Duchenne Muscular Dystrophy (DMD) Drugs Market

Global Duchenne Muscular Dystrophy (DMD) Drugs Market is valued at USD 2.1 Billion in 2021 and is expected to reach USD 16.6 Billion by 2028 with a CAGR of 34.2% over the forecast period.

Global Duchenne Muscular Dystrophy (DMD) Drugs Market: Global Size, Trends, Competitive, And Historical & Forecast Analysis, 2022-2028: Rising incidence of the condition, the emergence of mutation-specific therapies, and improved diagnostics along with government initiatives are some of the major factors driving the growth of the Global Duchenne Muscular Dystrophy (DMD) Drugs Market.

Scope-

Duchenne muscular dystrophy is a rare genetic illness with progressive muscle atrophy and weakening caused by sudden changes inside a body protein   called dystrophin. The illness is an X-linked recessive ailment mostly affecting males, with female carriers. While females are normally immune, some may display a lesser p henotype (“manifesting carriers”) such as heart abnormalities, moderate muscular weakness, and muscle cramps. Lack of dystrophin in muscle cells leads them to be weak and easily injured DMD is degenerative and most afflicted persons use a wheelchair by adolescence. Serious life-threatening problems including respiratory and cardiomyopathy issues finally develop and contribute to the death of DMD patients between 19 and 25 years of age.

Over the last five years, the FDA has also authorized a novel medicine dubbed exon-skipping treatments. These products include Vyondys 53 (for patients with mutations amenable to exon-53 skipping), Exondys 51 (for patients with mutants amenable to exon-51 skipping), Amondys 45 (for patients with mutations amenable to exon-45 skipping), and Viltepso (for patient populations with mutations amenable to exon-53 skipping) for DMD patients. These items are antisense oligonucleotides that overlook the mutant region of the dystrophin gene. This permits cells to connect a distinct set of exons together to form a protein that is smaller than typical but may have some function. These therapies have a superior tolerability and safety profile as compared to glucocorticoids.

The COVID-19 pandemic is projected to have a detrimental influence on the development of the worldwide Duchenne muscular dystrophy market. However, during the pandemic, despite the instructions to keep social distance, many persons treated their ailments by self-medication or other unorthodox techniques due to their anxiety about getting COVID during the lockdown. As of October 2020, the rate of COVID-19 virus among DMD/BMD sufferers was 6%, which was marginally lower than the prevalence of COVID-19 infection across all children in Israel, which was 8% as per PCR testing and 7% according to serology screening. During the pandemic, this is projected to have a major effect on the market.

The global Duchenne Muscular Dystrophy (DMD) Drugs market is segmented based on the type, distribution channel, and region & country level. Based on type, the global Duchenne Muscular Dystrophy (DMD) Drugs market is segmented into molecular therapies, steroid therapy, Non-steroidal Anti-Inflammatory Drugs (NSAIDs), and others. By distribution channel, the global Duchenne Muscular Dystrophy (DMD) Drugs market is segmented into hospital pharmacies, retail pharmacies, and online pharmacies.

The regions covered in the global Duchenne Muscular Dystrophy (DMD) Drugs market report are North America, Europe, Asia-Pacific, Latin America, and the rest of the World. Based on country level, the market of global Duchenne Muscular Dystrophy (DMD) Drugs is sub divided into U.S., Mexico, Canada, U.K., France, Germany, China, Italy, India, Japan, Southeast Asia, Middle East Asia (Saudi Arabia, UAE, Egypt) GCC, Africa, etc.

Key Players-

Some major key players for the global Duchenne Muscular Dystrophy (DMD) Drugs market report cover prominent players like -

• Fibrogen Inc.
• BioMarin
• NS Pharma Inc.
• Nobelpharma Co. Ltd
• PTC Therapeutics
• Pfizer Inc.
• Sarepta Therapeutics
• Santhera Pharmaceuticals
• ReveraGen BioPharma
• Hoffmann-La Roche AG
• others.

Segmentation-

By Type:

• Molecular therapies
• Steroid therapy
• Non-steroidal Anti-Inflammatory Drugs (NSAIDs)
• Others

By Distribution Channel:

• Hospital pharmacies
• Retail pharmacies
• Online pharmacies

By Regional & Country Level:

• North America

  • U.S.
  • Canada

• Europe

  • U.K.
  • France
  • Germany
  • Italy

• Asia Pacific

  • China
  • Japan
  • India
  • Southeast Asia

• Latin America

  • Brazil
  • Mexico

• Middle East and Africa

  • GCC
  • Africa
  • Rest of Middle East and Africa

Rising incidence of the condition, the emergence of mutation-specific therapies, and improved diagnostics along with government initiatives are some of the major factors driving the growth of the Global Duchenne Muscular Dystrophy (DMD) Drugs Market

Almost all big corporations are currently focused on R&D, which is expected to have a substantial influence on the market in the next years. Significant technological developments in recent years may assist research procedures, and the expansion of scientific knowledge of illness causes has made ambitious goals more sensible than ever. Furthermore, there is a big opportunity for enterprises to perform trials and introduce new drugs owing to increased public awareness of the condition and the government's demand for treatment, resulting in the market developing in the future years.

Furthermore, mutation-specific medicines are likely to be game changers in the treatment of DMD. Furthermore, the number of campaigns to raise awareness of DMD disorder has increased, as has the advancement of mutation-specific treatments, the pervasiveness of the disease, the number of treatments in development, and the emergence of therapies such as Exondys51 and Translarna, in addition to corticosteroid-based anti-inflammatory drugs. Thus, innovative drugs and treatments, disease-modifying therapies, considerable backing from numerous corporations for drug development, and patient advocacy organizations on the legislative approval process are important driving forces in the worldwide market for DMD.

Drug makers are also concentrating on creating novel medications and treatments for newborns, young babies, and DMD patients in their later stages. The therapies for DMD have advanced rapidly in recent years. However, a substantial number of patients are still unable to benefit from treatments and pharmaceuticals owing to their lack of knowledge AND high cost. Recently, in September 2022, a group of scientists at the University of Portsmouth, CNRS, I-STEM, AFM in France, and Maj Institute of Pharmacology of the Polish Academy of Sciences discovered that DMD begins much earlier in cells destined into becoming muscle fibers, known as myoblasts, in a study which revealed that DMD starts much previously in cells destined to become muscle fibers, known as myoblasts.

Government efforts to encourage target-specific therapies and favorable reimbursement guidelines are projected to fuel market expansion. Furthermore, the increasing acceptability of targeted medicines like Translarna, Exondys51, and Emflaza is expected to expand the therapeutic area.

The high cost of treatments continues to be a constraint to the expansion of the DMD medication landscape. However, authorities in all major areas are implementing inexpensive healthcare policies, which are influencing company pricing tactics as well as the reimbursement environment. Rising cost concern is threatening to stymie premium price potential for emerging DMD treatments.

Stringent regulatory clearances and a lack of established methodologies for determining medication clinical effectiveness continue to be key obstacles to overcome. However, expanding treatment options for disadvantaged populations such as babies and non-ambulant patients is expected to bode well for the industry in the near future.

North America is Expected to Capture a Major Share in the Global Duchenne Muscular Dystrophy (DMD) Drugs Market

Due to new product advancements, high healthcare spending, and government awareness initiatives, North America is predicted to lead the worldwide Duchenne Muscular Dystrophy (DMD) Drugs market over the forecast period. The U.S. has dominated the regional market and is likely to maintain its position due to growing illness incidence and anticipated releases of promising pipeline prospects. Furthermore, the market is expected to grow as more clinical studies are done throughout the world, notably in the United States. Elamipretide, for example, gained Orphan Drug Designation from the FDA Office of Orphan Products Development in May 2022 for the therapy of DMD. Similarly, the DA authorized Sarepta Pharmaceuticals' Amondys 45, an antisense oligonucleotide for the therapy of Duchenne muscular dystrophy, in February 2021.

News-

Pfizer to Open Initial U.S. Sites in Phase 3 Trial of Interventional Gene Therapy for Ambulatory Patients with Duchenne Muscular Dystrophy

On April 28^th, 2022; Pfizer Inc. announced its plans to establish the first U.S. sites in the Stage 3 research testing the experimental mini-dystrophin genetic engineering, fordadistrogene movaparvovec, in ambulatory individuals with Duchenne muscular dystrophy.

Elamipretide, developed by Stealth BioTherapeutics, has been designated as an orphan drug by the FDA for the treatment of Duchenne muscular dystrophy

On May 12^th, 2022; Stealth BioTherapeutics Corp, a clinical-stage biotech company focusing on the development, discovery, and commercialization of innovative medicines for disorders requiring mitochondrial dysfunction, announced that the US Food and Drug Administration (FDA) Division of Rare Products Development has given Orphan Drug Designation to elamipretide for the treatment of individuals with DMD.

Report Analysis	Details
Historical data	2016 - 2021
Forecast Period	2022 - 2029
Market Size in 2021:	USD 2.1 Billion
Base year considered	2021
Forecast Period CAGR %:	34.2%
Market Size Expected in 2028:	USD 16.6 Billion
Tables, Charts & Figures:	175
Pages	300
Companies	Fibrogen Inc., BioMarin, NS Pharma Inc., Nobelpharma Co. Ltd, PTC Therapeutics, Pfizer Inc., Sarepta Therapeutics, Santhera Pharmaceuticals, ReveraGen BioPharma, Hoffmann-La Roche AG, and others.
Segments Covered	By Type, By Distribution Channel
Regional Analysis	North America, U.S., Mexico, Canada, Europe, UK, France, Germany, Italy, Asia Pacific, China, Japan, India, Southeast Asia, South America, Brazil, Argentina, Columbia, The Middle East and Africa, GCC, Africa, Rest of the Middle East and Africa