Qarziba(Dinutuximab beta)

Qarziba (Dinutuximab beta) is a drug that received a positive opinion for marketing authorisation on 23 March 2017, following its orphan medicinal product designation on 8 November 2012. It is approved for patients 12 months and above. The marketing authorisation for Unituxin, a related product, was granted on 14 August 2015 but subsequently withdrawn on 22 March 2017.

In efficacy studies, Qarziba demonstrated an overall response rate (CR+PR) of 31%. For patients with relapsed/refractory (R/R) disease, the overall response rate (CR+PR) was 28%. The median duration of response was 313 days. The 1-year overall survival (OS) KM estimate for APN311 + IL-2 + 13-cis RA was 0.90, compared to 0.41 for the control group, with an OS difference p-value of 0.0009. The 2-year OS rate was 69% and 70% in two separate findings, with 1-year OS rates of 90% and 93% respectively.

Regarding safety, 39% of patients received at least 50% of planned ch14.18/CHO doses with IL-2, while 78% received at least 50% of planned ch14.18/CHO doses without IL-2. There were 70 IL-2 dose reductions and 29 IL-2 discontinuations. For ch14.18/CHO, there were 64 dose reductions and 34 discontinuations when administered with IL-2. The mean cumulative ch14.18 dose was 234 mg with IL-2 and 289 mg when ch14.18 was administered alone.