First FDA-Approved Medication for Rare Liver Disorder Launched

First FDA-Approved Medication for Rare Liver Disorder Launched

July 20, 2021 – Albireo Pharma’s Bylvay^TM (odevixibat) has become the first drug approved by the U.S. FDA to treat pruritus in individuals who are at least three months old and have been diagnosed with progressive familial intrahepatic cholestasis (PFIC).

PFIC is a rare genetic disorder that causes worsening liver disease, leading to liver failure as early as infancy in some patients. In PFIC, bile does not move from the liver to the intestines as it should. Instead, the bile accumulates in and damages the liver’s cells. Complications of the disease include pruritus (severe and debilitating itching), jaundice (yellowing of the skin and eyes), portal hypertension (high blood pressure in the vein supplying the liver with blood), hepatosplenomegaly (enlarged liver and spleen), and failure to gain weight and grow at expected rates. Treatment options are limited, generally consisting of nutritional management and invasive surgical procedures.

In clinical trials, Bylvay delivered sustained reduction of serum bile acids in addition to improved pruritus assessments, growth, and other markers of liver function. Recommended dosing is 40mcg/kg of the patient’s body weight taken with a meal each morning. The dosage can be increased after three months if clinical response to treatment is inadequate. Increases should occur in increments of 40mcg/kg, up to a maximum dose of 120mcg/kg and not exceeding a total daily dose of 6mg. Bylvay capsules can be swallowed whole or opened and sprinkled onto soft food. The medication should only be used under the supervision of a doctor who has experience in managing PFIC, and patients on exclusively liquid foods should not use Bylvay.

Albireo launched the product immediately upon approval at a wholesale acquisition cost (WAC) of $39,600 per 30-count of 1,200mcg capsules.