Erdnussprotein als entfettetes Pulver von Arachis hypogaea L., semen (Erdnüsse) (2) – Palforzia®

• Clinical trials
  • The benefit assessment is based on the randomised, double-blind ARC005 trial comparing peanut protein in the form of defatted powder from Arachis hypogaea L., semen (peanuts) (hereinafter: peanut protein) with a placebo.

Children aged 1 to 3 years with a confirmed diagnosis of peanut allergy

• In summary, for children aged 1 to 3 years with a confirmed diagnosis of peanut allergy, a non-quantifiable additional benefit is identified compared with the appropriate comparator therapy of watchful waiting.
• Overall, therefore, the G-BA derives a hint of the established additional benefit from the strength of the evidence.
• mortality
  • No deaths occurred in the ARC005 study.
• Morbidity – Allergic reactions following accidental exposure to peanuts
  • For the endpoint of allergic reactions following accidental exposure to peanuts, there is no statistically significant difference between the treatment groups.
  • In the ARC005 study, the children were required to follow a peanut-free diet throughout the entire study period. In the event of accidental peanut exposure during the study, parents or carers were instructed to contact the study centre within 24 hours, regardless of whether an adverse reaction had occurred.
• Morbidity – freedom from symptoms at all tested doses (up to 2000 mg) in the exit DBPCFC (double-blind, placebo-controlled food challenge)
  • For the endpoint ‘absence of symptoms at all tested doses (up to 2000 mg) in the exit DBPCFC’, there was a statistically significant advantage of peanut protein compared with a watch-and-wait approach.
  • The DBPCFC is a double-blind, placebo-controlled food challenge and comprises two challenge tests: on one day the food to be tested is administered, and on another day a placebo preparation is used.
  • Successful completion of a medically supervised food challenge is described in the literature as a surrogate marker for the efficacy of desensitisation.
• Morbidity – Maximum symptom severity across all tested doses (up to 2000 mg) in the exit DBPCFC
  • For the endpoint ‘Maximum symptom severity at all tested doses (up to 2000 mg) in the exit DBPCFC’, there was no statistically significant difference between the treatment groups for the severity level ‘severe’ (grade ≥ 3).
  • The severity grading for the endpoints ‘maximum symptom severity’ and ‘absence of symptoms’ at all tested doses (up to 2000 mg) was carried out in accordance with CoFAR (Consortium for Food Allergy) and thus according to a different classification system to that used for assessing tolerance to a particular dose in the DBPCFC.
• quality of life
  • No data are available for the category of health-related quality of life.
• Side effects – severe AEs, serious adverse events (SAEs)
  • For the endpoints of severe AEs and SUEs, there were no statistically significant differences between the treatment groups.
• Side effects – discontinuation due to side effects
  • For the endpoint ‘discontinuation due to adverse events’, there is no statistically significant difference between the treatment groups.
• Side effects – Specific side effects
  • For the endpoint ‘systemic allergic reactions’ (adverse events), there was no statistically significant difference between the treatment groups. No severe systemic allergic reactions (severe adverse events) occurred in the ARC005 study.
  • For the endpoint of gastrointestinal disorders, a statistically significant difference was observed to the disadvantage of AR101 compared with a ‘wait-and-see’ approach.
• Overall view
  • No events occurred in the mortality category during the study.
  • In the morbidity endpoint category, a statistically significant advantage was observed for treatment with peanut protein compared with a watch-and-wait approach for the endpoint ‘symptom-free at all tested doses’, as assessed during the DBPCFC exit challenge. No statistically significant difference was observed in the patient-relevant endpoint ‘allergic reactions following accidental exposure to peanuts’ – which is independent of the challenge test – or in the endpoint ‘maximum symptom severity at all tested doses’.
  • No data are available for the category of health-related quality of life.
  • In the category of side effects, there were no relevant statistically significant advantages or disadvantages overall.
  • Overall, treatment with peanut protein showed an advantage under study conditions in the endpoint for symptom-free status during provocation testing; however, this could not be confirmed in patient-relevant endpoints outside the context of provocation testing. The extent to which the advantage observed in provocation testing translates into a reduction in allergic reactions following accidental exposure, and in general, remains unclear at present. The extent of the advantage demonstrated during provocation testing is therefore classified as non-quantifiable.

Courtesy translation only, please refer to the German original.