Charcot–Marie–Tooth_Market Outlook and Competitive Landscape Report 2018 – 2030

Report Overview

 The report provides a comprehensive view of disease events, types, severity, progression, symptoms, risk factors, pathogenesis and treatment guidelines at country level

 Comprehensive insight on patient segmentation has been provided into the epidemiology of the Charcot–Marie–Tooth (CMT) and its treatment in the 8 MM countries, covering the United States, EU5 (Germany, Spain, France, Italy, UK), Japan, and China

 Drug utilization and analysis on the current clinical practice, KOL perspective on upcoming therapies, estimated TPP for pipeline assets, clinical characteristics upcoming therapies and future treatment paradigm

 Market aggregation on a newly diagnosed patient pool, Thelansis estimation on persistence, compliance, adherence and a historic view of changing market dynamics and product-level patient flow of Charcot–Marie–Tooth (CMT) in the 8 MM Countries for the period 2018-2030

 A comprehensive record of both the present and rising treatments for CMT are provided, along with the impact of the new treatments and patent expiry on the current clinical practice and overall market level

 A detailed review of 8MM current and forecasted CMT market is included in the report, covering drug outreach in the 8 MM Countries based on their order of entry

Epidemiology

 With reference to the study titled- “Management of Charcot–Marie–Tooth disease: improving long-term care with a multidisciplinary approach” by McCorquodale et al. 2016 the prevalence of CMT was ~2500 but it exhibited widespread variations across the territory ranging from one in ~1,200 to 9,200 in the USA

 Among the subtype of CMT, Type 1 considered as the most common category representing approximately ~75% of all CMT cases and the prevalence of CMT Type 2, on the other hand, ranges from 10-15 % of all CMT cases and comprises of the second major subtype of CMT

 According to Thelansis, globally ~ 2.5 MN patients are being diagnosed worldwide with Charcot–Marie–Tooth (CMT) among which 8MM countries are comprised of ~25% of the total yearly reported cases

 Total prevalent population of Charcot–Marie–Tooth (CMT) in 8 MM ranges from 5,50,384 in 2018 to 5,93,353 in 2030

 Diagnosed prevalent population of CMT in 8MM is expected to increase at a CAGR of 0.73% for the study period i.e. 2018 - 2030

 The estimates suggest a higher prevalence of Charcot–Marie–Tooth (CMT) in the United States with 131,308 cases in 2018 and 143,087 diagnosed cases in 2030

 Among the European countries, Germany had the highest prevalent population of CMT with XXXXX cases, followed by France which had a prevalent population of XXXX in 2018. On the other hand, Spain had the lowest diagnosed prevalent population of XXXXX in 2018. Japan XXXXX prevalent cases of Charcot–Marie–Tooth in 2018

 Considering the country level perspective the EU5 are contributing around ~33% of the total diagnosed patient pool, however, in terms of market USA is leading considering the current disease burden and treatment cost

Competitive landscape

Emerging therapies:

 The pipeline for Charcot–Marie–Tooth (CMT) is intense and diverse MoAs are under evaluation. Key MoAs include GABA B receptor agonist, Opioid mu receptor antagonist, Opioid kappa receptor antagonist (PXT3003; Pharnext), TGF-beta inhibitors(ACE-083; Acceleron), and ATP production of against axonal degeneration & activating acetyl-CoA carboxylase (MD1003; MedDay)

 Majorly the developments around the CMT1 and CMTX is the sub-segment type-1 patient population, hence a positive impact on future treatment paradigm being expected

 In June 2014, European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to PXT-3003 for the treatment of Charcot-Marie-Tooth disease type 1A (CMT 1A) and again in 2018 followed by Fast track designation in Feb 2019. PXT3003 was granted Priority Review for Charcot-Marie-Tooth Type 1A disease (CMT1A) by the China Food and Drug Administration (CFDA).

 ACE-083 is the lead product candidate in the neuromuscular therapeutic program of Acceleron Pharma, the asset received Fast Track and Orphan Drug designations from the U.S. FDA for FSHD

 MedDay Pharmaceutical is using the SPECMET research platform for the development of MD-1003. The MedDay SPECMET platform supports the discovery of additional pipeline assets by identifying new therapeutic targets through metabolomics analysis of cerebrospinal fluid (CSF)

Current therapies:

 Currently there is no curative therapy for Charcot-Marie-Tooth Disease (CMT), however, some treatment options may relieve signs and symptoms and delay the start of physical disabilities

 NSAIDS and Ibuprofens are being prescribed to provide the symptomatic pain relief, however the center point for neuropathic pain not been addressed. In case of failure of NSAIDS, tricyclic antidepressants (TCAs) are prescribed

 Sometimes antiepileptic drugs such as gabapentin, pregabalin, topiramate being prescribed to treat neuropathic pain

 Surgical treatment options are used to fuse bones in the feet or to remove part of a tendon that can sometimes alleviate pain, Physical Therapies uses as low-impact exercises to strengthen and stretch the muscles

Companies asset Pipeline-

 Phase III Therapies- Pharnext,
 Phase IV Therapies- Acceleron Pharma, MedDay Pharmaceuticals,
 Phase I Therapies- Inflectis BioScience
 Pre-clinical Therapies- Recursion Pharmaceuticals, Addex Therapeutics, Myotherix, Sarepta Therapeutics, Regenancy Pharma, Ionis Pharmaceuticals, Acetylon, Avicena, Murigenetics, aTyr Pharma, PharmatriphiX

KOL perspective:

 “…..Better AFO (leg-bracing) options, fast-tracking drug discoveries and clinical trials, applying for disability benefits, higher market access will be combining change the current market scenario. These are a real challenge to the patients as well as the HCP’s within CMT space…..”
(Academic & Researcher, Division of Neurology, Stanford University School of Medicine)

 “….The disease definition and theory on the genetic basis are not clear across the healthcare practitioners, standardization of diagnostic equipment used to diagnose the patients in the clinical setting must to be looked at, however, pharmaceutical companies need to see beyond the conventional method of drug discovery
(Neurosurgeon, Parkland Memorial Hospital: Neurology Department)…..”

 “…..CMT is one of the unique rare diseases requires multiple diagnosis confirmations, however, more than 80% of clinical practitioners feel there is a huge gap between unmet need and current clinical practice, requires diagnostic equipment efficient enough to scan the target biomarkers….”
(Neurology Medicine, Jefferson University Hospitals)

 “….Standardized treatment guidelines to be enforced which will state the drug regimen to be prescribed as a combination therapy to do the pain management and enhance the quality of life…..”
(Neurology Medicine, Johns Hopkins Hospital in Baltimore, Maryland)

Payer’s perspective:

 In the USA both FDA, NORD (National Organization for Rare Disorders) and NSGC (National Society for Genetic Counselors) are mutually keeping eye on cost savings as both at public and private payer’s settings in the USA as the agencies more concern about strong clinical outcomes data which have great evidence on managing the symptomatic conditions including quality of life
 Both the USA FDA and European nations regulatory bodies and payers are having a very conservative mindset with respect to the approval and access of potential assets, and diagnosis procedures which will make sure the impact on country level disease burden

Thelansis, as the name depicts Thelansis is specialized in “Therapy Area Landscape Analysis” along with market intelligence and consultancy that supports pharmaceutical, biotechnology, and diagnostics companies to successfully prepare, launch, and commercialize their products. Our Syndicated research reports include Market Insight, Pipeline Insight, Product Insight, Specialized Reports, Indication Outlook and Market Access Reports. Our focused therapeutic approach provides our clients with clinical and disease area expertise from an integrated team of academic, medical, and industry specialists of our panel, Ask for our report offerings, and Indication specific sample pages by sharing your email ID with us at clientsupport@thelansis.com

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This release was published on openPR.