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Primary mitochondrial myopathies: elamipretide is well tolerated but does not improve the 6-minute walk test or fatigue
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The results of the international MMPOWER-3 trial evaluating the safety and efficacy of double-blind, placebo-controlled subcutaneous elamipretide in 218 adults with primary mitochondrial myopathy have been published:
- the majority of participants (74%) had a mitochondrial DNAmacromolécule complexe, l’ADN est le support de l’hérédité (gènes). C’est le constituant des chromosomes. L’ADN est organisé en double hélice (deux brins complémentaires) et constitué de nucléotides de quatre types : adénine, guanine, cytosine et thymine. mutationModification soudaine et transmissible du matériel génétique. Elle peut être spontanée ou induite par des agents dits » mutagènes » (radiations, produits toxiques,…)., the others a nuclear DNA mutation;
- elamipretide subcutaneous for 6 months was well tolerated;
- neither the gain in distance covered in the 6-minute walk test, nor the improvement in the PMMSA (Primary Mitochondrial Myopathy Symptom Assessment) total fatigue score at the end of 6 months, the two main endpoints of the trial, were significantly different with elamipretide or placeboLe placebo est un produit dont la présentation est identique à celle d’un médicament, mais qui ne contient pas de principe actif. Dans un essai clinique, un placebo est utilisé pour mesurer l’action réelle du médicament testé, en comparant les effets du médicament testé contenant le principe actif et ceux du placebo.;
- however, a post-hoc analysis showed a significant improvement in the 6-minute walk test in participants with a nuclear DNA mutation;
- the NuPower trial, currently underway, is evaluating the efficacy of 48 weeks of subcutaneous elamipretide in 130 participants with primary mitochondrial myopathy associated with a nuclear DNA mutation.
Since May 2022, elamipretide has had orphan drug status for myopathic mitochondrial DNA depletion syndromes in Europe.