The US Food and Drug Administration (FDA) has approved Mirum Pharmaceuticals’ Livmarli (maralixibat) oral solution to treat cholestatic pruritus in patients aged five years and older with progressive familial intrahepatic cholestasis (PFIC).
Estimated to affect one in every 50,000 to 100,000 births in the US and Europe, PFIC is a rare genetic disorder that causes progressive liver disease, typically leading to liver failure.
Signs and symptoms of the condition usually begin in infancy and include severe pruritus (itching), jaundice and a failure to grow at the expected rate.
The FDA’s decision on the therapy covers a broad range of PFIC subtypes and is supported by positive results from the late-stage MARCH study, which included 93 patients across a range of genetic PFIC forms as well as unidentified mutational status.
Results showed a “highly statistically significant” reduction in pruritus severity for those receiving Livmarli compared with placebo, Mirum outlined.
Chris Peetz, Mirum’s chief executive officer, said Livmarli “has the potential to have a transformational impact for patients with cholestatic pruritus associated with PFIC” and “offers an option for those patients with the rarest of subtypes”.
The company has also submitted a supplemental new drug application that supports the use of a higher concentration formulation of Livmarli, potentially enabling a label expansion for younger PFIC patients.
Richard Thompson of King’s College London, and an investigator in the MARCH study, said: “Livmarli’s approval in cholestatic pruritus for patients with PFIC is a result of years of investigation and a collection of a strong body of clinical evidence showing meaningful improvements across a number of important parameters, including pruritus, affecting children with PFIC.”
Livmarli is already approved in the US to treat cholestatic pruritus in patients aged three months and older with Alagille syndrome, a rare genetic disorder that can affect multiple organ systems of the body, including the liver and heart.
Mirum announced last month that long-term results supporting the use of the drug in patients with Alagille syndrome had been published in the journal Hepatology.
The analysis demonstrated a statistically significant improvement in six-year event-free and transplant-free survival in patients who were treated with Livmarli compared with a natural history control group.