Orphan product development activities
- Orphan drug designations (ODDs) : the TMC team has written and submitted more than 150 successful ODD applications including submissions to the FDA, the EMA, Australian and Japanese authorities. We compile the applications and when required we attend EMEA oral explanations during ODD assessments
- Liaison with rare disease experts and patient organisations
- Full service management of clinical trials
- Advising on Pre-approval requirements for compassionate/named patient use sales
- Planning Marketing strategy
The many orphan disease areas in which we have worked include
- Rare oncology indications such as mesothelioma, lymphomas, CTCL, PTCL and BCL
- Urea cycle disorders
- Cystinosis
- Cystic fibrosis
- Duchenne's muscular dystrophy
- Porphyria
- Alpha-1 antitrypsin deficiency
- Pompe Disease
- Stargardt’s Disease
- Pancreatic cancer
- Epidermolysis bullosa