Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a severe type of muscular dystrophy characterized by progressive muscle degeneration and weakness. It is caused by mutations in the DMD gene, which encodes the protein dystrophin, essential for muscle fiber strength and stability. DMD primarily affects boys, with symptoms typically appearing between the ages of 2 and 5. As the disease progresses, it leads to loss of ambulation and respiratory and cardiac complications, significantly reducing life expectancy. The rising innovations in genetic research and increasing awareness and understanding of DMD among healthcare professionals and the public have led to earlier and more accurate diagnoses.

The Duchenne Muscular Dystrophy Market is expected to grow around ~7% during the forecast period (2024-2032). This is mainly because expanding treatment options will give patients access to a broader range of treatment options, enabling growth and penetration in the Duchenne Muscular Dystrophy industry as more therapies receive regulatory approval. For instance, on March 21, 2024, the U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for treating Duchenne Muscular Dystrophy (DMD) in patients six years of age and older. Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. This expansion will enhance personalized care approaches, allowing for better disease management based on individual patient needs.

• Based on the marketed therapies, the market has been divided into Vyondys 53, Viltepso, Emflaza, Exondys 51, Translarna, and Amondys 45. The Vyondys 53 is expected to grow with a significant CAGR in the forecast period (2024-2032). VYONDYS 53 is an FDA-approved Duchenne muscular dystrophy treatment for patients with a confirmed genetic mutation in the dystrophin gene that can be treated by skipping exon 53. In some patients, it helps the body make a shorter form of the dystrophin protein. Golodirsen was developed by Sarepta Therapeutics and granted accelerated FDA approval on December 12, 2019, due to the urgent need for this drug in patients suffering from a particular form of DMD. Golodirsen is a morpholino antisense oligomer that treats about 8% of Duchenne Muscular Dystrophy (DMD) patients.
• For a better understanding of the market adoption of Duchenne Muscular Dystrophy, the market is analyzed based on its 7MM presence in countries such as The United States, Germany, UK, France, Italy, Spain, Japan. The United States held a dominant share of the market in 2023 due to increasing awareness and access to genetic testing in the U.S., leading to early and accurate diagnosis of DMD. Moreover, The U.S. Food and Drug Administration (FDA) provides various incentives, such as orphan drug designation and accelerated approval pathways, encouraging pharmaceutical companies to invest in DMD treatment development. For instance, on June 22, 2023, the U.S. Food and Drug Administration approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who do not have a pre-existing medical reason preventing treatment with this therapy.
• Some of the major players operating in the market include Sarepta Therapeutics, Inc.; PTC Therapeutics; NIPPON SHINYAKU CO., LTD.; Pfizer Inc.; TAIHO PHARMACEUTICAL CO., LTD.; Percheron Therapeutics Limited; DAIICHI SANKYO COMPANY, LIMITED; Solid Biosciences Inc.; FibroGen, Inc.; Capricor Therapeutics, Inc.