2021-07-26| Trials & Approvals

Albireo Gets Back-to-Back Approvals for Rare Liver Disease Drug

by Daniel Ojeda
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On July 20th, Albireo announced that the FDA approved Bylvay to treat pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC). This is a huge milestone for the company, as Bylvay is its first commercially available drug in the US. In addition, the approval came just one day after the European Commission approved Bylvay. As a result, the company’s stock is up 7.36% in the past 5 days.  

PFIC is a rare pediatric genetic disorder that causes progressive, life-threatening liver disease. Patients suffer from cholestasis, or bile accumulation in liver cells. Symptoms include itchiness, jaundice, poor weight gain, and slow growth. Within the first 10 years of life, PFIC leads to cirrhosis and liver failure. Bylvay is the first drug approved for the treatment of this disease.

“Treating children with PFIC can be difficult and frustrating given the current treatment options. Bylvay gives us a non-surgical option and will change how we treat PFIC,” said Richard Thompson, Professor of Molecular Hepatology at King’s College London.


Bylvay – The First of Albireo’s Many Candidates for Rare Liver Disease

Spun out of AstraZeneca and based in Boston, Albireo focuses on developing therapeutics for rare liver disease. They use novel bile acid modulators to treat rare pediatric and adult liver diseases. 

Their pipeline includes A3907, an inhibitor of apical sodium-dependent bile acid transporter (ASBT), which is currently in Phase 1 clinical trial to treat adult liver diseases. Additionally, their treatment for chronic constipation, Elobixibat, is approved in Japan and Thailand. Additionally, their lead compound Bylvay is also being tested in several Phase 3 clinical trials to treat Biliary Atresia, Alagille Syndrome, and other cholestatic diseases.   

Bylvay is a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi). Its efficacy was determined in the PEDFIC1 and PEDFIC2 Phase 3 clinical trials. In the randomized, double-blind, placebo-controlled, global multicenter PEDFIC1 study, Bylvay successfully reduced serum bile acid responses and itchiness (pruritus). PEDFIC2 showed the reduction in both markers was sustained in patients up to 48 weeks. Besides, Bylvay was well tolerated, with diarrhea being the most common adverse effect.

After the FDA nod, Albireo announced the packing and shipping of Bylvay within days. Sales promotions will begin immediately. Additional areas of focus are access and reimbursement and patient support. 

“Bylvay is the first-ever approval by the FDA of a drug developed for a pediatric cholestatic liver disease and provides a non-surgical treatment for patients living with the burden of PFIC,” said Ron Cooper, President, and CEO of Albireo. “We’re humbled by the children, families, and investigators whose commitment to our clinical trials will bring hope and treatment benefits for so many future patients.”

The approval of Bylvay is crucial for Albireo as it will bring more revenue, and the FDA issued a Rare Pediatric Disease Priority Review Voucher that the company plans to sell. As of June 30th, 2021, Albireo had between $183.3 million in cash and cash equivalents. The company anticipates that the sum will cover its operating expenses into 2023.

Related Article: Merck’s FDA approval for Pneumonia Vaccine Sets Up Collision Course with Pfizer

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