Ocaliva Review – Should You Try It?
Ocaliva is the brand name for a generic drug named obeticholic acid. The drug was recently chosen for accelerated approval by the FDA. Here’s our guide to Ocaliva.
What is Ocaliva?
Ocaliva is a first-in-class farnesoid X receptor (FXR) agonist that is actively in development for the treatment of primary biliary cirrhosis, or PBC.
The drug is produced by Intercept Pharmaceuticals. The generic name of Ocaliva is obeticholic acid.
In April, 2016, an FDA advisory committee voted 17 to 0 to recommend accelerated approval for the drug, causing company shares to skyrocket 11%. The FDA specifically stated they had no major concerns regarding the drug.
A week after the FDA’s vote, Intercept Pharmaceuticals announced results from new analyses on the phase III trial, POISE, on Ocaliva, which is the company’s leading pipeline candidate.
Trials are currently underway as the drug prepares to hit the market. Although it’s not currently available, it may be available in the near future (assuming clinical trials show the drug works with no serious side effects).
How Does Ocaliva Work?
We’re going to get a little technical here, so bear with us.
Ocaliva works to treat primary biliary cirrhosis, or PBC, by reducing the activity of an enzyme named alkaline phosphatase, or ALP. It works as an agonist of the farnesoid X receptor (FXR).
Current studies on the drug are seeking to determine whether ALP is a “reasonably likely” predictor of improved outcomes in PBC treatment.
We already know that Ocaliva provides short-term benefits to the liver. Future testing is seeking to determine whether or not there will be long-term benefits.
One problem is that ALP measures are not the most accurate measure of liver functionality. However, that’s the marker we’re using to determine the effectiveness of Ocaliva. As one FDA panelist explained,
“It’s not the best [marker], but it’s the best we have.”
How to Use Ocaliva
When the FDA voted to accelerate approval for Ocaliva, they also accepted Intercept’s proposed dosage and administration for Ocaliva.
The pharmaceutical company recommends starting with a 5mg daily dosage before progressing to 10mg daily dosages after several months after the patient grows tolerant to the drug.
One concern the FDA had with this dosage is that there may be long-term “mild liver-related events” in patients taking the high dose for long periods of time, so future long-term studies are required.
What is Primary Biliary Cirrhosis (PBC)?
Primary biliary cirrhosis, or PBC, is a rare liver disease caused by autoimmune destruction of the bile ducts that transport bile acids out of the liver. This leads to a high level of bile within the liver, or a condition known as cholestasis.
The disease is primarily seen in women and it afflicts approximately one in 1,000 women over the age of 40.
Since 1988, PBC has been the second-leading overall cause of liver transplant in women in the United States (Hepatitis C is the number one cause).
Meanwhile, European doctors report that PBC accounts for approximately half of all liver transplants due to cholesteric diseases and 6% of all liver transplants in total.
About Intercept
Intercept is a biopharmaceutical company. The company’s About Us page claims that it’s:
“focused on the development and commercialization of novel therapeutics to treat chronic liver diseases utilizing our proprietary bile acid chemistry.”
So far, obeticholic acid (Ocaliva) is the company’s leading pipeline drug. Intercept owns global rights to OCA outside of Japan, China, and Korea, where the company has out-licensed the drug to a company named Sumitomo Dainippon Pharma.
Other pipeline drugs from the company include bile acid analogs like INT-767, which is currently in clinical development.
Intercept is based in New York City. You can get in touch with the company via their contact page here: Interceptpharma.com/about/contact-us/
