Market Challenges And Opportunities

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 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.

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.