Regulatory Updates

The beginning of September 2019 has already seen two orphan drug designations (ODD) for drugs designed to treat multiple myeloma (MM).

CT053

On the 2^nd September, 2019, the United States (US) Food & Drug Administration (FDA) announced it was granting ODD to CT053. CT053 is a fully human, investigational, anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T-cell product for the treatment of relapsed/refractory MM (RRMM). The FDA had previously awarded Investigational New Drug (IND) clearance to CT053 in June 2019.^1,2

This award is based on results from a phase I clinical trial conducted in China. The results of this trial were presented at the 5^th Annual Immunotherapy In Myeloma Scientific Workshop in Denver, US. As of the 28^th February 2019, 24 patients with R/R MM were treated, with 70.8% of patients achieving a complete response (CR) and 87.5% achieving an overall response. No events of grade ≥3 cytokine release syndrome (CRS) were reported.^1,2

Isatuximab

The Korean Ministry of Food and Drug Safety has granted ODD to isatuximab (Isa) for use in combination with pomalidomide (P) and dexamethasone (d, Isa-Pd) in patients who have previously received two or more treatments for R/R MM. This approval is based on the results of the ICARIA-MM trial, presented at the American Society of Clinical Oncology Meeting (ASCO) in June, 2019.³

In the ICARIA-MM phase III study, patients with R/R MM were randomized to either Isa-Pd (n= 154) or Pd alone (n= 153). At a median follow-up of 11.6 months the progression-free survival, as determined by independent review committee assessment, was 11.53 months for patients receiving Isa-Pd compared to 6.47 months for those receiving Pd (P = 0.001). The hazard ratio was 0.596, indicating Isa-Pd significantly improves PFS. Median overall survival was not reached in either arm. The triplet also had a manageable safety profile.⁴