dm+d

29024111000001106

Lactation Safety Information

Yes
-
No published evidence of safety
Naturally occurring primary bile acid
Used in full-term neonates from birth
12 May 2017

New Medicines

Kolbam (EU), Cholbam (US)Inborn error of metabolism in primary bile acid synthesis due to deficiencies of selected liver enzymes

Information

Kolbam (EU), Cholbam (US)
New formulation
Retrophin
Asklepion Pharmaceuticals LLC

Development and Regulatory status

Licence withdrawn
Licence withdrawn
Launched
Yes
Yes
Jul 20Travere withdraws the EU marketing authorisation for Kolbam as sales of Kolbam in Europe were not a material component of its business [7].
Jan 19NICE is helping to develop an NHS England specialised clinical commissioning policy for cholic acid. A scope was published in Jan 18 [5].
Jul 18In the EU, Retrophin plans to continue to partner with local distributors and certain field based personnel as necessary to conduct permitted commercial activities. Near-term efforts are focused on securing pricing and reimbursement approval for Kolbam [6].
Jun 17Nil in corporate overview re plans for EU launches [4].
Oct 15Kolbam granted a marketing authorisation under exceptional circumstances in the EU [3].
Sep 15If approved, Kolbam can be used in infants, children and adolescents aged 1 month to 18 years and adults [1].
Sep 15The CHMP adopts a revised opinion, recommending granting of a marketing authorisation for Kolbam, intended for treatment of inborn errors in primary bile acid synthesis due to deficiencies of the following liver enzymes: sterol 27-hydroxylase (presenting as cerebrotendinous xanthomatosis, CTX), 2- (or α-) methylacyl-CoA racemase (AMACR) or cholesterol 7α-hydroxylase (CYP7A1) [1].
Sep 15The European Commission requested the CHMP to review its previous scientific opinion [1].
Sep 15The General Court decision was based on inclusion of statements in the Kolbam SmPC and assessment report pertaining to efficacy in the treatment of deficiencies in 3β-HSD and Δ4-3-oxoR, which Orphacol, Laboratoires CTRS´ version of cholic acid, had already received approval and market exclusivity for in Sep 2013. The General Court ruled that inclusion of these statements could circumvent Orphacol’s market exclusivity because they are liable to facilitate prescribing of Kolbam off-label [2].
Jun 15The marketing authorisation of Kolbam in the EU was annulled following a judgement of the General Court [2].
Apr 15Launched in US [2].
Mar 15Retrophin exercises the exclusive right to purchase all worldwide rights, titles and ownership of cholic acid from Asklepion for treatment of bile acid synthesis defects [2].
Mar 15Approved in US for inborn genetic disorders [2].
Jan 15Filed in US for inborn genetic disorders in children who are treatment-experienced [2].
Apr 14Approved in EU [2].
Jan 14EMA´s CHMP recommends revised approval of cholic acid FGK for Inborn error metabolic disorders (in primary bile acid synthesis due to CTX, AMACR or CYP7a1 deficiencies) [2].
Nov 13Filed in EU & US [2].
Oct 09FGK Representative Service GmbH receives orphan drug status for Inborn genetic disorders (inborn errors in primary bile acid synthesis) in EU [2].
Jul 03Asklepion receives Orphan Drug status for Inborn error metabolic disorders in USA [2].

Category

A bile acid preparation. Orally administered cholic acid acts to replace the missing endogenous primary bile acids both in terms of their physiological functions and their metabolic regulation. Available in 50mg and 250mg capsules.
Inborn errors in primary bile acid synthesis responsive to treatment with cholic acid affect ~0.07 in 10,000 people in the EU equivalent to a total of around 4,100 people [1].
Inborn error of metabolism in primary bile acid synthesis due to deficiencies of selected liver enzymes
Oral

Trial or other data

Oct 09No studies have yet been conducted [2].

Evidence based evaluations