Amyotrophic Lateral Sclerosis: Strategies for Drug Developers

Published by: Decision Resources

Published: May. 19, 2008 - 49 Pages

Table of Contents

Executive Summary

Strategic Considerations

Stakeholder Implications

Introduction

Overview of ALS

The Nervous System

Familial vs. Sporadic ALS

Symptoms, Diagnosis, and Disease Progression

Epidemiology

Current Treatments

Sanofi -Aventis’s Rilutek

Symptomatic Treatments

Anti-Drooling Medications

Anti-Anxiety Drugs and Antidepressants

Muscle and Neuropathic Pain Medication

Insomnia Medication

Emerging Therapies

Antiglutamatergic Agents

Roche’s Rocephin

Teva’s Copaxone

AMPA Receptor Antagonists

Potential of Antiglutamatergic Agents in ALS

Induction of Heat Shock Proteins

CytRx’s Arimoclomol

Potential of Heat Shock Proteins in ALS

Antioxidants

Co-enzyme Q-10

Aeolus Pharmaceuticals’ AEOL-10150

Potential of Antioxidants in ALS

Neurotrophic Factors

Insulin-Like Growth Factor-1

Potential of Neurotrophic Factors in ALS

Lithium

Emerging Trends in ALS Treatment

Multidisciplinary Clinics

Noninvasive Positive Pressure Ventilation

Stem Cell Therapy

Outlook for ALS: Considerations for Drug Developers

Appendix A. Incentive Programs for the Research, Development, and Marketing of Orphan Drugs

FDA

Expanded Market Exclusivity

Tax Credits

Support During the Drug Approval Process

Grants

EMEA

Private Organization Incentives

Appendix B. Epidemiology Methods

Appendix C. Bibliography

Appendix D. Thought Leaders Interviewed for This Report—Amyotrophic Laterial Sclerosis

Tables

ES1. Thought Leaders’ Opinions of ALS Issues

1. Proposed Theories for the Cause of Sporadic ALS

2. The World Federation of Neurology Research Committee on Motor Neuron Diseases Guidelines for ALS Diagnosis

3. Commonly Used Drugs to Treat ALS Patients

4. Current Clinical Trials of Emerging Therapies to Treat ALS Patients

B1. Number of Diagnosed Prevalent Cases of Amyotrophic Lateral Sclerosis in the United States, 2007-2017

B2. Key Sources for Epidemiology Estimates, Amyotrophic Lateral Sclerosis

Figures

1. The Nervous System

2. Diagram of a Neuron

3. Age and Gender Distribution of Diagnosed ALS Prevalent Cases in the United States, 2007

4. Glutamate Excitotoxicity

5. Number of Emerging Therapies, By Mechanism of Action, for Amyotrophic Lateral Sclerosis

6. Multidisciplinary Approach to ALS Treatment

7. Worldwide Sales and Sales Growth of Rilutek, 1995-2007

A1. Number of Drugs Receiving FDA Orphan-Drug Designation and Approval, 1983-2007

A2. Growth in the Number of Orphan Drugs Available, 1983-2007

Abstract

Only one drug is approved for the treatment of ALS, and its only benefit is a modest increase in survival time. Commercial development in this disease has been minimal, with companies being dissuaded by the limited understanding of the mechanisms of the disease and by the small patient population. In addition, thought leaders interviewed by Decision Resources caution that recent failures of promising agents have re-emphasized the need for large trials to provide sufficient efficacy and safety data for emerging ALS treatments, and those requirements pose financial and operational challenges for companies choosing to enter this market. Nonetheless, the high unmet need in the treatment of ALS, plus the disease’s orphan status—which provides numerous financial, marketing, and drug-approval benefi ts—signals substantial opportunity for a company that develops an efficacious agent to treat this disease.

Get the Answers You Need to Shape Your Strategy

• ALS is a debilitating and fatal disease with limited treatment options. What are the current treatment options for ALS? What considerations drive neurologists’ prescribing choices? What do thought leaders identify as the primary goals of ALS treatment?
• A number of drugs, with a variety of mechanisms of action, are being investigated for efficacy in ALS. What new agents are in development? What drugs that are approved for other indications are being studied for use in ALS? What are thought leaders’ opinions of the potential of these agents in the treatment of ALS?
• The antibiotic minocycline, which showed promise in small-scale trials, was shown in a large clinical trial to be detrimental to ALS patients. What does the failure of minocycline mean for other emerging agents? What data do neurologists require before they will prescribe an emerging agent? What level of clinical data is necessary for an emerging agent, and what should companies expect when designing a clinical trial in ALS patients?
• The Orphan Drug Act of 1983 paved the way for the development and approval of numerous orphan drugs. How does the U.S. government encourage the pharmaceutical industry to investigate treatments that qualify for orphan status? What other organizations are willing to provide financial incentives to drug companies?

Scope

• Thought-leader opinions: Neurological experts give their opinions on the status of ALS treatment and explain their expectations for the potential of emerging treatments for the disease.
• Overview of ALS: The nervous system; familial vs. sporadic ALS; symptoms and disease progression. Epidemiology: Prevalent cases over a ten-year forecast period; age and gender trends.
• Current therapies: Rilutek; anti-drooling medications; anti-anxiety drugs and antidepressants; pain medication; insomnia medications.
• Emerging therapies: antiglutamatergic agents; induction of heat shock proteins; antioxidants; neurotrophic factors.
• Emerging trends in ALS treatments: multidisciplinary clinics; noninvasive positive pressure ventilation; stem cell therapy.
• Considerations for drug developers: the success of Rilutek; advantages of developing drugs for ALS; challenges in designing and running clinical trials.
• Benefits of orphan-drug status: Financial and marketing incentives associated with orphan-drug status.
• Orphan Disease Series: As competition increases in larger disease states, companies are turning to orphan diseases that offer substantial pricing advantages and lower regulatory hurdles because of high unmet need. The Orphan Disease Series provides critical market information, including disease prevalence, profiles of marketed and emerging agents, and insight into the prescribing choices of disease specialists for diseases with smaller patient populations. These reports will help you to maximize the potential of your agents in these challenging markets, including the regulatory benefits available and the potential for partnership with charitable organizations.

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