Regulatory support for a biotech’s EU orphan drug designation

A small Asia-based biotech planned to seek orphan drug designation (ODD) in the EU for a new combination medicinal product they were developing for a rare metabolic disease called Dercum’s disease (DD). The biotech asked ICON to manage the submission process as part of their strategic expansion to the EU. Obtaining ODD incentives in the EU was a critical part of the expansion plan, given the small size of the company and their lack of a presence in the EU. Their objective was to run a phase 3 study in the EU for their DD treatment. The disease is characterised by painful subcutaneous (SC) adipose tissue deposits throughout the body. The associated pain and discomfort can make it difficult for patients to perform routine tasks, work or engage in social activity, negatively impacting their quality of life. 

The sponsor had limited knowledge of EU regulatory legislation and no representative in the EU to act as a point of contact for the EMA ODD process. The biotech did not have company and drug profiles created in the European Medicines Agency IRIS portal. They sought ICON’s technical support for the IRIS submission and regulatory/medical support to write the ODD application plus provide details on the prevalence of DD in the EU. ICON advised about the treatment’s significant benefit, a criteria specific to EU ODD. However, with no EU company representation the sponsor would be unable to get an ODD under the biotech’s name. 

ICON Regulatory Consulting Services formed a multidisciplinary team of regulatory, clinical and epidemiology colleagues to support the ODD application. A regulatory project manager coordinated the work and communications between the team and sponsor. The team focussed on the justification of significant benefit and the target population. A medical writer composed the application, ensuring that feedback from multidisciplinary team and sponsor were captured in the final version. ICON assisted the client by creating a user profile and drug profile in IRIS and applying for the ODD on the company’s behalf to overcome their lack of an EU presence. The ODD could be transferred back to the sponsor at a later date once they established a presence in the EU. 

The EMA granted an ODD opinion on the sponsor’s drug as proposed in their submission. There were no questions from the EMA, resulting in a shorter timeline than anticipated. The EMA accepted the rationale recommended by ICON for the justification of the drug’s significant benefit. As a result of the successful EU ODD, and with ICON’s continued regulatory support, the biotech set up representation within the EU. The biotech can now benefit from the EU ODD incentives. The sponsor was happy with the positive outcome and ICON’s support in liaising with the EMA.