Intercept’s Ocaliva got Accelerated FDA Approval for the Treatment of PBC

Intercept, a biopharmaceutical company got FDA approval for Ocaliva (Obeticholic Acid), a drug used for treating Primary Biliary Cholangitis.

The company is focused on the development and commercialization of drugs used for the treatment of non-viral and progressive liver diseases.   Ocaliva is said to be the first new treatment for PBC for almost 20 years of having only one available drug.

Ocaliva will be used for the treatment of Primary Biliary Cholangitis or previously known as Primary Biliary Cirrhosis. It is used in combination with UDCA or ursodeoxycholic for adult’s patients who are not responsive to UDCA or as a monotherapy for adults who cannot tolerate UDCA.  Ocaliva is a farnesoid X receptor or FXR agonist that works as a nuclear receptor that is expressed in the intestine and liver. It is a key regulator of bile acids fibrotic, inflammatory and metabolic pathways.

Intercept Pharmaceutical was created in the hope that targeting FXR receptors will benefit patients suffering from liver diseases. Their aim is to create a particular drug that targets a liver disease with no or little treatment options.  Mark Pruzanki, President and Chief Executive Officer of Intercept, shared that Ocaliva’s approval marked a decade of hard work in finding a cure for PBC.  He also extends his gratitude for all the help of the patients, doctor, researchers and participants who made these things possible.

He did share that the accelerated approval was based on the reduction of ALP or alkaline phosphatase. ALP is a liver enzyme and biomarker used to detect liver and bone diseases.  An elevated ALP marks obstruction or presence of a tumor.

Although, the improvement in the survival and manifestations of the symptoms was not established, there is still a need to undergo clinical trials for verification purposes.

John Vierling, Past President of the American Association for the Study of Liver Diseases and Professor of Medicine and Surgery at Baylor College of Medicine mentioned that Ocaliva fills a need for PBC patients who are intolerant or has inadequate response to UDCA.  Ocaliva marks an improvement for lowering ALP which is used to track the progression of PBC.  Most importantly, Ocaliva was able to reduce ALP which is critical for the management of chronic diseases like PBC.

In the Phase 3 Clinical Trial of Intercept, they were able to identify that one of the possible side-effects of the drug is pruritus or itching.  But the side-effect can be lessened if the patient will undergo a dose titration regimen that consists of 5 mg once daily and then increasing the dose to 10 mg once a day. Other possible side effects noted are discomfort, rash, fatigue, abdominal pain, dizziness, arthralgia, constipation, eczema, thyroid function abnormality, and or pharyngeal pain.

Linie Moore, President of the PBCers Organization, the leading support group for PBC patients and also a PBC patient, said that they are thrilled to welcome this new drug.  For two decades, there is only one approve treatment for this condition, and it is good to have another treatment option. She mentioned that PBC affects people in the prime of their years and it is a constant concern because it may cause a potential need for liver transplant.

Ocaliva will be available in the US market a week from now.

A New Hope for Treating PBC

The accelerated approval of Ocaliva is definitely good news for PBC patients.  Aside from having another treatment option, it also targets patients who are unresponsive to UDCA.  It is also great news that this drug can help lower alkaline phosphatase, a very important enzyme and biomarker. Indeed, this drug has a lot of potential.

There is a high hope that patients who are suffering from PBC will no longer have to undergo liver transplantation. This new drug is expected to lessen the symptoms and stop the progression of PBC.

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