Date: 2017-05-16
Type of information: Granting of a patent
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.
A4250 has been granted orphan drug designation for PFIC in the United States and the European Union and has been granted access to the PRIority MEdicines (PRIME) program of the European Medicines Agency (EMA) for the treatment of progressive familial intrahepatic cholestasis.
Company: Albireo Pharmaceuticals (USA - MA) Albireo (Sweden)
Disease: progressive familial intrahepatic cholestasis
Latest news:
- • On June 13, 2012, the Committe 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 orphan medicinal product for treatment of progressive familial intrahepatic cholestasis.
Patents:
- • On May 16, 2017, Albireo Pharma announced that the United States Patent and Trademark Office (USPTO) has allowed U.S. Patent Application No. 15/276,446, "IBAT inhibitors for the treatment of liver diseases" (‘446 patent). The ‘446 patent claims a method of treatment for Albireo's lead product candidate A4250 in progressive familial intrahepatic cholestasis and other specified liver diseases. Upon issuance, the regular term of the ‘446 patent will expire in November 2031 . Albireo plans to initiate a Phase 3 clinical program for A4250 in progressive familial intrahepatic cholestasis patients in the second half of 2017.
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:
Orphan status UE: 2012-07-17
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
