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FDA Approves Sixth Pancrealipase Product for Cystic Fibrosis


May 23, 2012 — The US Food and Drug Administration (FDA) has approved pancrealipase delayed-release capsules (Pertzye, Digestive Care Inc) for the treatment of children and adults with exocrine pancreatic insufficiency (EPI) caused by cystic fibrosis (CF) or other conditions.
The unique pancreatic enzyme product contains bicarbonate-buffered enteric-coated microspheres and is protected by several US and international patents. It has been marketed by the company under the trade namePancrecarb MS-16 since 2004.
Representing the sixth such product for patients with CF, Pertzye comes on the heels of 2 other newly FDA-approved similar products (Ultresa, Aptalis Pharma US Inc; and Viokace, Confab Laboratories Inc), as reported byMedscape Medical News.
Other previously FDA-approved pancrealipase pharmaceuticals include Creon (Abbot Labs), Zenpep (Eurand SPA), and Pancreaze (Ortho-McNeil-Janssen Pharmaceuticals Inc).
Small Study Leads to Big Results
FDA approval was based on data from a small, randomized, double-blind study of 21 patients aged 8 to 43 years (mean, 20 years) who were randomly assigned to receive the study drug at individually titrated doses or matching placebo for 6 to 8 days of treatment, followed by crossover to the alternate treatment for an additional 6 to 8 days.
Results showed that the mean difference in coefficient of fat absorption (CFA) was significantly higher for the study drug relative to placebo, as determined by a 72-hour stool collection during both treatments to measure both fat ingestion and excretion (83% vs 46%; 95% confidence interval [CI], 28% - 45%; P < .001).
The coefficient of nitrogen absorption was likewise significantly increased (79% vs 47%), as evaluated using the same stool samples, with the assumption that proteins contain 16% nitrogen.
No differences were found between children and adults with respect to the severity of pancreatic insufficiency or response to the study medication.
"The improvement in mean CFA observed in the controlled study represents a clinically meaningful treatment benefit.... Availability of this unique buffered formulation of pancreatic enzyme is an important addition to the therapeutic options for CF and other patients with EPI," stated Michael W. Konstan, MD, chairman of pediatrics, Rainbow Babies and Children's Hospital and Case Western Reserve University School of Medicine, Cleveland, Ohio, in a company news release.
The most commonly observed adverse events were diarrhea, dyspepsia, and cough (10% vs 4% for those receiving placebo, for each event).
Recommended Dosing
Pancrealipase delayed-release capsules will be available in 2 formulations:
  • 8000 USP units of lipase with 28,750 USP units of protease and 30,250 USP units of amylase; and
  • 16,000 USP units of lipase with 57,500 USP units of protease and 60,500 USP units of amylase.
Children aged from 1 to less than 4 years and weighing 8 kg or more should begin with a dose of 1000 lipase units/kg body weight per meal (maximum, 2500 units or ≤10,000 units/kg/day) or less than 4000 lipase units/g fat ingested daily.
Older children and adults should start with a dose of 500 lipase units/kg body weight (maximum, 2500 units/kg per meal or ≤4000 lipase units/g fat ingested daily).
Because ingestion of larger amounts is linked to a risk for fibrosing colonopathy, the FDA warns patients to contact their clinician immediately if they have unusual or severe abdominal pain, bloating, and trouble passing stool, nausea, vomiting, or diarrhea.
Capsules should be taken whole to avoid irritation of the oral mucosa, but contents may be mixed with applesauce for infants and others having difficulty swallowing.
Treatment is associated with a risk for hyperuricemia, so uric acid level monitoring should be considered in patients with gout or renal impairment. Caution is advised when prescribing pancrealipase delayed-release capsules for patients with a known allergy to proteins of porcine origin.
 

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