Market Trends of Duchenne Muscular Dystrophy Treatment Industry
The Exon-skipping Segment is Expected to Hold a Major Market Share Over the Forecast Period
Internal deletions in the gene for dystrophin, a protein crucial for preserving the integrity of muscle cell membranes, are the primary cause of Duchenne muscular dystrophy. One prospective therapeutic strategy is to mask an exon near the location where the others are missing to allow the remaining exons to come together. Exon-skipping is one of the most effective treatment strategies for restoring the expression of a shortened but functional dystrophin protein.
Moreover, market players are focusing on frequent launches in exon skipping technology, which is a significant factor driving the segment, thereby propelling the market growth. For instance, in June 2022, Novartis announced that the European Commission (E.C.) approved Tabrecta (capmatinib) as a monotherapy for the treatment of adults with advanced non-small cell lung cancer (NSCLC) that has mutations that cause exon 14 (METex14) skipping and who need systemic therapy after receiving prior immunotherapy and/or platinum-based chemotherapy. Similarly, in February 2021, Sarepta Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) had approved AMONDYS 45 (casimersen). AMONDYS 45 is an antisense oligonucleotide from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 45 skipping.
Therefore, owing to product launches and technological advancements, considerable segment growth is expected over the forecast period.
North America Region is Expected to Witness Considerable Growth Over the Forecast Period
The North American region is expected to grow considerably due to new product innovations, high healthcare expenditure, and government awareness programs. Due to the increased disease prevalence and anticipated launches of attractive pipeline candidates, the United States has led the regional market and is expected to keep this position. Additionally, the market is anticipated to expand as more clinical trials are conducted globally, particularly in the United States. For instance, Elamipretide received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development in May 2022 to treat patients with Duchenne mascular dystrophy (DMD). Similarly, in February 2021, the FDA approved Sarepta Therapeutics' Amondys 45 (casimersen injectable), an antisense oligonucleotide, for treating people with Duchenne muscular dystrophy. Additionally, Viltepso (viltolarsen) injection received accelerated clearance from the U.S. Food and Drug Administration in August 2020 for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a verified mutation of the DMD gene that is susceptible to exon 53 skipping. Thus, such factors are anticipated to propel market growth in the North American region.
The Duchenne muscular dystrophy treatment market is expected to grow significantly in the United States due to huge unmet needs and favorable initiatives by the key players. For instance, in April 2022, Pfizer Inc. announced plans to open the first United States sites in the Phase 3 study evaluating the investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne muscular dystrophy (DMD). Furthermore, according to market estimates for DMD disease, pipeline drugs are expected to conquer the market in the coming years, causing exon-skipping therapies' market share to shrink threefold and steroids' market share to decrease by half. Eight new medications in the pipeline, the high price of gene and cell therapies, and the anticipated increase in treatment rates will all be significant growth drivers for the DMD market. Thus, significant growth in market growth is expected over the forecast period in the United States.
