Products

Date: 2012-07-23

Type of information: Granting of the orphan status in the US

Product name: A4250

Compound: (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro- 1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid

Therapeutic area: Hepatic diseases - Liver diseases - Rare diseases

Action mechanism: ileal bile acid transporter (IBAT) inhibitor. The product belongs to a class of inhibitors of the ileal bile acid transporter. 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.

Company: Albireo (Sweden)

Disease: primary biliary cirrhosis

Latest news:

• • On November 8, 2012, Albireo announced that A4250 has been granted orphan-drug designation by the FDA for the treatment of primary biliary cirrhosis.
• • On July 11, 2012, the Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro- 1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4 hydroxyphenyl)acetyl]amino}butanoic acid for designation as an orphan medicinal product for treatment of primary biliary cirrhosis.

Patents:

Submission of marketing authorization application USA :

Submission of marketing authorization application UE:

Withdrawal of marketing authorization application USA:

Withdrawal of marketing authorization application UE:

US authorization:

UE authorization:

Favourable opinion UE:

Favourable opinion USA:

Orphan status USA: 2012-10-31

Orphan status UE: 2012-08-09

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes