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Regulatory strategy

The regulatory strategy provides information on the submission for a potential marketing authorization in Europe.

EU submission

We submitted an application to the European Medicines Agency, EMA, in April 2021, for conditional marketing authorization of melflufen (melphalan flufenamide) in the EU, based on the pivotal phase 2 HORIZON study in relapsed refractory multiple myeloma. Melflufen will be subject to a regulatory assessment according to the standard timelines.

Orphan designation

Melflufen has been granted orphan designation in both the US and Europe, which may provide benefits such as market exclusivity for seven and ten years respectively. Orphan designation is granted to treatments that target rare diseases where it´s unlikely that the costs of developing such treatments would be recovered through sales.