TMC has special experience in developing orphan products

Orphan product development

Orphan drug designations (ODDs)
Full collation and presentation of the data required to obtain an orphan designation, especially for the challenging issue of the prevalence of rare diseases in Europe.

TMC has written and submitted numerous successful ODD applications.

• Appearance at EMEA oral explanations during ODD assessments
• Liaison with rare disease experts and patient organisations
• Clinical trials
• Pre-approval requirements for compassionate/named patient use sales
• Marketing strategy
• Conversion of US orphan NDAs into MAAs for European approval

The many orphan disease areas in which we have worked include:
• Rare oncology indications such as lymphomas, CTCL, PTCL and BCL
• Urea cycle disorders
• Cystinosis
• Cystic fibrosis
• Duchenne's muscular dystrophy
• Porphyria
• Alpha-1 antitrypsin deficiency
• Pompe Disease
• Stargardt Disease
• Pancreatic cancer
• Epidermolysis bullosa