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Delivering Protein Therapeutics to the CNS

ERAD Therapeutics Inc. is focusing on the development for therapeutics for Orphan Diseases, based on the discovery at the Hospital for Sick Children in Toronto, Canada that certain bacterial proteins could be exploited to rescue mis-folded but functional proteins and help restore normal cellular function in diseases such as Gaucher Disease and Cystic Fibrosis. We continue to strengthen our platform technology to bring new therapeutic inventions and hope to patients with these (and other) rare genetic diseases.


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


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

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)