Committee for medicinal products for human use (CHMP)

Scope: Oral explanation
Action: Oral explanation to be held on Wednesday 09 November 2016 at time 14:00
List of Outstanding Issues adopted on 15.09.2016. List of Questions adopted on 28.04.2016.
BWP report
2.1.4. aceneuramic acid - Orphan - EMEA/H/C/004176
Ultragenyx UK Limited; treatment of Hereditary Inclusion Body Myopathy (HIBM)
Scope: Oral explanation
Action: Oral explanation to be held on Tuesday 08 November 2016 at time 14:00
List of Outstanding Issues adopted on 15.09.2016. List of Questions adopted on 28.01.2016.
2.2. Re-examination procedure oral explanations
2.3. Post-authorisation procedure oral explanations
2.3.1. Translarna - ataluren - Orphan - EMEA/H/C/002720/II/0020 &
EMEA/H/C/002720/R/0022
MAH: PTC Therapeutics International Limited,
Rapporteur: Johann Lodewijk Hillege, Co-Rapporteur: Concepcion Prieto Yerro, PRAC
Rapporteur: Sabine Straus,
Scope: Oral explanation to be held on Tuesday 08 November 2016 at time 16:00
Type II variation
“Update of sections 4.4, 4.6, 4.7, 4.8, and 5.1 of the SmPC and Annex II in order to reflect the
result from the submitted study TC124-GD-020-DMD object of SOB 001. The Package Leaflet
and the RMP are updated accordingly. In addition, the Marketing authorisation holder (MAH)
took the opportunity to include some minor editorial changes throughout the Product
information.”
Oral explanation was held on 11 October 2016. Request for Supplementary Information
adopted on 21.07.2016, 01.04.2016.
Renewal of Marketing Authorisation
Action: For adoption
Participation of patients’ representatives
See 9.1.2
Committee for medicinal products for human use (CHMP)
EMA/CHMP/682760/2016 Page 8/30