Duchenne muscular dystrophy treatment market

The global Duchenne muscular dystrophy treatment market was valued at US$ 2,787.6 Mn in 2022 and is forecast to reach a value of US$ 63,519.4 Mn by 2030 at a CAGR of 47.85% between 2023 and 2030. The Duchenne muscular dystrophy treatment market is experiencing strong growth due to the increase in prevalence of Duchenne muscular dystrophy and increase in demand for novel drugs or therapies. Moreover, increase in research and development (R&D) and increase in awareness among people about DMD and available drugs/therapies is expected to boost the growth of the market. However, factors such as lack of standardization and stringent rules and regulations are expected to hamper growth of the market.

Global Duchenne Muscular Dystrophy Treatment Market: Regional Insights

Based on geography, the global Duchenne muscular dystrophy treatment market is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa.

Among regions, North America is expected to gain highest share in the market over the forecast period owing to the rising disease burden of Duchenne muscular dystrophy, introduction of novel therapies, high healthcare expenditure, and rise in awareness among people in the region. For instance, market players are focusing on launching novel drugs or therapies for the treatment of DMD. In May 2022, Elamipretide received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development for the treatment of patients with Duchenne muscular dystrophy.

Europe is also expected to witness significant growth in the global Duchenne muscular dystrophy treatment market due to the increase in prevalence of DMD, increase in awareness among people, increase in demand for safe and effective therapies in the region. For instance, with the increase in awareness among people about DMD, the demand for novel drugs or therapies is also increasing with a rapid pace. According to the European Medicines Agency, DMD affected less than 0.5 in 10,000 people in the European Union. This was equivalent to a total of fewer than 26,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000.

Figure 1. Global Duchenne Muscular Dystrophy Treatment Market Share (%), by Region, 2023

Global Duchenne Muscular Dystrophy Treatment Market Drivers:

Increase in burden of DMD to augment market growth

One of the key factors expected to augment the growth of the global Duchenne muscular dystrophy treatment market over the forecast period is the rise in prevalence of Duchenne muscular dystrophy. Such a high burden of DMD leads to an increase in demand for safe and effective drugs or therapies across the world. According to the National Organization for Rare Disorders (NORD), Duchenne muscular dystrophy is a rare muscle disorder but it is one of the most frequent genetic conditions affecting around one in 3,500 male births worldwide. It is estimated that around 20,000 children are diagnosed with DMD worldwide each year.

High demand for novel drugs/therapies to underpin market growth

Another factor which is driving the growth of the global Duchenne muscular dystrophy treatment market is the increase in demand for novel drugs or therapies around the world owing to increase in awareness among people. For instance, players in the market are focusing on launching novel drugs or therapies for the treatment of DMD. In August 2020, NS Pharma receive the U.S. FDA approval for viltolarsen (Viltepso) for the treatment of patient with DMD. The agent was granted accelerated approval via its priority review, fast track, orphan drug, and rare disease designations after its new drug application was accepted earlier this year.

Global Duchenne Muscular Dystrophy Treatment Market Opportunities:

Increase in research and development (R&D) is expected to offer significant growth opportunities for players in the global Duchenne muscular dystrophy treatment market. For instance, in January 2023, Regenxbio started recruiting patients for the Phase 1/2 clinical trial to assess the safety and effectiveness of RGX-202, an investigational gene therapy for DMD. Regenxbio is also recruiting DMD patients ages 12 or younger for an observational study to screen for antibodies against adeno-associated virus variant 8 (AAV8), the viral vector used in RGX-202. RGX-202 is granted orphan drug status by the U.S. Food and Drug Administration (FDA).  

Increase in awareness among people about DMD and available drugs/therapies is expected to offer significant growth opportunities for players in the global Duchenne muscular dystrophy treatment market. For instance, each year, the World Duchenne Awareness Day takes place on September 7. On this day, many government and non-governmental organizations from around the world raise awareness for Duchenne Muscular Dystrophy. Every year, World Duchenne Organisation sets a special theme to the day that deserves more attention. Awareness Day is created to address this lack of awareness and support people with the disease.

Global Duchenne Muscular Dystrophy Treatment Market Trends:

Increase in number of clinical trials is a recent trend

There has been a significant increase in the discovery and development of pharmaceutical drugs. Clinical trials are being conducted worldwide to evaluate potential treatments for DMD. Pharma companies and drug manufacturers worldwide have completed number of clinical trials for DMD over the past few years. Moreover, multiple potential candidate are currently in Phase II and III across the world. This trend is expected to continue over the forecast period.

Introduction of novel drugs/therapies is another trend

Players in the market are focusing on developing and launching safe and effective drugs/therapies for the treatment of Duchenne muscular dystrophy. Moreover, players in the market are focusing on launching exon skipping technology or technologies (exon skipping is a treatment approach for people with DMD), which is a significant factor augmenting the growth of the market. This trend is expected to continue over the forecast period.

Global Duchenne Muscular Dystrophy Treatment Market Restraints:

Lack of standardization to hinder market growth

Lack of standardization to measure clinical efficacy across all stages of DMD is one of the vital factors expected to hamper growth of the global Duchenne muscular dystrophy treatment market. There are quite a few on‐going clinical trials in DMD, which use the diverse outcome measures to quantify the disease progression, including the 6‐minute walking test, the performance of the upper limb, the time taken to rise from the floor, the time taken to climb four steps, and the north star ambulatory assessment scale. There is need for other better outcome measures which may capture the progression between ambulant and non‐ambulant phases of the disease.

Stringent regulatory framework to hamper market growth

Another factor which is hampering growth of the global Duchenne muscular dystrophy treatment market is the stringent rules and regulations. The approval of novel drugs or therapies by regulatory agencies, such as the US FDA and European Medicines Agency have stringent rules & regulations for drug approvals and a lot of procedures. Thus, very few companies (pharmaceutical companies spend huge amounts on the R&D of drugs) come into front for the new drug research for the rare diseases because it needs bigger funds.

Figure 2. Global Duchenne Muscular Dystrophy Treatment Market Share (%), by Therapeutic approach, 2023

Global Duchenne Muscular Dystrophy Treatment Market Segmentation:

The global Duchenne muscular dystrophy treatment market report is segmented into Therapeutic Approach and Treatment Type, End User, and Geography.

Based on Therapeutic Approach & Treatment Type, the market is segmented into Molecular-based Therapies (Mutation Suppression and Exon Skipping), Steroid Therapy (Corticosteroids), Nonsteroidal Anti-Inflammatory Drugs (NSAIDs), and Other Therapeutic Approaches and Treatment Types. Molecular-based Therapies further segmented into Mutation Suppression and Exon Skipping. Out of which, Exon Skipping Segment is expected to dominate the market over the forecast period and this is attributed to the introduction of novel exon skipping technologies. Around 50-90% of patients suffering from DMD mutations can benefit from single-exon skipping and multi-exon skipping, respectively.

Steroid Therapy (Corticosteroids) Segment is also expected to witness significant growth in the near future and this is due to the rise in demand for corticosteroids. Pharmacological corticosteroid therapy is the standard of care in DMD that aims to control symptoms and slow disease progression through potent anti-inflammatory action.

Based on End User, the market is segmented into Hospitals and Clinics, Ambulatory Centers, and Other End Users. Out of which, Hospitals/Clinics Segment is expected to dominate the market over the forecast period and this is attributed to the increase in prevalence or burden of DMD.

Ambulatory Centers Segment is also expected to witness robust growth in the near future and this is owing to increase in number of outpatient surgeries. Duchenne Muscular Dystrophy patients require minor surgery to be performed and needs only a primary care.

Global Duchenne Muscular Dystrophy Treatment Market: Key Developments

In April 2022, Pfizer Inc. planned to open the first U.S. sites in the Phase III study evaluating the investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne muscular dystrophy.

In February 2021, Sarepta Therapeutics, Inc. announced that the U.S. Food & Drug Administration (FDA) has approved AMONDYS 45 (casimersen). AMONDYS 45 is an antisense oligonucleotide from the Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, for the treatment of DMD in patients with a confirmed mutation amenable to exon 45 skipping.

In February 2021, the District Attorney (DA) of the United States approved Sarepta Therapeutics' Amondys 45 (casimersen injectable), an antisense oligonucleotide for the treatment of people with Duchenne Muscular Dystrophy.

Global Duchenne Muscular Dystrophy Treatment Market: Key Companies Insights

The Duchenne muscular dystrophy treatment market is highly competitive. This is attributed to the increase demand for safe and effective DMD treatment, as a result, players in the market are focusing on launching novel products in the market.

Some of the key players in the global Duchenne muscular dystrophy treatment market are Pfizer Inc., Fibrogen Inc., BioMarin, Sarepta Therapeutics, Santhera Pharmaceuticals, PTC Therapeutics, NS Pharma Inc., Nobelpharma Co. Ltd., Bristol-Myers Squibb, and Eli Lilly and Company.

*Definition: Duchenne muscular dystrophy is a form of muscular dystrophy. It is caused by a defective gene for dystrophin (a protein in the muscles). It is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of dystrophin (a protein) that helps keep muscle cells intact.   

Detailed Segmentation:

• Global Duchenne Muscular Dystrophy Treatment Market, By Therapeutic Approach and Treatment Type
  • Molecular-based Therapies
  • Steroid Therapy
  • Nonsteroidal Anti-Inflammatory Drugs (NSAIDs)
  • Other Therapeutic Approaches and Treatment Types
• Global Duchenne Muscular Dystrophy Treatment Market, By End User
  • Hospitals/Clinics
  • Ambulatory Centers
  • Other End Users
• Global Duchenne Muscular Dystrophy Treatment Market, By Geography
  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East and Africa