News 
Market Data 
Discover
Replies to post #174171 on Biotech Values
On 23 January 2014, the Committee for Medicinal Products for Human Use (CHMP) adopted a revised positive opinion, recommending the granting of a marketing authorisation for the medicinal product cholic acid FGK, 50 and 250 mg capsule intended for the treatment of inborn errors in primary bile acid synthesis due to Sterol 27-hydroxylase (presenting as cerebrotendinous xanthomatosis, CTX) deficiency, 2- (or a-) methylacyl-CoA racemase (AMACR) deficiency or Cholesterol 7a-hydroxylase (CYP7A1) deficiency in infants, children and adolescents aged 1 month to 18 years and adults.
The Committee for Medicinal Products for Human Use (‘CMPHU’), a part of the EMA, issued a positive opinion, followed by a revised opinion, which was also positive, in December 2010 and April 2011 respectively, recommending that an MA be granted. However, in July 2011 the Commission submitted to the Standing Committee on Medicinal Products for Human Use (‘the Standing Committee’) a draft decision refusing to grant CTRS an MA for Orphacol. In October 2011, the Standing Committee issued a negative opinion on the Commission’s draft decision refusing to grant an MA. In the same month, the Commission submitted the draft decision refusing to grant an MA to the Appeal Committee. In November 2011, the Appeal Committee also issued a negative opinion on the Commission’s draft decision refusing to grant an MA.
On 12 January 2012, CTRS brought an action before the General Court seeking a declaration that the Commission had failed to act in unlawfully failing to adopt a final decision in relation to its application for an MA for the medicinal product Orphacol and, in the alternative, applying for the annulment of the decision, allegedly contained in a letter of 5 December 2011 from the Commission, refusing to grant that MA.
By its judgment delivered today, the General Court annuls the Commission’s decision of 25 May 2012 refusing to grant the MA for the medicinal product Orphacol.
During the assessment of cholic acid FGK, on 13 September 2013, the European Commission granted a
marketing authorisation for the orphan medicinal product Orphacol, for which the marketing
authorisation holder is Laboratoires CTRS, containing the same active substance cholic acid. In
accordance with Articles 8(1) and 8(3) of Regulation (EC) No 141/2000, the applicant submitted a
report on similarity of cholic acid FGK to Orphacol and a report claiming the derogation of Orphacol’s
market exclusivity based on clinical superiority to Orphacol. The CHMP considered that cholic acid FGK
was similar to Orphacol with respect to two of the five therapeutic indications claimed i.e. 3ß-HSD and
?4-3-oxoR deficiencies. The CHMP also concluded that cholic acid FGK was not considered safer, more
effective or clinically superior to Orphacol. Following the outcome of the clinical superiority report, the
applicant withdrew from the application the indications for 3ß-HSD and ?4-3-oxoR deficiencies. The
scope of the application was reduced to the following 3 indications: CTX, AMACR and CYP7A1
deficiencies.
