Fourth-Quarter and Full-Year 2019 Orphan and Rheumatology Segment Results
U.S.
Indication
Market
Growth
Strategy
HORIZON
Durable Orphan Franchise
RAVICTI®
(glycerol phenylbutyrate) Oral Liquid
Urea cycle disorders (UCDs)
UCDs are rare and life-threatening genetic
diseases resulting in the body's inability to
remove ammonia from the blood stream(1)
UCDs cause hyperammonia that can lead to
intellectual disability, seizures, coma or
death(2)(3)
~2,600 people with UCDS
~1,000 diagnosed population (4)
Conversion from older-generation nitrogen-
scavengers to RAVICTI
Increase awareness of label expansion to position
RAVICTI as first-line therapy
Increase awareness and diagnosis of UCDS
°
PROCYSBI®
(cysteamine bitartrate)
delayed-release capsules
Nephropathic cystinosis (NC)
NC is a rare and life-threatening, progressive,
multisystem metabolic disorder (1)
Without cysteamine-depleting treatment,
high intracellular cystine concentrations can
occur in virtually all organs and tissues,
leading to irreversible cellular damage,
progressive multi-organ failure and death
~500-600 diagnosed patients
~400-450 diagnosed patients on
cystine-depleting therapy (4)
Conversion from older-generation cysteamine-
depleting therapy to PROCYSBI
Increase awareness of label expansion to position
PROCYSBI as first-line therapy
Increase persistence of and adherence to
treatment
ACTIMMUNE
(Interferon gamma-1b)
Ⓡ
Chronic granulomatous disease (CGD)
CGD is a life-threatening inherited primary
immunodeficiency disease that limits the
body's ability to fight off certain pathogens (1)
Patients have increased susceptibility to
severe and recurrent bacterial and fungal
infections, along with the formation and
development of granulomas in most organs
~1,600 people with CGD(4)
Increase awareness and diagnosis of CGD
Drive adoption of "triple prophylaxis" therapy -
immunomodulation (IFNg) + antifungal + antibiotic
Increase persistence of and adherence to
treatment
(1) See full prescribing information at www.RAVICTI.com, www.PROCYSBI.com and www.ACTIMMUNE.com. Information found on or accessible through these websites is not a part of or
incorporated by reference in this presentation.
(2) Summar, 2001. (3) Haeberle, 2019. (4) Horizon estimate.
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