AOC 1001, an investigational treatment for Myotonic Dystrophy Type 1 (DM1), a late-developing muscle disorder, has received fast track designation from the U.S. Food and Drug Administration (FDA). The FDA’s fast track program’s overall goal is to expedite the review of therapies that have the potential to improve the care of people with serious illnesses. Avidity Biosciences, the company behind AOC 1001, will be able to communicate with the FDA more frequently due to the designation.

Source: Avidity Biosciences

Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy in adults. DM1 is a dominant disorder characterized by multisystemic clinical features affecting skeletal muscle, heart, and the nervous and endocrine systems.

• The prevalence varies by region and is most common in individuals of European descent. According to Thelansis disease modeling data, literature reviews, surveys, and registry analyses combined prevalence reported as 1 in 8,000 (12.5 per 100,000).
• DM1 is caused by expansion of a CTG repeat in the 3′ noncoding region of the DMPK gene on chromosome 19q13.3, which codes for the myotonic dystrophy protein kinase.
• Normal individuals have between 5 and 37 repeats, but symptomatic patients typically have > 50 repetitions.
• DM1 is categorized into four subtypes: congenital, childhood, classic, and minimal / late-onset.

However, the current Myotonic Dystrophy Type 1 (DM1) treatment market share, market uptake, attribute analysis concerning the most potential emerging therapies (AOC 1001, ERX-963, Pitolisant, Tideglusib, SomatoKine/IPLEX, Ranolazine, and IONIS-DMPKRx) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for Myotonic Dystrophy Type 1 (DM1) treatment. The key companies in the advanced development stage are Avidity Biosciences, Inc., Expansion Therapeutics, Inc., Harmony Biosciences, LLC, AMO Pharma Limited, etc.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook report on Myotonic Dystrophy Type 1 (DM1) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2020 to 2030.

Thelansis specializes in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area, including rare / ultra-rare and mainstream indication. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

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