Research & Development
EMA grants orphan drug designation to Moleculin's Annamycin for AML
18 April 2024 -

Clinical stage pharmaceutical company Moleculin Biotech Inc (Nasdaq:MBRX) announced on Thursday that Annamycin, its next-generation anthracycline, has received orphan drug designation from the European Medicines Agency (EMA) for the treatment of Acute Myeloid Leukaemia (AML).

Annamycin's non-cardiotoxic profile, validated in multiple US and European studies, underscores its potential in AML therapy. Clinical data from a European trial demonstrated a notable 60% preliminary complete response rate in second-line AML subjects, with overall interim rates reaching 39%. The drug's durability in responses is developing, with one subject achieving a durable complete response beyond one year.

The EMA's orphan drug designation for Annamycin acknowledges its potential to address rare, life-threatening conditions like AML. Benefits include reduced regulatory fees, clinical support, research grants and up to 10 years of market exclusivity in the European Union upon approval.

Annamycin currently has fast track status and orphan drug designation from the US Food and Drug Administration (FDA) for the treatment of relapsed or refractory AML, in addition to orphan drug designation for the treatment of STS lung mets.