DSP-7888 Granted Orphan Drug Status for Myelodysplastic Syndrome
The US Food and Drug Administration granted orphan drug designation to DSP-7888 for myelodysplastic syndrome.
Boston Biomedical, Inc announced today that the US Food and Drug Administration (FDA) granted orphan drug designation to DSP-7888 for the treatment of myelodysplastic syndrome (MDS), a group of hematologic cancers characterized by the production of poorly formed blood cells or blood cell dysfunction.1
MDS is diagnosed in approximately 10,000 to 15,000 patients yearly in the US, and about a third of these patients progress to acute myeloid leukemia.
DSP-7888, an investigational cancer peptide vaccine, induces the Wilms' tumor gene 1 (WT1)-specific cytotoxic T-lymphocytes (CTL) and helper T cells, which attack cancerous cells in various solid and hematologic cancers expressing WT1.
A phase 1/2 trial that enrolled patients with MDS who progressed on or after receiving first line azacitidine therapy demonstrated that patients not only tolerated DSP-7888 well, but also showed early signs of potential clinical efficacy.
DSP-7888 is being evaluated in 3 phase 1/2 monotherapy trials; a phase 1 trial for advanced malignancies (ClinicalTrials.gov Identifier: NCT02498665), a phase 1/2 study for pediatric patients with relapsed or refractory high-grade glioma (ClinicalTrials.gov Identifier: NCT02750891), and a phase 1/2 trial for myelodysplastic syndrome (ClinicalTrials.gov Identifier: NCT02436252).
- Boston Biomedical announces orphan drug designation by FDA for investigational WT1 cancer peptide vaccine DSP-7888 in myelodysplastic syndrome [news release]. Cambridge, MA: PR Newswire; July 10, 2017. http://www.prnewswire.com/news-releases/boston-biomedical-announces-orphan-drug-designation-by-fda-for-investigational-wt1-cancer-peptide-vaccine-dsp-7888-in-myelodysplastic-syndrome-300484721.html. Accessed July 10, 2017.