Corporate Presentation
Rare diseases
CHI
Opportunity to treat up to 800 patients with diffuse
CHI at ultra-rare disease price levels in the US
Patients eligible for dasiglucagon
treatment in the US
ZEAL&
ZEALAND PHARMA
Ultra-rare disease therapy analogues with clear
clinical value command premium prices in US5
Annual treatment cost (k$)
700-800
Diffuse CHI patients
1,500
•
~60% of CHI patients are diagnosed with diffuse CHI1
~45-70 newly diagnosed diffuse CHI patients/year 2,3,4
1,250
Naglazyme
All diffuse CHI patients considered eligible for
dasiglucagon treatment4
Kanuma
1,000
Procysbi
Majority of patients are treated and closely followed by
pediatric endocrinologists in few Centers of Excellence4
750
• Elaprase
Focal CHI patients
500
400-500
Most focal patients are cured after pancreatic
surgery, while few patients will require continued
short- or longer-term medical treatment
Aldurazyme
Ultomiris
250
# of CHI patients
0
500
Diffuse CHI
Focal CHI
1Arya et al. Plos One 2014;9:e98054; 2Arnoux JB et al. 2011 Orphanet J Rare Dis;6:63; 3Yau et al. Plos One 2020;15(2):4 Based on KOL interviews (2022); 5 Zealand Pharma Payer & Pricing Research,
December 2022
Indications by product: Procysbi (nephropathic cystinosis); Naglazyme (Maratolamy syndrome); Ultomoris (atypical hemolytic uremic syndrome); Kanuma (lysosomal acid lipase deficiency); Luxturna
(biallelic RPE65 mutation-associated retinal dystrophy); Elaprase (Hunter syndrome); Aldurazyme (Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I).
Luxturna
1,000
1,500
Patient population
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