Mitochondrial Disease Therapies: Unlocking Treatment for Orphan Diseases

In the US, mitochondrial diseases are known to annually affect 1 in 5,000 individuals. It is also estimated that 1,000-4,000 children are born with a mitochondrial disease every year, in the same region. Primary mitochondrial diseases are usually difficult to diagnose. Such clinical conditions are rare and are known to affect multiple parts of the body, including neurons and nerves, kidneys, heart, liver, eyes, ears, and pancreas. It is worth highlighting that over 300 mutations that are associated with various mitochondrial diseases have been identified.

I have highlighted below some of the most interesting insights from our study. You can also access additional details here.

Despite various obstacles, mostly related to accurate diagnosis, currently, a number of personalized and off the shelf therapeutic options are being investigated for the treatment of primary mitochondrial diseases

Presently, around 60 therapies, including certain repositioned / repurposed drugs, are being evaluated for the treatment of primary mitochondrial diseases by both industry players and academic institutions, worldwide

Majority of drug / therapy candidates are in the early to mid stages of clinical development, and are designed to treat a variety of mitochondrial diseases exploiting the oral route of administration

Efforts to innovate in this field is evident from the R&D activity, which can be quantified in terms of volume of published scientific literature, focused on multiple diseases and having diverse aims and objectives

A number of prominent scientists from renowned universities, owing to their involvement in clinical development efforts, have emerged as key opinion leaders

Over the time, several non-profit organizations have extended financial support for research efforts in this domain; the current focus is on understanding the disease etiopathology and developing effective therapies

The success of the first approved therapy (Raxone®) will determine the current state of the market; the future growth is likely to be driven by the safety and efficacy outcomes of the late-stage molecules, once approved

The estimated future opportunity is likely to be distributed across diverse types of therapies, different classes of therapeutic interventions and routes of administration

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