Dr Carlos R. Camozzi

Orphan Drugs Development and Rare Diseases Expert

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Expert in rare diseases and orphan drugs, capable to evaluate the regulatory and financial feasibility and sustainability of the most challenging research and development programmes. Moved by the ultimate objective to bring to all patients’ access to innovative, advanced, safe and effective therapeutic options in the shortest possible time and the most efficient R&D pathways.  Physician by training, with specialisations in Paediatrics, Clinical Pharmacology and Neuropsychiatry, with more than 25 years international experience in the biopharma industry, reviewed, revised and generated outstanding solutions to the challenging designs and execution of more than 12 oncology, 8 haematology, 31 paediatric and 32 orphan drugs clinical trials. Successfully led several regulatory interactions, consultations, orphan drug designations (ODD), paediatric investigational plans (PIP) and marketing authorisations (MA) with both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA); with outstanding achievements like (among others) the FDA approval of Carbaglu in NAGS deficiency and the first ever gene-therapy (Glybera) approval in the western world (EMA). Actively participated in several M&A, due diligences, fundraising and financial rounds (Series A, B and C), and collaborated with worldwide investors to review, analyse and define the investment attractiveness of the Orphan Drugs development strategies of innovative R&D companies.