BLINCYTO (blinatumomab) - Lymphoblastic leukaemia (ALL) in paediatric

The clinical benefit of BLINCYTO (blinatumomab) 38.5 micrograms powder for concentrate and solution for solution for infusion is substantial in the indication “as monotherapy for the treatment of paediatric patients aged from 1 month to 1 year with high-risk first relapsed Philadelphia chromosome negative CD19 positive B-cell precursor ALL as part of the consolidation therapy”.

Considering: 

• the results obtained in paediatric patients aged 1 year and older, with:
  • evidence of a superiority of blinatumomab compared to conventional chemotherapy,
  • the absence of evidence of an improvement in overall survival versus chemotherapy, an exploratory secondary endpoint,
  • the favourable safety profile of blinatumomab, marked by a lower haematotoxicity compared to chemotherapy, despite more frequent general and neurological disorders,
  • the expected improvement in care conditions compared to chemotherapy given the possibil ity of administering blinatumomab to patients mainly at home;
• pharmacokinetic data indicating similar exposure to that observed in paediatric patients aged from 1 to under 18 years and in adults with ALL, for the dose of 15 micrograms/m²/day;
• the results of the study by van der Sluis et al. suggesting an efficacy of blinatumomab in infants with newly diagnosed B-cell precursor ALL and with a KMT2A rearrangement;
• the partially met need at this stage of the disease; 
• and despite: 
• the absence of efficacy, safety and quality of life data in the context of consolidation therapy in infants aged from 1 month to 1 year with high-risk first relapsed B-cell precursor ALL;
• uncertainties related to extrapolation of the results from studies conducted in patients aged 1 year or older;
•  uncertainties related to transposition of the data obtained in infants aged from 1 month to 1 year, with newly diagnosed B-cell precursor ALL, 

The Committee considers that BLINCYTO (blinatumomab) 38.5 micrograms powder for concentrate and solution for solution for infusion provides a moderate clinical added value (CAV III) compared to conventional chemotherapy in the treatment of paediatric patients aged from 1 month to 1 year with high-risk first relapsed Philadelphia chromosome-negative CD19-positive B-cell precursor ALL as part of the third block of consolidation therapy.