ADZYNMA (ADAMTS13r) - Congenital thrombotic thrombocytopenic purpura (cTTP)

The Committee deems that the clinical benefit of ADZYNMA 500 IU and 1,500 IU (rADAMTS13) is substantial in the MA indication.

Considering:

• comparative efficacy results versus the standard of care (plasma-based therapy) observed in the pivotal crossover study (281102):
  • no acute TTP episodes occurring during the periods of prophylactic treatment with rADAMTS13 versus one episode during the periods of prophylactic treatment with the standard of care (plasma-based therapy),
  • one confirmed episode, treated and resolved by rADAMTS13 in 3 days versus 2 confirmed acute TTP episodes, treated and resolved by the standard of care (plasma-based therapy) in 14.8 days and 1.5 days,
  • the lower incidence of subacute episodes and isolated clinical manifestations with rADAMTS13 versus standard of care (plasma-based therapy),
• the known limitations of the current standard of care (plasma-based therapy) in terms of efficacy, safety, quality of life, socioeconomic impact and accessibility,
• the efficacy results observed in the extension study (3002), supporting those of the pivotal study, with a single confirmed acute TTP episode occurring in a patient in the prophylactic treatment cohort out of 65 patients included in the cohort,
• the more favourable safety profile of rADAMTS13 compared to plasma-based therapy,
• the purely descriptive nature of the results of the two interim analyses, with no formalised statistical hypothesis, limiting interpretation of the comparative results,
• the lack of robust data demonstrating an additional impact on quality of life and the organisation of care, although this impact is expected,
• the absence of data on morbidity and mortality in terms of the incidence of complications,
• uncertainties with respect to the long-term safety, particularly concerning the development of anti-rADAMTS13 neutralising antibodies,

the Committee deems that ADZYNMA 500 IU and 1,500 IU (rADAMTS13) provides a moderate clinical added value (CAV III) in the current care pathway for congenital thrombotic thrombocytopenic purpura (cTTP) due to ADAMTS13 deficiency, which includes relevant comparators (paragraph 5.2) as prophylactic treatment and on demand in the event of an acute cTTP episode.