Intercept’s lead product has won US regulatory approval for a rare liver disease, which may multiply its takeover potential
Intercept Pharmaceuticals Inc. (ICPT), which has previously attracted takeover interest based solely on its pipeline, has won approval for its first product. Late on Tuesday, the biotech company said that the Food and Drug Administration (FDA) has granted accelerated but conditional approval to Ocaliva (obeticholic acid), for treatment of a rare liver condition called primary biliary cholangitis (PBC).
Ocaliva’s Potential in PBC
The chronic liver disease causes damage to the bile duct, which can eventually lead to permanent scarring and even develop into cirrhosis or liver failure. It affects approximately 40 to 400 people in every one million, with a higher onset rate in women and people aged 40 or above. The disease has had only one approved treatment called ursodeoxycholic acid (UDCA) for the past two decades. Ocaliva will provide much-needed recourse to up to 40% of PBC patients who do not respond adequately to UDCA.
The FDA has cleared Ocaliva for use in combination with UDCA in adult patients who respond inadequately to the latter. Ocaliva can be administered as a monotherapy to those who cannot tolerate UDCA altogether. The drug has been granted accelerated approval based on a reduction in alkaline phosphatase (ALP) – an important marker for liver damage. However, since Ocaliva’s ability to improve survival or disease-related symptoms has not yet been established, its continued approval for the indication is contingent upon clinical benefit to be delivered in confirmatory trials.
Intercept plans to make Ocaliva available through its specialty pharmacy network within seven to ten days. The company has applied a list price of $69,350 for a year’s worth of treatment, justified by chief commercial and corporate affairs officer Lisa Bright as such: “When establishing the price for Ocaliva, we considered several factors including the benefit that Ocaliva offers to people living with PBC, which of course is an orphan disease for which there have been no new treatments in nearly 20 years. And, finally, we consider the consequences of an inadequate treatment in a progressive liver disease, including… liver transplants and death and the savings associated with slowing disease progression.”
The company said it would take several months for Ocaliva to be added to insurers’ formularies. Management projects gross peak annual sales of more than $1.6 billion from the drug in 2022 and profitability in 2019.