CORAL GABLES, Fla. – The Food and Drug Administration has given the Green light to the first drug to help treat symptoms of Progressive Familial Intraheptic Cholestatis, or PFIC.
Dr. Ruben Gonzalez-Vallina, a pediatric gastroenterologist with Nicklaus Children’s Hospital, said the approval of the drug called Bylvay will help treat the intense itching brought on by the disease which can progress to cirrhosis and liver failure within 10 years.
“With this medication, we can give it at 3 months of age or older and you just open the capsule, sprinkle it into two to three tablespoons of baby food and the itching goes away, you stop the disease practically and it’s a medication you can give all the way to 16 years old and above,” he said.
The inherited disease is caused by certain gene mutations and appears within the first few months after birth.
Convalescent plasma study
A study by the National Institutes of Health found no significant benefit from the use of convalescent plasma for treating COVID-19 outpatients with early symptoms.
The final results of the clinical trial showed the therapy did not prevent disease progression in a high-risk group of patients.
COVID-19 convalescent plasma is derived from people who have recovered from the virus.
Last year it was approved by the FDA for use in hospital patients with COVID-19.