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Regulatory strategy

The regulatory strategy provides clear guidance on potential submissions for marketing authorization, applications for broadening of indications, or abilities to enter in new geographies.

Regulatory submission in the US

The FDA submission of a New Drug Application for accelerated approval of melflufen in combination with dexamethasone in triple-class refractory multiple myeloma, is major step to establish melflufen as a potential treatment option for heavily treated patients who often lack other alternatives. An eventual accelerated approval in the U.S. needs to be confirmed with clinical data from a randomized study.

The ongoing OCEAN study is planned to serve as confirmatory study.

EU strategy

We have informed the European Medicines Agency about our intention to apply for a conditional marketing authorization of melflufen in the EU.

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.