Intended for healthcare professionals

Letters Orphan drugs

BioMarin Europe replies

BMJ 2010; 341 doi: (Published 07 December 2010) Cite this as: BMJ 2010;341:c7006
  1. Adrian Quartel, medical director1,
  2. James Lennertz, vice president and general manager1
  1. 1BioMarin Europe, London WC2H 8HL, UK
  1. aquartel{at}

Among the inaccuracies in the open letter of Nicholl and colleagues,1 is the statement that the chemical 3,4-diaminopyridine base (3,4-DAP) has an excellent safety record. This cannot be substantiated by evidence since 3,4-DAP has not undergone systematic safety and toxicology testing nor does it have appropriate safety monitoring and pharmacovigilance systems in place to capture, analyse, and report such data. 3,4-DAP is not a generic drug but …

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