Global Neuromuscular Disease Therapeutics Market - 2022-2029

Global Neuromuscular Disease Therapeutics Market - 2022-2029

Market Overview

The global neuromuscular disease therapeutics market size was valued at US$ XX million in 2021 and is estimated to reach US$ XX million by 2029, growing at a CAGR of XX % during the forecast period (2022-2029).

Neuromuscular disorders affect the peripheral nervous system, including all motor and sensory nerves that connect the brain and spinal cord to the rest of the body. The most common symptom of these disorders is progressive muscle weakness.

Market Dynamics

The factors influencing the global neuromuscular disease therapeutics market are the increasing research and development and the growing prevalence of neuromuscular disease.

The increasing research and development is expected to drive the market growth in the forecast period

Neuromuscular diseases (NMDs) are a broad category of disorders that cause progressive motor function impairment. Motor neurons (MNs), skeletal muscle (SkM), or their synaptic connection, the neuromuscular junction, are the most common NMDs (NMJ). MNs [e.g., amyotrophic lateral sclerosis (ALS) caused by direct loss or retrograde degeneration of MNs], muscle [e.g., Duchenne muscular dystrophy (DMD) or myotonic dystrophy (DM)], NMJs [e.g., myasthenia gravis (MG) or congenital myasthenic syndromes (CMS)], or a combination of these [e.g. (e.g., Pompe disease). Regardless of its source, the structural and functional deficit in a targeted tissue will resonate throughout the motor unit, resulting in multiple shared symptoms among different NMDs.

Methods to differentiate hiPSCs into neuronal cells have advanced rapidly in the last decade, thanks to an increased understanding of early neural development and the commitment of neuroprogenitor cells to highly specialized neural subtypes, including MNs. MNs are found throughout the CNS and are divided into upper MNs (UMNs) and lower MNs (LMNs), which are developmentally and genetically distinct despite their shared nomenclature. Different NMDs can target UMNs and LMNs, making their distinction important when modelling NMDs. The cerebral cortex's pre-motor and primary motor regions are where UMNs originate. As a result, many recent breakthroughs in neuromuscular disease therapy development have been made. There are nearly 200 products in the pipeline for neuromuscular disorders, with a roughly 50:50 ratio of preclinical to clinical development. Most target amyotrophic lateral sclerosis (ALS) and Duchenne's muscular dystrophy (DMD). Recent technological advancements have aided the development of several different types of therapies. In 2018, small molecules (targeting receptor modulation, epigenetic reprogramming, redox metabolism, and other mechanisms), gene therapy, and antisense oligonucleotides (ASOs) accounted for nearly half of the nearly 200 potential therapies for neuromuscular diseases under development (Aitken et al., 2018). The US Food and Drug Administration (FDA) recently approved ASO and gene therapy.

The side effects associated with neuromuscular disease therapeutics are expected to hamper the market growth

Myasthenia gravis (MG) is a neuromuscular junction disorder that causes varying degrees of weakness in the body's skeletal muscles. Although there is currently no cure for MG, traditional therapies such as acetylcholinesterase inhibitors and immunomodulating therapies (corticosteroids, azathioprine, and mycophenolate mofetil) enable most patients to live productive lives with an average life expectancy. These therapies, however, are associated with side effects such as nausea, vomiting, gastrointestinal (GI) upset, increased infection risk, weight gain, and liver damage, which can deter patients from using them. Poor adherence is also a result of unsatisfactory treatment outcomes with traditional MG therapies, with 23 percent of patients reporting poor treatment compliance. Despite this, patient-reported information on the exact nature, duration, and severity of these side effects are scarce.

COVID-19 Impact Analysis

The COVID-19 pandemic has prompted a rapid reorganization of hospital settings and patient service provision to deal with emerging but unmet medical needs. The prevention strategies, in particular, have an impact on the management of patients with NMD. Immune-mediated pathogenesis is linked to some types of NMD. Patients with NMD who receive immunomodulatory therapy (IMT) are more likely to contract COVID-19 infections that are more severe. In order to guide patients and clinicians during the COVID-19 pandemic, a consensus statement on IMT management has recently emerged. Patient compliance and caregiver support, dose reduction of certain IMTs, or switching to alternative agents for high-risk NMD patients can all be considered depending on the pandemic burden of the region. NMD experts should be consulted before temporarily suspending, reducing, or changing IMT, and patients should not proceed without first consulting them. Hence, covid-19 is expected to have impacted the global neuromuscular disease therapeutics market due to delays in appointments and fear of contraction of covid.

Segment Analysis

Spinal Muscular Atrophy (SMA) segment is expected to dominate the market growth in the forecast period

Spinal muscular atrophy (SMA) is a genetic disorder characterized by muscle wasting (atrophy) and weakness (skeletal muscles). It is caused by the loss of motor neurons, which are specialized nerve cells that control muscle movement. When comparing muscles close to the center of the body (proximal) to muscles further away from the center of the body (distal), the weakness is more severe in the proximal muscles (distal). Muscle weakness usually worsens as people get older. Changes in the same genes cause many different types of spinal muscular atrophy. The types differ in terms of onset age and severity of muscle weakness, but there is some overlap. Other types of spinal muscular atrophy and related motor neuron diseases are caused by mutations in other genes, such as spinal muscular atrophy with progressive myoclonic epilepsy, spinal muscular atrophy with lower extremity predominance, and X-linked infantile spinal muscular atrophy, and spinal muscular atrophy with respiratory distress type 1. Around the world, 1 in 8,000 to 10,000 people suffer from spinal muscular atrophy. The most common spinal muscular atrophy type is type I, which accounts for roughly half of all cases.

Moreover, on 29th July 2021, Roche launched Evrysdi, a prescription medicine used to treat spinal muscular atrophy (SMA) in adults and children aged 2 months and older, in India, at a maximum retail price of around Rs 6 lakh per bottle. The US FDA first approved Evrysdi in August 2020, and it is now available in India just 11 months later. Hence, the increasing FDA approvals and growing prevalence of spinal muscular atrophy (SMA) are expected to boost the market growth.

Geographical Analysis

North America region is expected to hold the largest market share in the global neuromuscular disease therapeutics market

The increasing prevalence of neuromuscular diseases and increasing FDA approvals in this region is expected to boost the market growth. For instance, neuromuscular diseases (NMDs) are a broad category of disorders that affect an estimated 250,000 people in the United States.

Moreover, on 17th December 2021, Vyvgart (efgartigimod) is approved by the US Food and Drug Administration for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody.

Competitive Landscape

The global neuromuscular disease therapeutics market is moderately competitive with mergers, collaborations, and product launches. Some of the key players in the market are Biogen Inc., Brainstorm Cell Therapeutics Inc., F. Hoffmann-La Roche Ltd., Novartis AG, Pfizer Inc., PTC Therapeutics Inc., Sanofi SA, Sarepta Therapeutics Inc., NMD Pharma, BioMarin

Sarepta Therapeutics Inc.

Overview: Sarepta Therapeutics, Inc. is a medical research and drug development company based in Cambridge, Massachusetts, with headquarters and research facilities. The company was founded in 1980.

Product Portfolio: LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4) is a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan (beta-SG) proteins. SRP-9003 is being developed for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4). SRP-9003 is a gene therapy construct that transduces skeletal and cardiac muscle and delivers a gene that codes for the full-length beta-SG protein, the sole cause of the disease's progressive degeneration and shortened lifespan.

Key Development: On 2nd February 2022, Sarepta Therapeutics partnered with GenEdit to develop gene-editing therapies for neuromuscular diseases through a research partnership and option agreement. The alliance will use GenEdit's NanoGalaxy platform and Sarepta's gene-editing technology to develop the therapies.

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