1. Market Research
  2. > Pharmaceutical
  3. > Therapy Market Trends
OpportunityAnalyzer: Duchenne Muscular Dystrophy - Opportunity and Market Analysis to 2019

OpportunityAnalyzer: Duchenne Muscular Dystrophy - Opportunity and Market Analysis to 2019

  • April 2015
  • 154 pages
  • ID: 3085664
In this report:
ON THE OTHER HAND, CATENA AND TADALAFIL ARE EXPECTED TO TREAT UP TO ##.##% OF ELIGIBLE DMD PATIENTS (FOR EXAMPLE, PATIENTS WITH DECLINING PULMONARY FUNCTION AND AMBULANT PATIENTS BETWEEN AGES FIVE AND ## YEARS, RESPECTIVELY, WHO ARE NOT RECEIVING THERAPIES IN ADDITION TO CORTICOSTEROIDS) BY 2019.
SAREPTA THERAPEUTICS HAS INITIATED US CLINICAL TRIALS FOR ETEPLIRSEN IN NON-AMBULANT PATIENTS AND IS EXPECTED TO HAVE THE FIRSTMOVER ADVANTAGE IN LABEL EXPANSION, WHICH IS EXPECTED TO BRING AN ADDITIONAL ##.##% IN SALES TO THE US MARKET IN 2018 FROM NEWLY ELIGIBLE NON-AMBULANT PATIENTS.

Summary

Table of Contents

Search Inside

OpportunityAnalyzer: Duchenne Muscular Dystrophy - Opportunity and Market Analysis to 2019

Briefy

Duchenne muscular dystrophy is a rare genetic disorder affecting approximately 1 in 3,500 newborn boys and is characterized by progressive muscle degeneration, loss of ambulation, and death by the late 20s. There are no marketed pharmacological therapies that are indicated for DMD. The current standard of care treatment involves generic corticosteroids which are used to marginally prolong muscle function but do not offer preventative treatment. The anticipated launch of PTC Therapeutics' Translarna in 2015 in the US and 5EU, followed by the 6MM launch of Exon-skipping therapies- BioMarin/Prosensa's drisapersen and Sarepta Therapeutics' eteplirsen, in 2015 and 2016, respectively, are set to change the treatment landscape and drive growth in the DMD market. GlobalData estimates that the uptake of Translarna and exon-skipping therapies will be very high due to the lack of effective disease-modifying therapies for DMD. GlobalData estimates that the DMD market was valued at $8.2m across the 6MM in 2014, and it is anticipated to sharply increase to approximately $990.0m in 2019, at a Compound Annual Growth Rate of 160.50 percent.

Key Findingss

Key questions & answers

- How will the DMD market landscape change within the 2014-2019 forecast periods in the 6MM?

- What are the most favourable late-stage pipeline drugs and how will their launch shape the future treatment landscape in the DMD market?

- How do the clinical and commercial attributes of late-stage pipeline drugs compare to one another and against existing treatment options?

- Which patient population are most likely to be targeted by late-stage pipeline drugs?

- What are the meaningful unmet needs and remaining opportunities in the DMD market?

Main results

- Due to strong demand from patient community and clinicians, high uptake is expected of pipeline drugs for DMD that are anticipated to launch during the 2014-2019 forecast period.

- Mutation-specific therapies are anticipated to emerge as the main R&D breakthrough and future market drivers for DMD.

- With ongoing research, important pharmaceutical companies, small biotechs, and research institutions have discovered a multitude of therapeutic strategies to treat DMD. There are 19 ongoing clinical trials in DMD with 11 molecules in Phase II and eight molecules in Phase I.

- The lack of consensus over clinical trial design and endpoints for the development of DMD therapies continues to be a barrier to clinical development of additional drugs.

- The clinical stage pipeline is primarily focussed on ambulatory DMD patients with particular mutations, leaving a big unaddressed population and vast opportunity for developers to deliver additional therapies, and for continued growth in the DMD market beyond 2019.

Overviewe

- Overview of DMD, including epidemiology, etiology, pathophysiology, symptoms and current treatment options

- Annualized DMD therapeutics market revenue, annual cost of therapies and estimations for five years to 2019.

- Key topics covered include tactical product assessment, market characterization, unmet needs, R&D strategies, clinical trial design and implications for the DMD therapeutics market.

- Pipeline analysis: exhaustive data split across distinct phases, emerging trends and mechanisms of action under development, including nonsense mutation readthrough inducer, exon-skipping therapies, synthetic electron transporter, PDE5 inhibitor.

- Analysis of the current and future market competition in the U.S. and five main EU DMD therapeutics market. Clinical and commercial benchmarking of encouraging pipeline products versus standard of care treatments and ambitious assessment of all therapies. Insightful review of the top industry drivers, restraints and challenges.

Great reasons to purchasey

- Identify the unmet needs and remaining opportunities in the DMD therapeutics market.

- Develop strategic management by understanding the trends shaping and driving the U.S. and five main EU DMD therapeutics market.

- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a challenging advantage.

- Assess the clinical and commercial viability of favorable pipeline products.

- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies.

- Specified effective sales and marketing strategies by understanding the ambitious landscape and by analyzing the performance of different emerging therapies.

- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and tactical partnerships.

- Drive revenues by understanding the main trends, innovative products and technologies, market and segments likely to impact the U.S. and five main EU DMD therapeutics market in future.

Get Industry Insights. Simply.

  • Latest reports & slideshows with insights from top research analysts
  • 150+ Million searchable statistics with tables, figures & datasets
  • More than 25,000 trusted sources
  • Single User License — provides access to the report by one individual.
  • Department License — allows you to share the report with up to 5 users
  • Site License — allows the report to be shared amongst all employees in a defined country
  • Corporate License — allows for complete access, globally.
Veronica helps you find the right report:
Testimonials

The research specialist advised us on the best content for our needs and provided a great report and follow-up, thanks very much we shall look at ReportLinker in the future.

Kate Merrick

Global Marketing Manager at
Eurotherm by Schneider Electric

We were impressed with the support that ReportLinker’s research specialists’ team provided. The report we purchased was useful and provided exactly what we want.

Category Manager at
Ikea

ReportLinker gave access to reliable and useful data while avoiding dispersing resources and spending too much time on unnecessary research.

Executive Director at
PwC Advisory

The customer service was fast, responsive, and 100% professional in all my dealings (...) If we have more research needs, I'll certainly prioritize working with ReportLinker!

Scott Griffith

Vice President Marketing at
Maurice Sporting Goods

The research specialist provided prompt, helpful instructions for accessing ReportLinker's product. He also followed up to make sure everything went smoothly and to ensure an easy transition to the next stage of my research

Jessica P Huffman

Research Associate at
American Transportation Research Institute

Excellent customer service. Very responsive and fast.

Director, Corporate Strategy at
Ingredion

I reached out to ReportLinker for a detailed market study on the Air Treatment industry. The quality of the report, the research specialist’s willingness to solve my queries exceeded my expectations. I would definitely recommend ReportLinker for in-depth industry information.

Mariana Mendoza

Global Platform Senior Manager at
Whirlpool Corporation

Thanks! I like what you've provided and will certainly come back if I need to do further research works.

Bee Hin Png

CEO at
LDR Pte Ltd

The research specialist advised us on the best content for our needs and provided a great report and follow-up, thanks very much we shall look at ReportLinker in the future.

Kate Merrick

Global Marketing Manager at
Eurotherm by Schneider Electric

FAQ
  • How we can help
    • I am not sure if the report I am interested in will fulfill my needs. Can you help me?
    • Yes, of course. You can call us at +33(0) 4 37 65 17 03 or drop us an email at researchadvisor@reportlinker.com to let us know more about your requirements.
    • We buy reports often - can ReportLinker get me any benefits?
    • Yes. Set up a call with a Senior Research Advisor to learn more - researchadvisor@reportlinker.com or +33(0) 4 37 65 17 03.
    • I have had negative experiences with market research reports before. How can you avoid this from happening again?
    • We advise all clients to read the TOC and Summary and list your questions so that we can get more insight for you before you make any purchase decision. A research advisor will accompany you so that you can compare samples and reports from different sources, and choose the study that is right for you.

  • Report Delivery
    • How and when I will receive my Report?
    • Most reports are delivered right away in a pdf format, while others are accessed via a secure link and access codes. Do note that sometimes reports are sent within a 12 hour period, depending on the time zones. However, you can contact us to escalate this. Should you need a hard copy, you can check if this option is offered for the particular report, and pay the related fees.
  • Payment conditions
    • What payment methods do you accept?
      1. Credit card : VISA, American Express, Mastercard, or
      2. You can download an invoice to pay by wire transfer, check, or via a Purchase Order from your company, or
      3. You can pay via a Check made out in US Dollars, Euros, or British Pounds for the full amount made payable to ReportLinker
    • What are ReportLinker’s Payment Terms?
    • All payments must normally be submitted within 30 days. However, you can let us know if you need extended time.
    • Are Taxes and duties included?
    • All companies based in France must pay a 20% tax per report. The same applies to all individuals based in the EU. All EU companies must supply their VAT number when purchasing to avoid this charge.
    • I’m not satisfied. Can I be refunded?
    • No. Once your order has been processed and the publisher has received a notification to send you the report, we cannot issue any refund or cancel any order. As these are not ‘traditional’ products that can be returned, reports that are dispatched are considered to be ‘consumed’.
  • User license
    • The license that you should acquire depends on the number of persons that need to access the report. This can range from Single User (only one person will have the right to read or access the report), or Department License (up to 5 persons), to Site License (a group of persons based in the same company location), or Corporate License (the entire company personnel based worldwide). However, as publishers have different terms and conditions, we can look into this for you.
Purchase Reports From Reputable Market Research Publishers
Dry Eye Syndrome: Global Drug Forecast and Market Analysis to 2026

Dry Eye Syndrome: Global Drug Forecast and Market Analysis to 2026

  • $ 10995
  • Industry report
  • June 2018
  • by GlobalData

Dry Eye Syndrome: Global Drug Forecast and Market Analysis to 2026SummaryDry Eye Syndrome (DES) is a multifactorial disease of the tears and ocular surface that causes ocular discomfort, visual disturbances, ...

Gene Therapy in Oncology

Gene Therapy in Oncology

  • $ 7995
  • Industry report
  • June 2018
  • by GlobalData

Gene Therapy in OncologySummaryGene therapy refers to any procedure that aims to treat or alleviate a disease by genetically modifying the cell of a patient. The material to be transferred may consist ...

Neuroblastoma - Market Insights, Epidemiology and Market Forecast-2027

Neuroblastoma - Market Insights, Epidemiology and Market Forecast-2027

  • $ 6250
  • Industry report
  • June 2018
  • by Delve Insight

DelveInsight’s ‘Neuroblastoma - Market Insights, Epidemiology and Market Forecast-2027’ report delivers an in-depth understanding of the disease, historical & forecasted epidemiology as well as the ...


ref:plp2015

Reportlinker.com © Copyright 2018. All rights reserved.

ReportLinker simplifies how Analysts and Decision Makers get industry data for their business.

Make sure you don’t miss any news and follow us on