Children with PFIC, a rare liver disease, face the prospect of cirrhosis and liver failure before the age of 10. During this process, the disease manifests as severe itching, and there is no FDA-approved treatment. Until this week.
Regulator Tuesday Approved odevixibat, An Albireo Pharma drug used to treat severe itching or itching in patients with progressive familial intrahepatic cholestasis (PFIC) of liver disease.The decision was given by the European regulator nod To the drug. Albireo, based in Boston, said that the drug will be launched in the United States in the next few days and sold under the name “Bylvay.”
PFIC is an inherited liver disease in which the organs cannot discharge bile normally, causing the accumulation of digestive juices. The disease is divided into different subtypes based on genetic mutations. The only treatment for this condition is surgery to transfer bile or liver transplantation. In a conference call on Wednesday, Albireo CEO Ron Cooper stated that interhepatic cholestasis was first described in the mid-1900s, and the specific gene for PFIC was characterized in 1998. The FDA approved Albireo for all subtypes of PFIC in patients 3 months and older.
Cooper said: “It took more than 20 years to turn the impossible into possible.” “We are now providing the first non-surgical treatment for PFIC patients and their families.”
According to Albireo, out of an estimated 100,000 patients with cholestatic liver disease, about 15,000 have PFIC, which does not include patients in China and India. The company calculated that there are approximately 600 PFIC patients in the United States. The way in which pruritus develops in patients with PFIC is not fully understood. Albireo drug is a small molecule that reduces the reabsorption of bile acids. Manufactured as a capsule, the medicine can be swallowed in this form or sprinkled on soft food.
The FDA approval is based on data from two phase 3 clinical trials. In the first randomized, double-blind, placebo-controlled study, the drug achieved its main goal of achieving statistically significant changes in pruritus and bile acids over the course of 72 weeks of treatment. These results were confirmed in an open-label Phase 3 expansion study, where the drug continued to show a continuous reduction in blood bile acids and an improvement in itching for up to 48 weeks. Patients tolerated the drugs well; the most common adverse reactions included elevated liver enzymes and diarrhea.
Itching has proven to be a tricky target for drug developers.Small molecules developed Menlo Therapeutics fails in clinical trial testing It is used for unexplained itching and itching associated with atopic dermatitis. The company located in Bridgewater, New Jersey changed its name to VYNE Therapeutics. Stopped the development of the compound last yearEarlier this year, Cara Therapeutics, headquartered in Stamford, Connecticut, reported on its experimental treatment of pruritus in mild to severe atopic dermatitis Phase 2 study failed.
Among companies seeking to treat pruritus associated with liver and kidney disease is San Diego Essent Pharmaceuticals, backed by venture capital.Last week, the company Report the results of the first stage It shows that its small molecule EP547 is safe and can be well tolerated by healthy volunteers and patients with chronic liver or kidney disease.
Pamela Stephenson, Albireo’s chief commercial officer, said that Bylvay was administered by weight. The average weight of the patients in the clinical study was 18 kg (approximately 39.6 lbs). Based on an average weight of 18 kg, the average annual price of Bylvay is $385,000 before any rebates or discounts. The drug will be provided in bottled form, each bottle can be supplied for 30 days. Stephenson said that the price of each bottle will be announced later.
With Bylvay’s approval, Albireo also received a rare disease priority review certificate. These coupons allow the company to get priority reviews of future drug candidates more quickly. But companies can also sell these vouchers for up to $100 million. Chief Financial Officer Simon Harford said the company plans to sell its priority review coupons, but the timing and price of the sale will depend on the market and the number of companies that want to buy such coupons.
Albireo reported that as of the end of June, its cash position was $186.3 million. Excluding any proceeds from the sale of priority review vouchers, the company estimates that its cash will support the company by 2023.
Albireo is also studying Bylvay to treat other rare liver diseases that affect children. Two phase 3 studies are ongoing, one for biliary atresia and the other for Alagille syndrome.
Photo by Albireo Pharma
