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2022-10-12| Trials & Approvals

Albireo Signals To Apply For FDA Approval After Trial Win

by Joy Lin
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Rare disease company Albireo Pharma has signaled its intention to apply for approval in the US and EU after its bile acid modulator Bylvay (odevixibat) scored a win in the Phase 3 Assert study in Alagille syndrome (ALGS), a rare inherited disorder that affects the liver. Previous discussions with the FDA and EMA indicated that a single successful study would provide sufficient grounds for approval. 

Bylvay is approved in the US to treat pruritus (itchy skin) in patients aged 3 months and older with progressive familial intrahepatic cholestasis (PFIC). In the EU and the UK, the medicine is indicated for PFIC patients aged 6 months and older. The drug has orphan exclusivity for its approved PFIC indications in the US and Europe.

Related Article: FDA Accepts and Boosts sBLA for Takeda’s HAE Treatment for Pediatric Patients

Reducing Pruritus And Bile Acids

ALGS is a rare genetic disease that affects multiple organs, such as the liver, heart, and kidney. Patients with the disease do not have enough bile ducts, which leads to a buildup of bile in the liver, causing liver damage. Approximately 95% of patients present with chronic cholestasis (interrupted bile flow) while up to 88% also present with severe, uncontrollable itching. ALGS impacts around 25,000 people globally, said Albireo. 

Bylvay, a once-daily ileal bile acid transport inhibitor (IBATi), is designed to block a protein from transporting bile acids from the intestine into the liver. It acts locally in the small intestine and therefore, has minimal systemic exposure. 

According to topline data from the Phase 3 Assert study, Bylvay brought about statistically significant reductions in pruritus, meeting the trial’s primary endpoint. The drug also achieved its secondary endpoint of reducing serum bile acids. 

On the safety front, the study recorded low rates of drug-related diarrhea (11.4% vs. 5.9% placebo), a common side effect. Other side effects of the medication include soft stools, belly pain, and an enlarged liver. None of the patients in Assert discontinued Bylvay. 

“Our Assert Phase 3 study demonstrated early, rapid, and sustained effects on reducing pruritus and bile acids in Alagille syndrome, as it did in Phase 3 PFIC study,” said Ron Cooper, President and CEO of Albireo. 

Besides having completed two of three pediatric liver disease studies and waiting for full enrollment in a third for biliary atresia, the company possesses sufficient resources — more than $270 million — to execute their plans, said Cooper. The company expects to see an acceleration in PFIC revenue with expected Q3 Bylvay sales exceeding $7 million, he said. 

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