On 17 October 2019, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Kalydeco. The marketing authorisation holder for this medicinal product is Vertex Pharmaceuticals (Ireland) Limited.
The CHMP recommended approval of a new 25-mg strength, for Kalydeco granules, and an extension to the existing indication to allow use in infants from 6 months of age and weighing from 5 kg to less than 25 kg.
For information, the full indication for Kalydeco granules will be as follows:*
'Kalydeco granules are indicated for the treatment of
children with cystic fibrosis (CF)infants aged 12 at least 6 months, toddlers and older and children weighing 75 kg to less than 25 kg with cystic fibrosis (CF) who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R (see sections 4.4 and 5.1).'
Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published in the revised European public assessment report (EPAR), and will be available in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.
*New text in bold, deleted text in strikethrough
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This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.
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Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 14-17 October 201918/10/2019
Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 15-18 October 201819/10/2018
Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 21-24 September 201525/09/2015