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

DUBLIN--()--Research and Markets (http://www.researchandmarkets.com/research/pdfp64/opportunityanalyze) has announced the addition of the "OpportunityAnalyzer: Duchenne Muscular Dystrophy - Opportunity and Market Analysis to 2019" report to their offering.

Duchenne muscular dystrophy (DMD) 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. The publisher 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. The publisher estimates that the DMD market was valued at $8.2m across the 6MM in 2014, and it is expected to sharply increase to approximately $990.0m in 2019, at a Compound Annual Growth Rate (CAGR) of 160.5%.

Key Findings

- 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 expected to emerge as the major R&D breakthrough and future market drivers for DMD.

- With ongoing research, large 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 new drugs.

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

Key Topics Covered:

1 Tables & Figures

2 Introduction

3 Disease Overview

4 Epidemiology

5 Current Treatment Options

6 Unmet Needs Assessment and Oppportunity Analysis

7 R&D Strategies

8 Pipeline Assessment

9 Pipeline Valuation Analysis

10 Appendix

Companies Mentioned

  • Acceleron
  • Akashi
  • Asklepios
  • BioMarin
  • BMS
  • Catabasis
  • Eli Lilly
  • Italfarmaco
  • Milo Biotechnology
  • Nobelpharma
  • Pfizer
  • Prosensa
  • PTC
  • Santhera
  • Sarepta
  • Shenzhen Beike
  • Summit
  • Taiho
  • Tarix

For more information visit http://www.researchandmarkets.com/research/pdfp64/opportunityanalyze

Contacts

Research and Markets
Laura Wood, Senior Manager
press@researchandmarkets.com
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For U.S./CAN Toll Free Call 1-800-526-8630
For GMT Office Hours Call +353-1-416-8900
U.S. Fax: 646-607-1907
Fax (outside U.S.): +353-1-481-1716
Sector: Pharmaceuticals

Contacts

Research and Markets
Laura Wood, Senior Manager
press@researchandmarkets.com
For E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call 1-800-526-8630
For GMT Office Hours Call +353-1-416-8900
U.S. Fax: 646-607-1907
Fax (outside U.S.): +353-1-481-1716
Sector: Pharmaceuticals