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Kinesys’ Principals are experts at managing the designation, development and registration of Orphan Medicinal Products (OMPs). Here we set out some of the challenges and explain why we are ideally placed to help overcome them.

The Challenge

Special products need special attention from people who understand.Orphan medicinal products (OMPs) are niche therapies for patients with special needs. OMPs can be life-saving treatments but their development is often fraught with difficulty. Taking an OMP to market requires specific knowledge and expertise. Some key issues and challenges include:

  • Knowing when is the right time to file the application for orphan designation to access the benefits without unnecessarily divulging information to competitors.
  • Justifying the business decision to dedicate resources to development of the product
  • Accessing sufficient prevalence data and justifying medical plausibilityto support the OMP designation process
  • Understanding the different needs of new products for orphan indications and re-profiled marketed products for a new orphan indication
  • Specific problems in designing clinical trials and accessing an adequate patient population to support registration
  • Getting to market first to ensure market exclusivity and understanding the nuances of significant benefit, market exclusivity, patent life anddata protection

Projects Successfully Completed

  • A biological agent for pancreatic cancer
  • An anti-epileptic drug for Lennox-Gastaut syndrome
  • A topical agent for chemotherapy-induced toxicity
  • Two stem cell agents for anal fistulae
  • A hormonal product for high risk pregnancies
  • A re-profiled product for prevention of GVHD
  • A NCE for prevention of oral mucositis
  • A respiratory product for premature infants, two indications:
    • acute respiratory distress syndrome
    • meconium aspiration syndrome
  • Various oncology and haematology agents for indications, such as:
    • chronic lymphocytic leukemia
    • acute myeloid leukemia
    • brain metastases secondary to breast cancer
    • glioma