The company Orchard Therapeutics (Netherlands) BV has stated its willingness to participate in a joint assessment of the autologous gene therapy Libmeldy (atidarsagene autotemcel) within the framework of the Beneluxa initiative. Intensive consultations have been going on between the consortium and the company since then.
The European Commission granted the marketing authorisation to this orphan drug for the treatment of patients with the rare condition of metachromatic leukodystrophy (MLD) in December 2020. It is authorised for children with late infantile or early juvenile forms of the disease who have not yet developed symptoms; or for those with early juvenile MLD who have initial symptoms but can still walk independently and before the onset of cognitive decline.
In December 2021, the company submitted a dossier of clinical and pharmacoeconomic evidence to support reimbursement in Belgium, Ireland and the Netherlands. As a consequence, HTA agencies in these three countries are diligently carrying out the health technology assessment of Libmeldy. Based on the outcome of the HTA assessment, involved parties will determine if the joint assessment could be followed by a joint price negotiation.
According to national regulations, the end of this assessment procedure is scheduled for June 2022, but these timelines do not take into account possible clock stops at different times during the procedure.
Last update: 22 February 2022