Albireo Pharma reported dazzling Phase III ASSERT data of Bylvay for Alagille syndrome (ALGS). The company is headed to U.S. and European regulators with expectations of regulatory filings in Q1 of 2023.
Albireo Pharma reported positive Phase III ASSERT data of Bylvay for Alagille syndrome (ALGS) Tuesday. The company is headed to U.S. and European regulators with expectations of regulatory filings in Q1 of 2023.
In a conference call, Ron Cooper, president and CEO of Albireo, said he was “pleased to announce a positive outcome for [the] trial, the first and only gold-standard” study for Alagille syndrome.
Because it would be a supplemental filing with Orphan Drug Designation in both the U.S. and Europe, Albireo expects a “rapid review and approval” to make the drug available to the Alagille community in 2023.
ALGS is a rare, multisystem genetic disease. It affects about 25,000 people worldwide. Although it can affect numerous organs, including the liver, heart, skeleton, eyes, central nervous system, kidneys and facial features, insufficient bile ducts cause liver damage.
About 95% of patients have chronic cholestasis caused by poor transport of bile acid from the liver to the intestine. This typically occurs within the first three months of life.
A major symptom of the disease, affecting about 88%, is severe itching, which has a major negative impact on the quality of life of the patients and their caregivers.
The ASSERT trial demonstrated early, rapid and sustained decreases in pruritus and bile acids in Alagille syndrome patients.
The study evaluated 52 patients with genetically confirmed diagnoses of ALGS at 32 sites across North America, Europe, the Middle East and Asia Pacific. It examined the safety and efficacy of 120 micrograms/kg/day of Bylvay for 24 weeks.
The primary endpoint was relief of pruritis.
“There was a highly statistically significant change in pruritus and serum bile acids,” said Jan Mattsson, Ph.D., CSO, managing director (Sweden) and co-founder of Albireo, in the conference call with reporters.
Because the itching is often so intense, children affected often have sleep issues.
The study found “profound sleep improvements over time,” Mattsson said.
Mattsson said that sleep deprivation can have a major impact on quality of life for patients and caregivers.
The study also hit the key secondary endpoint of a statistically significant decrease in serum bile acid concentration from baseline to the average of weeks 20 and 24.
The drug was well tolerated, with adverse events occurring about as often as they did in the placebo group.
The company is close to full enrollment of its third Phase III BOLD study of the drug in biliary atresia.
Bylvay (odevixibat) is a once-daily, non-systemic ileal bile acid transport inhibitor (IBATi). It has been approved in the U.S. for treatment of pruritus (itching) in patients three months of age and older in all forms of progressive familial intrahepatic cholestasis (PFIC) and in Europe for the same indication in all patients six months or older.
Albireo reported $270 million in cash, with enough funds to execute on its plans. The company expects to see PFIC revenue increase with third-quarter Bylvay sales to surpass $7 million.
Cooper noted that with possible expansion from PFIC into Alagille patients, the potential patients should increase from 25,000 to 40,000 between the two indications.
The drug stands to have a long-term impact on the lives and health of children with Alagille syndrome and their families, said Nadia Ovchinsky, M.D., pediatric gastroenterologist and hepatologist, Children’s Hospital at Montefiore and ASSERT principal investigator.
