FDA Approval for Colbam (Cholic Acid) Capsules
Today the U.S. Food and Drug Administration approved Cholbam (cholic acid) capsules, the first FDA approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders (including Zellweger spectrum disorders).
Patients with these rare, genetic, metabolic conditions exhibit manifestations of liver disease, steatorrhea (presence of fat in the stool) and complications from decreased fat-soluble vitamin absorption.
Individuals with these rare disorders lack the enzymes needed to synthesize cholic acid, a primary bile acid normally produced in the liver from cholesterol. The absence of cholic acid in these patients leads to reduced bile flow, accumulation of potentially toxic bile acid intermediates in the liver (cholestasis), and malabsorption of fats and fat-soluble vitamins in the diet.
If untreated, patients fail to grow and can develop life-threatening liver injury.
Cholbam is approved as an oral treatment for children aged three weeks and older, and adults. The manufacturer of Cholbam was granted a rare pediatric disease priority review voucher–a provision that encourages development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
“This approval underscores the agency’s commitment to making treatments available to patients with rare diseases,” said Julie Beitz, M.D., director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research (CDER). “Prior to today’s approval, patients with these rare bile acid synthesis disorders had no approved treatment options.”
(Read more at here)
Guidance for Industry and FDA Staff: Current Good Manufacturing Practice Requirements for Combination Products
This guidance describes and explains the final rule on CGMP requirements for combination products (final rule as codified in 21 CFR part 4) that FDA issued on January 22, 2013.
Prior to issuance of the final rule, although CGMP regulations were in place to establish requirements for drugs, devices, biological products, and Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps), there were no regulations to clarify and explain the application of these CGMP requirements to combination products.
The final rule was intended to provide such clarification and specify how compliance with applicable CGMP requirements may be demonstrated.
Section II of this document provides the definition of a combination product, an overview of the final rule, and the role of the lead center and other agency components with respect to combination product CGMP issues.
Section III addresses certain general considerations for CGMP compliance for combination products.
Section IV presents the purpose and content of specific CGMP requirements addressed in the final rule.
Section V analyzes hypothetical scenarios intended to clarify how to comply with certain CGMP requirements addressed in the final rule.
Throughout this guidance, the agency also refers to existing guidance and additional sources of information that address CGMP requirements for drugs, devices, biological products, and HCT/Ps, as further inform combination product manufacturers on how to comply with CGMP requirements.