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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 process in the US

The accelerated approval of Pepaxto (melphalan flufenamid) in combination with dexamethasone in triple-class refractory multiple myeloma in February 2021, is a major step to establishing melphalan flufenamid as a treatment option for heavily treated patients whose therapeutic alternatives are exhausted. The accelerated approval in the U.S. must be confirmed with clinical data from a randomized study.

The OCEAN-study is planned to serve as confirmatory study. Following the presentation of Overall Survival data from the study, the FDA has issued a safety alert, pending further analysis. For more information, click here.

EU strategy

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. Pending a positive validation from EMA, 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.