Q4 2021 Results
Participants
Company overview
Pharmaceuticals
Oncology
Financial review
Conclusion
Appendix
ZolgensmaⓇ grows +46% in 2021 to USD 1.4bn
Due to geographic expansion as the foundational therapy for SMA
Sales evolution
USD m, % cc
Ex-US
US
FY 920m
FY 1.4bn +46%
375
342
319
315
291
254
249
205
224
200
169
209
170
148
44
100
126
122
105
106
119
126
106
118
Q1
Q2
Q3
Q4
Q1
Q2
Q3
Q4
2020
2021
Q4 highlights
CE
zolgensma
K &K
L
■ Driven by expanding access ex-US, +58% sales
■
Over 1800 patients treated worldwide¹
Approval now in 42 countries; access pathways in 26 countries
Newborn screening reached ~85% in US, 20% in EU
Future outlook
Continued growth ex-US
■ Newborn screening: Goal of 38% in EU by YE22
■
US: Steady US sales driven by incident patients
Advancing robust data in SMA with IT²
▪ STEER: Ph3 currently initiating treatment-naive Type 2 patients
■ STRENGTH: Start H2 2022 in patients who have discontinued
treatment with nusinersen and/or risdiplam
1. Commercially, via managed access programs and in clinical trials 2. With investigational OAV101 intrathecal administration
28 Investor Relations | Q4 2021 Results
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