Gothenburg, Sweden, July 24, 2012– Albireo AB (Gothenburg, Sweden; “Albireo”), a biopharmaceutical company specializing in gastroenterology, today announced that the European Medicines Agency Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on an application for orphan medicinal product status for the company’s lead hepatology candidate, A4250, for the treatment of:
• Primary Biliary Cirrhosis (PBC)
• Progressive Familial Intrahepatic Cholestasis (PFIC)
• Alagille Syndrome
The positive opinion of the COMP has now been forwarded to the EU commission for final approval. The designation would allow Albireo ten years of marketing exclusivity in EU member countries after obtaining market authorization, as well as streamlined regulatory review processes and registration.
“We are very pleased with the EMA´s recognition of our technology with A4250 as an orphan medicinal product for the treatment of these liver diseases in Europe”, said Dr. Hans Graffner, Chief Medical officer at Albireo. “This designation is an important milestone in our efforts to provide a treatment for patients with cholestatic liver diseases; a disease entity often leading to severe symptoms, liver transplantation and with reduced survival. A4250 is an inhibitor of the bile acid transport mechanism and will decrease the toxic levels of bile acids in the liver cells.”
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About Primary Biliary Cirrhosis (PBC)
PBC is a slowly progressive autoimmune disease of the liver, primarily affecting women; average age when symptoms start is around 40-50 years of age. It is characterized by destruction of bile ducts resulting in an increased bile acid concentration in the liver inducing inflammation and cirrhosis. There are more than 100.000 patients with PBC in Europe. Main symptoms are fatigue, severe itching and symptoms of cirrhosis. There is no cure for PBC, some therapeutic alternatives may slow disease progression and relieve symptoms but some patients may need liver transplantation.
About Progressive Familial Intrahepatic Cholestasis (PFIC)
PFIC is a rare disease (estimated prevalence at birth 1/50.000 corresponding to more than 10.000 patients in the European Union) caused by a genetic defect impairing the transport of bile acids thereby inducing toxic levels of bile acid products in the liver inducing severe symptoms such as itching and scarring of the liver (cirrhosis) early in life. Although milder forms exist, most patients will develop symptoms in early childhood. Without any treatment, PFIC will lead to cirrhosis by age 10-20 years. In addition to supportive measures, the most common therapeutic modality is a surgical procedure thereby a portion of the bile production is either diverted to a stoma bag or by intestinal bypass to the large bowel. If the patient does not get better, or if there is evidence of liver cancer, then liver transplantation may be needed.
About Alagille Syndrome
Alagille syndrome is an inherited disease in which the patient has fewer than the normal number of bile ducts leading to increased concentration of bile acids in the liver which induce damage to the liver cells. The estimated number of patients with Alagille syndrome in the European Union is approximately 10.000-20.000. Jaundice, severe itching and growth problems are caused by the liver failure. Approximately 75% of the children diagnosed with Alagille syndrome live to 20 years of age; deaths most often caused by liver failure or heart complications.
A4250 belongs to a class of inhibitors of the ileal bile acid transporter (IBAT, syn. apical sodium-dependent bile acid transporter ASBT). Usually bile excreted into the small bowel is being reused by a transport mechanism in which bile acids are absorbed in the distal part of the small bowel. A4250 decreases this re-absorption and will reduce the toxic levels of bile acids in the diseases described above. By using a specialized delivery technology, the bile acids will be neutralized in the large bowel.
Albireo AB is a Swedish biotechnology company focused on the development of novel therapeutic alternatives in the gastrointestinal area. In addition to A4250, Albireo has a pipeline of drug candidates for the development in areas such as IBD and functional gastrointestinal diseases. The lead program, elobixibat, is ready to enter Phase III in Chronic constipation and is also in development for IBS with constipation. Elobixibat is partnered with Ferring and with Ajinomoto. The Albireo management team has a broad experience in drug development, in particular in the GI area and has an extensive network in the international scientific and clinical communities. Albireo was created as a spin-out of AstraZeneca in 2008, financed by a syndicate of leading healthcare investors, led by Phase4 Ventures, and joined by TPG Biotech, TVM Capital and Scottish Widows Partnership.