Orsini, Pantherx pharmacies will dispense Livdelzi for PBC in US
Partnership follows last week's FDA approval of second-line oral therapy
Specialty pharmacy services for Livdelzi (seladelpar), the newly approved second-line therapy for adults with primary biliary cholangitis (PBC), will be provided in the U.S. by Orsini and Pantherx Rare.
The partnership between these specialty pharmacies and Gilead Sciences, which owns Livdelzi, follows last week’s clearance of the oral therapy by the U.S. Food and Drug Administration (FDA). Livdelzi will be available in 10 mg oral capsules that are taken once per day, with a list price of $12,606 for 30 days of treatment.
“Livdelzi is an important treatment option to slow the progression of PBC and transform patients’ quality of life,” Brandon Tom, CEO of Orsini, said in a company press release. “We’re thrilled to partner with Gilead to address this need for people living with PBC and their families.”
The therapy can be used in combination with ursodeoxycholic acid (UCDA), the first-line therapy for PBC, in patients who don’t respond to UCDA, or alone in those unable to tolerate UCDA.
Eligible patients include those without cirrhosis, or permanent liver scarring, as well as individuals with well-compensated cirrhosis, meaning the liver can still perform its functions despite extensive scarring.
“Orsini is now dispensing Livdelzi … to treat primary biliary cholangitis (PBC),” the release states.
Patient support on Livdelzi coverage available from Gilead
Gilead Support Path, offered by the company, provides information and resources for understanding coverage and financial options for Livdelzi. It also offers screening for multiple programs — the patient assistance program, co-pay coupon program, and interim support program — all of which can be accessed via patient enrollment forms.
Those submitting a form can expect a call from a specialist within two days. That specialist will assist the patient with the next steps in the process and answer questions.
PBC is a chronic form of cholangitis, or inflammation of the bile ducts that carry bile, a digestive fluid, from the liver to the intestines. Over time, cholangitis affects bile flow, resulting in bile accumulation to toxic levels in the liver, damaging the organ and allowing bile to leak into the bloodstream. The disease ultimately leads to itching and other symptoms.
While the disease can occur among both male and female patients, it’s more common among women. According to the American Liver Foundation, PBC is estimated to affect about 65 of every 100,000 women in the U.S.
Livdelzi works by activating a protein known as PPAR-delta, which helps regulate genes related to bile production, inflammation, and scarring — all processes related to PBC. As such, the therapy is expected to reduce bile production and lessen liver damage and itching.
Therapy’s US approval based on positive clinical trial results
The treatment’s U.S. approval was mainly based on findings from the Phase 3 RESPONSE clinical trial (NCT04620733). In that study, after one year, a significantly greater proportion of patients receiving Livdelzi achieved a biochemical response relative to those given a placebo (61.7% vs. 20%).
The primary goal was defined as reaching a blood level of alkaline phosphatase (ALP) less than 1.67 times the upper limit of normal with at least a 15% decline from the trial’s start, as well as a level normalization of bilirubin. Both ALP and bilirubin are markers of liver damage, and high ALP levels are associated with a greater risk of needing a liver transplant or death.
In addition, 25% of Livelzi-treated patients saw their ALP levels return to the normal range versus none of those given a placebo. The therapy also was associated with a significantly greater drop in patient-reported itching relative to placebo after six months (by 3.2 vs. 1.7 points). Itching was assessed with a daily numerical rating scale.
These results and findings from other trials allowed the FDA to grant accelerated approval to Livdelzi. This type of approval path allows an experimental therapy to be marketed in the U.S. based on preliminary efficacy data.
People living with rare and devastating diseases deserve access to treatment options that work for them.
Continued and formal full approval will be dependent on additional trial data proving the treatment’s efficacy in terms of clinical outcomes such as liver-related events and survival. The ongoing Phase 3 AFFIRM trial (NCT06051617), running through 2029, is expected to provide such data.
The study is still recruiting up to 192 adults with PBC and compensated cirrhosis at sites in the U.S., Turkey, and South Korea. It’s testing the therapy’s ability to prevent such events and prolong survival relative to a placebo.
“People living with rare and devastating diseases deserve access to treatment options that work for them,” Rob Snyder, executive chair of Pantherx, said in a separate press release from that specialty pharmacy. “The partnership with Gilead Sciences aims to positively impact key clinical outcomes for those suffering from primary biliary cholangitis and we are pleased to be a part in that.”
Orsini is an accredited pharmacy that offers multiple services, including pharmacy distribution, patient services, and clinical management. Pantherx is a dual-accredited pharmacy that specializes in rare and orphan diseases.
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