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Unlocking the potential of advanced therapies developed for rare diseases

How to leverage regulatory strategies and interactions with the EU EMA and the U.S. FDA to accelerate development

Currently, no treatments exist for 95% of rare diseases, representing a significant unmet medical need in this patient population. Most orphan diseases have a genetic origin – some are driven by inherited genetic defects and others driven by de novo mutations – making these diseases an exciting target for advanced therapies.

The development of advanced therapies has grown tremendously in recent years and is poised to continue. These products are expected to offer treatments for currently untreatable diseases, as well as deliver more effective treatment options for diseases with existing therapeutics. For rare (orphan) diseases in particular, advanced therapies present unique and challenging opportunities.

This whitepaper outlines:

  • Common hurdles of advanced therapies for orphan diseases
  • A regulatory strategy and interaction plan for sponsors developing cell and gene therapy products for rare diseases
  • Considerations for abbreviated development in rare disease advanced therapies

Whitepaper