Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

• ALT and AST Changes From Baseline [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]
  Changes from baseline (study LAL-CL01) to specific post-treatment time points in study LAL-CL04 for alanine aminotransferase (ALT) and aspartate aminotransferase (AST).
  
  

• Change From Baseline in Liver Volume [ Time Frame: From baseline (study LAL-CL04) to week 10 or 12, week 24, week 52, week 104 ]
  Change in liver volume (in multiples of normal [MN]) from the LAL-CL04 baseline to the indicated post-treatment time point in study LAL-CL04, as assessed by MRI
  
  
• Change From Baseline in Liver Fat Content [ Time Frame: From baseline (study LAL-CL04) to week 10 or 12, week 24, week 52, week 104 ]
  Percentage change in liver fat content from the LAL-CL04 baseline to the indicated post-treatment time point in study LAL-CL04, as assessed by multi-echo gradient-echo MRI
  
  
• GGT and ALP Changes From Baseline [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]
  Changes from baseline (study LAL-CL01) to specific post-treatment time points in study LAL-CL04 for gamma glutamyltransferase (GGT) and alkaline phosphatase (ALP).
  
  
• Lipid Changes From Baseline [ Time Frame: From baseline (study LAL-CL01) to week 10 or 12, week 24, week 52, week 104 ]
  Change from baseline in total cholesterol (Total-C), high density lipoprotein cholesterol (HDL-C), low density lipoprotein cholesterol (LDL-C), and triglycerides (TG).
  
  
• Serum Ferritin Change From Baseline [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]
  
• High Sensitivity C-reactive Protein (Hs-CRP) Change From Baseline [ Time Frame: From baseline (study LAL-CL01) to week 12, week 24, week 52, and week 104 ]
  

Cholesteryl Ester Storage Disease (CESD) is the late onset phenotype for Lysosomal Acid Lipase (LAL) Deficiency, a Lysosomal Storage Disorder, which also has an early onset phenotype known as Wolman Disease that primarily affects infants. CESD can present in childhood but often goes unrecognized until adulthood when the underlying pathology is advanced. Many of the signs and symptoms are common to patients with other liver conditions.

CESD is an autosomal recessive genetic condition and is characterized by hepatomegaly, persistently abnormal liver function tests (LFTs) and type II hyperlipidemia. Splenomegaly and evidence of mild hypersplenism may affect some patients. Untreated, CESD may lead to fibrosis, cirrhosis, liver failure and death.