Efficacy
Primary Efficacy Endpoint in AURORA and BEACON
Percent change from baseline in PPIX
Mostly commonly reported adverse event with btiopertin
dizziness
EPP is caused by loss of function in this enzyme
ferrochelatase
Bitopertin is indicated for the treatment of
EPP and XLP in adults and adolescents aged 12 years and older
Number of patients randomized to bitopertin in AURORA
51
Total number of EPP/XLP patients treated in AURORA and BEACON
101
What percentage of patients are reported to have liver failure due to EPP?
Up to 5%
What color are bitopertin tablets?
Yellow
Bitopertin dosage and administration for adults
60 mg once daily on an empty stomach
Stratification factor used in AURORA
baseline light tolerance (<30 min or >=30 min)
Total number of patients in the bitopertin safety database
>4000
XLP accounts for approximately what percentage of erythropoietic protoporphyrias?
10%
Glycine undergoes a condensation reaction with this in the first step of heme synthesis.
succinyl-CoA
What is bitopertin contraindicated in?
nothing
Prespecified key secondary endpoint in AURORA
Total hours of sunlight exposure on days with no pain from 10:00am to 6:00pm.
What SAEs were reported in AURORA
obstructive pancreatitis (in placebo)
This term refers to an aspect of phototoxicity whereby sun exposure on one day enhances sensitivity to sunlight on the following days
priming effect
What is the half-life of bitopertin?
49 h (~2 days)
What fruit should be avoided when taking bitopertin?
grapefruit
Number of phototoxic reactions occurring in the 60 mg dose group during the last 60 days of AURORA
zero
How many patients discontinued early from AURORA due to a treatment-emergent adverse event?
2 (dizziness and rash)
Percentage of metal-free protoporphyrin in EPP
>90%
Other indications that bitopertin was studied in
schizophrenia, obsessive compulsive disorder, and beta thalassemia
Recommended dosage for strong CYP3A4 inhibitors
Avoid coadministration with strong CYP3A4 inhibitors