The Inherited Mitochondrial Disorders drugs in development market research report provides comprehensive information on the therapeutics under development for Inherited Mitochondrial Disorders, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. GlobalData’s report assesses key aspects of the companies and drugs in development for Inherited Mitochondrial Disorders. Buy the report here.

Smarter leaders trust GlobalData

The report also covers the descriptive pharmacological action of the therapeutics and the latest news and press releases. Additionally, the report provides an overview of the key players involved in therapeutic development for Inherited Mitochondrial Disorders and features dormant and discontinued products.

GlobalData tracks 22 drugs in development for Inherited Mitochondrial Disorders by 19 companies/universities/institutes. The top development phase for Inherited Mitochondrial Disorders is preclinical with 13 drugs in that stage. The Inherited Mitochondrial Disorders pipeline has 19 drugs in development by companies and three by universities/ institutes. Some of the companies in the Inherited Mitochondrial Disorders pipeline products market are: UCB, Khondrion and Ixchel Pharma.

The key targets in the Inherited Mitochondrial Disorders pipeline products market include Prostaglandin E Synthase (Microsomal Glutathione S Transferase 1 Like 1 or Microsomal Prostaglandin E Synthase 1 or p53 Induced Gene 12 Protein or PTGES or EC, Peptidyl Prolyl Cis-Trans Isomerase F Mitochondrial (Cyclophilin D or Cyclophilin F or Mitochondrial Cyclophilin or Rotamase F or PPIF or EC, and Nuclear Factor Erythroid 2 Related Factor 2 (HEBP1 or Nuclear Factor Erythroid Derived 2 Like 2 or NFE2L2 or NRF2).

The key mechanisms of action in the Inherited Mitochondrial Disorders pipeline product include cGMP Specific 3′,5′ Cyclic Phosphodiesterase (cGMP Binding cGMP Specific Phosphodiesterase or PDE5 or PDE5A or EC Inhibitor with one drug in Unknown. The Inherited Mitochondrial Disorders pipeline products include four routes of administration with the top ROA being Oral and five key molecule types in the Inherited Mitochondrial Disorders pipeline products market including Small Molecule, and Gene Therapy.

Inherited Mitochondrial Disorders overview

Inherited mitochondrial disorders represent a group of genetic conditions characterized by abnormalities in mitochondrial DNA or nuclear DNA, impacting mitochondrial function. These disorders affect various organs with high energy demands, such as the brain, muscles, and heart, leading to a wide range of clinical manifestations. Examples include mitochondrial myopathy, Leigh syndrome, and MELAS (mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke-like episodes). Diagnosis involves genetic testing and clinical evaluation, while management focuses on symptom alleviation and supportive care, as curative therapies are currently limited.

For a complete picture of Inherited Mitochondrial Disorders’s pipeline drug market, buy the report here.

Data Insights


The gold standard of business intelligence.

Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s pipeline drugs offers detailed profiles of pharmaceutical drugs in all stages of pre-clinical and clinical development, from discovery through to pre-registration. Coverage is limited to novel human medicinal drugs and biosimilars seeking market approval proprietary and is one of two primary repositories of pharmaceutical drug information offered by GlobalData through its Pharmaceutical Intelligence Center.