After two and a half years and four tries, uniQure B.V.'s gene therapy Glybera alipogene tiparvovec finally won the backing of the European Medicines Agency's CHMP. Approval by the European Commission would make Glybera the first gene therapy to win approval outside China, and complete the story of management's campaign to remind policy makers about Europe's commitment to rare diseases.

The tale also provides a window into the inner workings of EMA and its scientific committees.