Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER

Thompson WR, Manuel R, Abbruscato A, et al. · Genetics in Medicine

The longest human exposure to daily subcutaneous elamipretide: 186 adverse events in 10 patients, dominated by injection-site erythema and pruritus (80% each) and pain (70%); two discontinued for urticaria or drug eruption; no treatment-related serious events and no deaths. Six-minute-walk distance was 96.1 m above pre-drug baseline at week 168 (p = 0.003), uncontrolled.

Population

10 Barth syndrome patients continuing from TAZPOWER into the open-label extension

Intervention

Elamipretide 40 mg subcutaneous daily for up to 168 weeks

Comparator

None (comparison to pre-drug baseline)

Limitations

Open-label, no control arm, 10 patients; Stealth BioTherapeutics employees among authors; efficacy claims rest on baseline comparison.