Cholbam Approved to Treat Bile Acid Synthesis Disorders
March 17, 2015 – The U.S. Food and Drug Administration (FDA) approved Cholbam (cholic acid) capsules manufactured by Asklepion Pharmaceuticals. Cholbam is indicated for treating rare disorders of bile acid synthesis caused by a defect in one enzyme and can be used for patients as young as three weeks old.
Cholbam is also approved to be used in combination with standard treatments for peroxisomal disorders, a group of very serious inherited conditions that result from missing or malfunctioning peroxisomes (parts of cells that produce enzymes to break down fatty acids). Babies born with a peroxisomal disorder generally are delayed in behavioral, mental and physical development which can include hearing, speech and vision problems. Many babies with peroxisomal disorders are on the autism spectrum and most have enlarged livers.
The cost of Cholbam is not yet available, but it is expected to be similar to the cost of other drugs that treat very rare conditions at approximately $400,000 per year. Cholbam is expected to be launched by early April and will be available exclusively through one specialty pharmacy.
Adverse reactions or quality problems experienced with the use of a recalled product may be reported to the FDA’s MedWatch Adverse Event Reporting Program either online, by regular mail or by fax.
- Complete and submit the report Online: www.fda.gov/medwatch/report.htm
- Regular Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1-800-FDA-0178
Actual drug patent expiration dates and a availability of new medications are subject to change due to patent litigation, settlement agreements, additional patents, exclusivities, and final FDA approval. Distribution and availability of new medications at pharmacies may not occur immediately following FDA approval. Patients are advised to speak with their healthcare professional or pharmacist regarding appropriateness as well as actual availability.
*This is provided for information only. The reference to any medication above does not mean the medication is covered by your plan.