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MISSION STATEMENT

To improve the lives of patients suffering from rare genetic diseases (“RGD or Orphan Diseases”), and the lives of their families, friends and colleagues. Most patients suffering from Orphan Diseases typically have few, if any, treatment options. Our mission is to bring new biotherapeutics that can improve the lives of these patients via a robust research and development program.

OUR SCIENCE

 

ERAD Therapeutics Inc.

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INNOVATIVE SCIENCE

Delivering Protein Therapeutics to the CNS

NEUROMUSCULAR DISEASE

Neuromuscular diseases are a large group of disorders that primarily disrupt the normal functioning of muscles and the nerves that control them. This leads to muscle weakness, impaired movement, and often progressive disability.

Examples of neuromuscular diseases are:

  • Muscular Dystrophy including Duchenne, Becker and many more;
  • Charcot- Marie-Tooth Disease and other neuropathies;
  • Myesthenia-gravis and more

GAUCHER DISEASE

Gaucher Disease is the most common Lysosomal Storage Disease, yet it is still considered an Orphan Disease. Currently affecting over 8,000 American’s, it is a genetic disorder resulting in an enzyme deficiency. Type 1 disease usually results in an enlarged liver, enlarged spleen, anemia and bone disease. Without therapy this significantly impacts both the quality and duration of life. Type 3 disease impacts all of the above as well as causing progressive and ultimately fatal neurological deterioration.

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An Orphan Drug receives an Orphan Drug Designation from the US FDA and the European Medical Agency. In the US, rare genetic diseases such as Tay-Sachs and Gaucher Disease are designated as Orphan Diseases because they have fewer than 200,000 patients in the US. The FDA created the Orphan Drug act in order to compel the pharmaceutical and biotechnology industries to develop drugs for small patient groups. Orphan Diseases and Orphan Drugs can be attributed to as few as 25 or 50 patients.

Orphan drugs are often brought to market quickly, through addressing small, targeted, often ignored patient populations, thus increasing the potential for strategic partnerships  with Top Tier Pharmaceutical Companies

Key Advantages:

  • Shorter timeline to market
    • Requires significantly less resources
    • Dramatically smaller clinical trials
    • <100 patients for gaucher disease phase iii clinical trials
    • Tax credits
    • Phase III dramatically cheaper than non-orphan drugs
    • Waived FDA fees and protocol assistance
  • FDA is often more flexible with approvals, resulting in shorter timeline to market
  • Marketing exclusivity (7 years from approval in the U.S.)
  • Lower marketing costs as patients are easily located
  • Faster uptake and market acceptance
  • Generally well reimbursed

 

Top Tier Pharmaceutical Companiese are extremely active in the orphan drug space:

  • 40% of acquired biotechs between 2008-2012 had an orphan drug in development
  • Novartis, GSK, Roche and Pfizer are largest orphan drug companies
  • Pfizer, Gilead, Roche, Shire, BMY and Celgene are leading orphan drug acquirer
  • 50% of top 20 orphan drugs were either acquired or in-licensed by Top Tier Pharmaceutical Companies

Key Market Statistics:

  • Worldwide orphan drug sales forecast to grow to $178 billion by 2020 with a CAGR of +11.7% (2015 to 2020); almost double overall prescription market growth
  • Orphan drugs set to be 20% of worldwide prescription sales by 2020 (excluding generics)

The Orphan Drug Act (ODA), a federal law established in 1983, aims to encourage pharmaceutical companies to develop drugs for rare diseases with small markets

  • Focused on rare diseases affecting less than 200,000 patients in the U.S.
  • Over 55 million people are estimated to suffer from a rare disease in the U.S. and Europe
  • Global estimates are between 5,000 and 7,000 rare diseases
  • ODA provides multiple financial incentives for orphan drug development
  • Although the patient population is small orphan drugs have a higher per patient cost, acting as an incentive to develop drugs for orphan diseases
  • Since 1983, more than 400 orphan products have received U.S. market authorization
  • Japan and Europe have enacted similar legislation