The U.S. Food and Drug Administration (FDA) has approved Digestive Care’s Pertzye (pancrelipase) to be given to cystic fibrosis patients through a so-called G-tube — a feeding tube delivering nutrition directly to the stomach through a hole in the abdomen.
Pertzye is an enzyme replacement therapy for exocrine pancreatic insufficiency caused by cystic fibrosis or other conditions.
Although cystic fibrosis is well-known for problems with the airways, mutations in the defective CFTR gene in patients can also affect other organs, like the pancreas. This organ releases digestive enzymes — necessary to break down food — into the gut.
The CFTR protein is a channel that allows chloride ions to pass over a cell membrane, and the loss of this ability makes mucus in the pancreas sticky, preventing it from releasing enzymes.
As a result, attaining proper nutrition can be difficult for cystic fibrosis patients, but pancreatic enzyme replacement therapy can significantly improve the intake of nutrients and prevent gut problems.
“We are pleased to offer the first FDA approved G-tube administration of enteric-coated pancreatic enzyme microspheres through a gastrostomy tube. This will allow physicians and dieticians to work together with EPI [exocrine pancreatic insufficiency] patients who have G-tubes and their caregivers to determine which method of administration of Pertzye is most appropriate,” Tibor Sipos, president and chief scientific officer of Digestive Care, said in a press release.
A compound that is enteric-coated is protected from being broken down in the stomach. It is not released until it reaches the small intestine, where pancreatic enzymes normally enter the gut.
To allow administration through a G-tube, researchers adapted a pediatric dose form of the treatment — a capsule with 4,000 lipase units — using smaller size pancreatic enzyme microspheres. This allowed the enzymes to pass through a feeding tube of French size 14 (a diameter of 4.67 mm or 0.184 inches) or larger.
“Digestive Care, Inc. and Chiesi are committed to delivering therapeutic treatment options that meet the unique health needs of CF [cystic fibrosis] patients,” said Alan Roberts, senior vice president of scientific affairs at Chiesi, which is Digestive Care’s marketing partner.
“We continue to support patients and caregivers alike, through exploring treatment options that bring innovative solutions to CF patients and our various touch points within the CF community,” added Roberts.
The FDA’s labeling revision approval is considered by Digestive Care a milestone achievement in Pertzye’s development program.