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news.Albireo Pharma's lead product candidate, A4250, has been granted access to the PRIority MEdicines (PRIME) program of the European Medicines Agency (EMA) to treat progressive familial intrahepatic cholestasis (PFIC).
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Albireo president and CEO Ron Cooper said: “PFIC is a highly debilitating genetic liver disorder that is life altering for affected children and their families.
“There is an urgent need for an effective pharmacological treatment for PFIC, as there are currently no approved drug options in the United States or Europe. A4250’s acceptance into the PRIME program serves as validation of its promise to meet that need. We look forward to collaborating with the EMA on the development plan for A4250, as well as the potential accelerated assessment of A4250 in Europe.”
The PRIME program was launched by the EMA to provide enhanced support to developers of investigational medicines that target an unmet medical need, with a focus on those that may offer a major therapeutic advantage over existing treatments or address a disease with no current treatment option.
The program is designed to provide early engagement with the EMA to optimize development plans and speed up evaluation, with the goal of helping patients benefit as early as possible from therapies that may significantly improve their quality of life.
As of August 2016, only 13 out of 57 requests for PRIME eligibility had been accepted into the program. Having been accepted into the program, A4250 may be eligible for accelerated assessment of a potential future marketing authorization application (MAA) in Europe.
A4250 is currently being evaluated in children with chronic cholestasis in a Phase 2 clinical trial that is intended to support a potentially pivotal clinical trial in PFIC planned to be conducted in the United States and Europe.
Albireo anticipates meeting with the U.S. Food and Drug Administration (FDA) regarding the planned PFIC trial in the first quarter of 2017.
About A4250
A4250 is a first-in-class product candidate in development for progressive familial intrahepatic cholestasis (PFIC) and potentially other orphan pediatric cholestatic liver diseases. A4250 is a highly potent and selective inhibitor of the ileal bile acid transporter (IBAT), has minimal systemic exposure and acts locally in the gut.
A4250 is currently being evaluated in children with chronic cholestasis in a Phase 2 clinical trial that is intended to support a potentially pivotal clinical trial in PFIC.
About Progressive Familial Intrahepatic Cholestasis
Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive, life-threatening liver disease, which may start early after birth or at a young age and rapidly progress to end-stage liver disease.
Three alternative gene defects have been identified that correlate to three separate PFIC subtypes, known as types 1, 2 and 3. The precise prevalence of PFIC is unknown, but PFIC has been estimated to affect between one in every 50,000 to 100,000 children born worldwide.
PFIC is commonly associated with elevated serum bile acids. Prominent symptoms of PFIC include pruritus, which is associated with severe sleep disturbance and diminished overall quality of life, and poor growth. First-line treatment in PFIC is typically off-label ursodeoxycholic acid (UDCA).
Notwithstanding treatment with UDCA, many PFIC patients will require partial external biliary diversion (PEBD) surgery and ultimately liver transplantation. Although success rates vary, published third-party studies have shown that PEBD surgery can slow, and in some cases stop, the progression of liver disease and lead to reduced pruritus and improved sleep.