Following the referral of idebenone for Duchenne muscular dystrophy to NICE by the Department for Health and Social Care, the company have now confirmed that following a re-examination by the Committee for Medicinal Products for Human Use (CHMP), at the company’s request, a further refusal of the marketing authorisation was given on 26 January 2018. Therefore, the planned appraisal of idebenone for treating Duchenne muscular dystrophy has been suspended on the technology appraisals work programme. For information, during September 2017, the CHMP adopted its first negative opinion, recommending the refusal of the marketing authorisation for idebenone. NICE note that the company remains committed to the development and introduction of idebenone for patients with Duchenne muscular dystrophy and that the company will continue their dialogue with the regulators to define a clear path forward for idebenone in this indication. As this appraisal has been referred, NICE will continue to monitor any developments and provide an update if the situation changes.
 
Status Suspended
Process TAG

Timeline

Key events during the development of the guidance:

Date Update
22 February 2018 Suspended, Following the referral of idebenone for Duchenne muscular dystrophy to NICE by the Department for Health and Social Care, the company have now confirmed that following a re-examination by the Committee for Medicinal Products for Human Use (CHMP), at the company’s request, a further refusal of the marketing authorisation was given on 26 January 2018. Therefore, the planned appraisal of idebenone for treating Duchenne muscular dystrophy has been suspended on the technology appraisals work programme. For information, during September 2017, the CHMP adopted its first negative opinion, recommending the refusal of the marketing authorisation for idebenone. NICE note that the company remains committed to the development and introduction of idebenone for patients with Duchenne muscular dystrophy and that the company will continue their dialogue with the regulators to define a clear path forward for idebenone in this indication. As this appraisal has been referred, NICE will continue to monitor any developments and provide an update if the situation changes.
10 March 2017 (10:00) Scoping workshop (London)
19 January 2017 - 16 February 2017 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

For further information on how we develop guidance, please see our page about NICE technology appraisal guidance