Albireo Receives Positive Opinion for Orphan Drug Designation for A4250 for Severe Liver Diseases
GOTHENBURG, Sweden, July 24, 2012 /PRNewswire/ –
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.”
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
SOURCE Albireo AB