Preservation." Ophthalmol Sci, 2025;5(1):100628. PMID: 39605874.
- [pubmed] Karaa A, et al. "Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial." Neurology, 2023;101(3):e238-e252. PMID: 37268435.
- [pubmed] Hornby B, et al. "Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome." Orphanet J Rare Dis, 2022;17(1):336. PMID: 36056411.
Sources & Citations
Molecular data from Forzinity FDA prescribing information (NDA 215244, approved September 19, 2025) and PubChem. Clinical trial data from peer-reviewed publications in Genetics in Medicine, Neurology, Ophthalmology Science, Journal of Cardiac Failure, J Am Soc Nephrol, and Orphanet Journal of Rare Diseases. Regulatory status from FDA approval documents, Stealth BioTherapeutics press releases, and the Barth Syndrome Foundation. Development history and post-approval mechanistic context drawn from Shirley 2025 Drugs 'First Approval' review (PMID 41335372) and Birk 2013 foundational mechanism paper (PMID 23813215). Forzinity pricing and access information reflect publicly available data as of early 2026 and should be verified with the prescribing specialist and Stealth patient support. All eight cited PMIDs were verified against PubMed during authoring.
Medical Disclaimer: This content is for educational and informational purposes only. It is not medical advice, and nothing here is an instruction to obtain or use any compound. Talk to a licensed clinician about your own situation.