panzyga® for your patients with ITP

The efficacy and tolerability of panzyga^® in adults with primary chronic immune thrombocytopenia (ITP) were assessed in the NGAM-02 trial (NCT01349790).

Trial overview5

*Threshold platelet count <100 × 109/L; other causes of thrombocytopenia excluded through history, physical examination and blood test results.

†Safety, bleeding severity and response rates as defined by the European Medicines Agency guideline on the clinical investigation of human normal immunoglobulin for intravenous administration (IVIg) EMA/CHMP/BPWP/94033/2007 rev. 2) 2010: Alternative response (AR), increase in platelet count to ≥30 x 109/L and to ≥2x baseline platelet count, confirmed on ≥2 separate occasions at least 7 days apart, and absence of bleeding; Complete response (CR), increase in platelet count to ≥100 x 109/L, confirmed on ≥2 separate occasions at least 7 days apart, and absence of bleeding; Loss of AR/CR, AR/CR which subsequently deteriorated, to a platelet count of <30 x 109/L/100 x 109/L or to a level <2x the baseline count, or because of bleeding; No response, Platelet count of <30 x 109/L or <2x increase in baseline platelet count, confirmed on 2 separate occasions ~1 day apart, or the presence of bleeding.

Efficacy

Rapid and sustained high response rates5

Full analysis set (N=36)

Highly significant ~5-fold increase in platelet count was observed mostly within 24 h after the first dose (p<0.0001)5

Normal range platelet counts were observed within 3 days after the first dose⁵

Platelet count over time in the full analysis set (N =36).

Reduced severity of bleeding5

(Data for 2 patients missing at Day 8)

For almost 80% (18/23) of patients who had bleeding at baseline, the haemorrhages resolved completely by Day 85

Severity of bleeding was assessed by the investigator using a 6-point verbal rating scale: 1=none (no haemorrhage of any kind); 2=minor [few petechiae (≤100 total) and/or ≤5 small bruises (≤3 cm diameter) but no mucosal bleeding]; 3=mild [more than 100 petechiae and/or >5 large bruises (>3 cm diameter) but no mucosal bleeding]; 4=moderate (overt mucosal bleeding, such as epistaxis, gum bleeding, oropharyngeal blood blisters, menorrhagia or gastrointestinal bleeding, not requiring immediate medical intervention); 5=severe [mucosal bleeding or suspected internal haemorrhage (e.g. in the brain, lung, muscle or joint) requiring immediate medical intervention]; 6=life-threatening/fatal (documented intracranial haemorrhage or life-threatening or fatal haemorrhage at any site).5

Tolerability

Favourable tolerability profile5

Safety analysis set (N=40)

Laboratory tests and vital signs assessment demonstrated no significant safety concerns. In each of the haematology and chemistry parameters, changes to abnormal laboratory values considered clinically significant were reported in three and two patients, respectively. Laboratory abnormalities consistent with haemolysis were found in 6 of 40 patients (15.0%), and 1 patient had clinically evident haemolysis that was reported as a TEAE and considered to probably be related to the study drug. Fluctuations in vital signs were minor and within expected range for patients undergoing infusion. No changes in viral status were assessed during the study. Two patients (5.0%) died during the study. Both deaths were deemed not related to the study drug.5