IPTACOPAN ASH UPDATE
Opportunity to redefine PNH treatment paradigm
~6k
Prevalent¹
PNH patients in US
30%
~400
Incident²
PNH patients/year in US
Treated with
complement
inhibitor³
Untreated
70%
•
■
■ Current market -USD 2bn WW (USD 1bn US)4
■ Of C5-treated patients, -80% have Hb < 12g/dL5
Still experiencing symptoms
-
- Managing life around infusion schedule
Some still receiving transfusions
Varying views of when treatment should be started
Heterogeneous presentation of symptoms
Some unwilling to commit to regular infusions
Some still receiving transfusions
Displace
Anti-C5
Potentially
increase
treatment rate
Start appropriate
patients on
iptacopan
1. Prevalence: 12-18 per million individuals in the US (Jalbert JJ, 2019, Mon Pere N, 2018). 2. Incidence: 1.0-1.5 per million individuals (Hill A, 2017). 3. Treated with anti-C5 or anti-C3 4. Based on C5i therapies only
5. Debureaux PE et al. Bone Marrow Transplant 2021;56:2600-2 Source: Patient journey market research 2022
27
IPTACOPAN ASH UPDATE | DECEMBER 13, 2022
NOVARTIS | Reimagining MedicineView entire presentation