Pirfenidone post-authorization safety registry (PASSPORT) update

Background: PASSPORT is a post-authorization safety registry for pirfenidone to collect “real-world” data in EU patients with idiopathic pulmonary fibrosis (IPF).

Objectives: 1) Assess the safety of pirfenidone as monotherapy and with N-acetylcysteine (NAC) and/or corticosteroids (CS); 2) examine country-specific differences.

Methods: 109 EU sites dosed 1009 patients; largest enrolling countries were Germany (N = 452), France (N = 214) and UK (N = 184). Safety data were recorded at routine clinic visits for up to 2yrs. Adverse drug reactions (ADR: noxious, unintended drug response) were collected.

Results: At baseline, mean ± SD age was 70 ± 8.5 yr and time since IPF diagnosis 1.6 ± 2.5 yr; 80% were male; supplemental O₂ was used by 27%; mean ± SD FVC was 2.56 ± 0.78 L; mean ± SD % predicted FVC was 66 ± 16% (15% had % FVC< 50%). Most common comorbidities (> 10%) were hypertension, GERD, hypercholesterolemia and coronary artery disease.

At this interim analysis, median time on pirfenidone was 12 mo; total exposure was 1027 PY. 70% of patients received pirfenidone alone; 12%, 9% and 9% also received NAC, CS, and NAC+CS, respectively. ADR incidence was generally consistent for these subgroups except weight decrease which occurred more often in the pirfenidone+CS group (21.1% vs. 9.8%-11.8%). 73% had ≥1 ADR; most common were nausea, 19%; fatigue, 18%; decreased appetite, 15%; decreased weight, 15%; rash, 12%; diarrhea, 10%.

Patient characteristics and ADRs were similar in the 3 largest enrolling countries.

Conclusion: In this real-world setting, pirfenidone was generally safe and well tolerated as monotherapy or combined with NAC and/or CS. Characteristics and safety profile of German, French and UK patients were similar.