Date: 2016-08-23
Type of information: Granting of the Breakthrough Therapy status
Product name: SL-401 - recombinant human interleukin-3 truncated diphtheria toxin fusion protein
Compound: recombinant human interleukin-3 truncated diphtheria toxin fusion protein
Therapeutic area: Cancer - Oncology - Rare diseases
Action mechanism: fusion protein. SL-401 is a targeted therapy directed to the interleukin-3 receptor (IL-3R) present on CSCs and tumor bulk of a wide range of hematologic cancers. Three multicenter clinical trials with SL-401 are currently open in seven indications. SL-401 is currently completing the lead-in stage of the pivotal trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN). Clinical studies with SL-401 are also open in additional hematologic indications including acute myeloid leukemia (AML) in first complete remission (CR) with minimal residual disease (MRD), relapsed/refractory AML, and four types of advanced high-risk myeloproliferative neoplasms (MPN), including systemic mastocytosis, advanced symptomatic hypereosinophilic disorder, myelofibrosis, and chronic myelomonocytic leukemia. Previously, SL-401 demonstrated single-agent activity, including multiple durable CRs, in a Phase 1/2 trial in BPDCN and relapsed/refractory AML.
Company: Stemline Therapeutics (USA - NY)
Disease: blastic plasmacytoid dendritic cell neoplasm
Latest news: * On August 23, 2016, Stemline Therapeutics announced that the FDA has granted Breakthrough Therapy Designation to SL-401,a targeted therapy directed to the interleukin-3 receptor (CD123), for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). This Breakthrough Designation request was supported by efficacy and safety data from the Phase 2 trial evaluating SL-401in blastic plasmacytoid dendritic cell neoplasm patients in both the first-line and relapsed/refractory settings.
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