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Pipeline Insight: Orphan Diseases in Neurology - Part III: Myasthenia Gravis

Introduction

The myasthenia gravis market is set to grow from $44m in 2007 to $239m in 2017 across the seven major pharmaceutical markets in Datamonitor's base scenario forecast. Amarin's pipeline candidate EN101 is forecast to become the market leading brand with sales of $153m in 2017.

Scope

  • Scenario based analysis and forecasts of the commercial potential of the myasthenia gravis market to 2017.
  • Analysis of the patient potential, commercial viability, and unmet needs of the myasthenia gravis market.
  • Indication-specific sales forecasts of marketed and pipeline drugs to 2017.
  • Assessment of the treatment algorithms used in this disease.

Highlights

Although myasthenia gravis is an uncommon disease (106,000 patients across the seven major markets), it is more prevalent than Huntington's disease or amyotrophic lateral sclerosis (31,000 and 34,000 patients, respectively), both orphan neurological diseases with larger late-stage pipelines.

In the majority of patients, symptoms can be well controlled with current treatments. However, very poor side-effect profiles and inconvenient dosing regimens of these therapies impact on the patient's quality of life, and represent key unmet needs in myasthenia gravis treatment.

Amarin's EN101 is forecast to become the market leading brand with sales of $153m in 2017 in Datamonitor's base scenario. In an alternative scenario EN101 proves to be steroid sparing enhancing its commercial potential ($621m in 2017). However, poor bioavailability of RNA therapies enhances the risk of EN101 failing in larger scale trials.

Reasons to Purchase

  • Access the lucrative potential of this underdeveloped neurological auto-immune disease.
  • Benchmark key late-stage compounds against current leading therapies.
  • Understand the opportunities in this market based on the comments of interviewed key opinion leaders.

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