Yesterday's All Party Parliamentary Group (APPG) for Muscular Dystrophy meeting organised by the Muscular Dystrophy Campaign gave MPs and Peers the opportunity to discuss last month's announcement that the European Medicines Agency is recommending that Duchenne muscular dystrophy drug Ataluren (now to be known as Translarna) receives conditional approval.
Baroness Thomas of Winchester, a Trustee of the Muscular Dystrophy Campaign, and Mary Glindon MP, jointly chaired the session at which a panel of experts from the Medicines and Healthcare products Regulatory Agency and NHS England were challenged by parliamentarians on ensuring that Translarna is approved and commissioned for availability to patients as soon as possible.
The panel included:
Dr Siu Ping Lam, Director of Licensing, Medicines and Healthcare Products Regulatory Agency
Greg Markey, UK MHRA Representative on the Committee for Medicinal Products for Human Use (CHMP), European Medicines Agency
Paul McManus, Lead Pharmacist - specialised services (South Yorkshire and Bassetlaw), NHS England
On 23 May the European Medicines Agency announced that it will recommend that Duchenne muscular dystrophy drug ataluren receives conditional approval. The next step will be for the European Commission to review this decision. Ataluren has been developed by PTC Therapeutics to overcome a specific change in the DNA called a nonsense mutation which causes 10-15% of cases of Duchenne muscular dystrophy. Translarna, as the drug will now be known will be the first ever drug to treat an underlying genetic cause of Duchenne muscular dystrophy in children and young people affected, outside of a clinical trial.
Translarna may have the potential to treat around 200 individuals with Duchenne muscular dystrophy in the UK (of a total of around 2,400). The next step for the drug will be for the European Commission to decide whether to grant a conditional licence (which normally takes approximately three months).
an overview of the conditional approval announcement for Translarna
the extent of the licence for Translarna
the process for accelerated review in Europe for drugs proceeding to accelerated approval in the USA
the new Early Access to Medicines Scheme and adaptive licensing pilot project
cost assessment and funding for the treatment
involvement of Clinical Reference Groups
Following the conclusion of the meeting, the Muscular Dystrophy Campaign held a meeting with the National Institute for Care Excellence (NICE) to establish how they might be involved in the assessment for Translarna in providing an evidence summary assessment.
The next stage of the process will be ensuring a 'commissioning policy' is set up by NHS England over the next few months with a health economic assessment carried out, and the Muscular Dystrophy Campaign will be meeting with NHS England on Friday to discuss the timeframes for Translarna and the details for the 'commissioning policy'.
For more information and to get involved in the Muscular Dystrophy Campaign's faster access to treatments campaign, please get in touch on 020 7803 4838 or by email at firstname.lastname@example.org
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