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Albireo (ALBO) Gets FDA Nod for Rare Liver Disease Drug

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Albireo announced that the FDA has approved Bylvay (odevixibat) to treat intense itching or pruritus in progressive familial intrahepatic cholestasis (“PFIC”).

PFIC, a rare disorder, is a type of cholestasis or impaired bile flow caused by genetic mutations. This results in bile build-up in liver cells that causes progressive, life-threatening liver diseases including cirrhosis and liver failure. Pruritis is the most prominent and problematic ongoing PFIC symptom.

The FDA approval makes Bylvay — a potent, non-systemic ileal bile acid transport inhibitor — the first and only drug to treat pruritus in all subtypes of PFIC.

The company is anticipating an immediate launch of Bylvay for commercialization. The company is also partnering with Travere Therapeutics (TVTX - Free Report) for sales promotion of the product in the United States.

Shares of Albireo have declined 8.5% so far this year in comparison with the industry’s 2.3% decrease.

Zacks Investment Research
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Bylvay was approved in Europe earlier this week. The commercial launch of the drug in Europe is expected in September, starting with Germany, having the largest EU market potential.

The FDA and EU approvals were based on positive data from phase III PEDFIC 1 and PEDFIC 2 studies that evaluated patients with PFIC. Both the studies achieved sustained serum bile acid reduction and improvement in pruritus assessment, with no serious treatment-related adverse events.

PFIC mainly affects young children. Until now, there were only surgical options for those suffering from PFIC, without which patients could not survive past 30 years of age. Albeiro provides a non-invasive therapy option as Bylvay can be administered either as a capsule or sprinkled onto soft foods. Bylvay has the potential of changing the course of treatment for PFIC and is expected to drive the company’s prospects.

We inform investors that the FDA has issued a Rare Pediatric Disease Priority Review Voucher to Alberio, which it plans to monetize in order to fund its operating expenses and capital expenditure requirements beyond 2023.

Albireo is also developing Bylvay in other rare pediatric cholestatic diseases. A phase III study — BOLD — is evaluating the drug in biliary atresia. Another late-stage study, ASSERT, is evaluating the drug in patients with Alagille syndrome. Top-line data from the BOLD and ASSERT studies is anticipated in 2024 and 2022, respectively.

Zacks Rank & Stocks to Consider

Albireo currently carriesa Zacks Rank #4 (Sell).

Some better-ranked stocks in the biotech sector include Biomarin Pharmaceutical (BMRN - Free Report) and Exact Sciences (EXAS - Free Report) , each sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Biomarin’s earnings estimates for 2022 have increased from $2.33 to $2.35 per share in the past 60 days.

Exact Sciences’ loss estimates for 2021 have narrowed from $2.65 to $2.51 per share in the past 60 days.


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