Aptalis gains double win from US FDA for pancreatic enzymes
This article was originally published in Scrip
Aptalis Pharma won the US FDA's blessing on 1 March to market Ultresa (pancrelipase) and Viokace (pancrelipase) to treat exocrine pancreatic insufficiency (EPI), a condition in which the body is unable to properly digest fat, protein and carbohydrates and absorb nutrients.
EPI is most often caused by diseases that destroy the exocrine function of the pancreas, such as cystic fibrosis, chronic pancreatitis or pancreatic cancer. It affects about 90% of cystic fibrosis patients.
Prior to Ultresa's and Viokace's approval, there were only three drugs on the US market to treat EPI: Abbott Laboratories' Creon (pancrelipase), Eurand Pharmaceuticals' Zenpep (pancrelipase) and Johnson & Johnson's Pancreaze (pancrelipase). Those drugs, like Ultresa and Viokace, are porcine-derived pancreatic enzyme products (PEPs), which are complex mixtures of lipases, proteases and amylases and other enzymes.
"With the FDA's approval of these products, more options are now available to those patients, their caregivers and their physicians," said Dr Frank Verwiel, president and CEO of privately held Aptalis. "This is an important milestone which reinforces Aptalis' longstanding commitment to serving their needs."
The labelling for Viokace and Ultresa emphasize that the drugs are not interchangeable with any other pancrelipase product.
Ultresa is specifically indicated as a treatment for EPI in adults and children due to cystic fibrosis or other conditions, while Viokace, in combination with a proton-pump inhibitor, is indicated in adults to treat EPI due to chronic pancreatitis or pancreatectomy.
Viokace's labelling also notes that the safety and efficacy of the drug has not been established in children.
PEPs have been marketed in the US for several decades, with many of those products predating the passage of the Food, Drug and Cosmetic Act of 1938. But until recently, none of those products were marketed under approved NDAs.
But in April 2010, the FDA ordered makers of unapproved PEPs to stop manufacturing and distributing their drugs in the US under a rule first announced by the FDA in 2004.
The FDA's action was aimed at ensuring all PEPs have undergone scrutiny and testing.
The FDA began its process of evaluating the safety and effectiveness of PEPs in the late 1970s, with the initial intent of establishing a monograph for over-the-counter marketing of the drugs.
But because of bioavailability problems associated with the use of PEPs and other concerns reported to the agency, regulators in 1985 reconsidered that approach and determined that an OTC drug monograph would not be sufficient to adequately regulate PEPs.
In 2004, the FDA decided to consider PEPs new drugs, and announced new conditions for continued marketing. The agency also proposed new draft guidelines for manufacturers that year.
The FDA initially set April 2008 as the deadline for drugs to come into compliance with the NDA process. But the agency in 2007 extended that date to 28 April 2010.
In April 2009, Solvay Pharmaceuticals, which was swallowed up by Abbott in September 2009, was the first to gain the FDA's OK for a PEP with Creon.
Eurand followed four months later in August 2009 with the approval of Zenpep, with J&J gaining the FDA's nod in April 2010 for Pancreaze.
The FDA said other firms have submitted applications for PEPs.
Lilly tried and failed to gain the FDA's approval to market its non-porcine pancreatic enzyme replacement therapy liprotamase as a treatment for EPI.
In a complete response letter, the FDA said it wanted another clinical trial before it would permit liprotamase to enter the US market as a treatment for EPI (scripintelligence, 19 April 2011).
The FDA's decision followed a in January 2011 vote of 7-4, with one abstention, by the agency's Gastrointestinal Drugs Advisory Committee, which said the drug's potential risks outweighed its benefits in treating EPI –determining that the experimental therapy was not any better than the already approved medicines (scripintelligence, 13 January 2011, 11 January 2011).