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New HTA Decisions in France
June 2020
Drug name
TRANSLARNA® (ataluren)
Company
PTC Therapeutics France
Decision date
23/10/2019
Therapeutic area
Genetic conditions
Therapeutic sub area
Genetic conditions: general and other
Official notice date
18/11/2019
Reimbursement %
15
Decision (SMR)
Weak
Decision (ASMR)
No improvement (V)
Indication
TRANSLARNA® is indicated for the treatment of Duchenne muscular dystrophy, resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years or older.
Decision Type
Extension of indication
Summary
The committee considered the actual benefit of TRANSLARNA® to be 'low' in the indication extension of the Marketing Authorisation. Considering: - efficacy results based on the evaluation after 28 weeks of treatment of clinical endpoints that were secondary endpoints from an open, non-comparative phase II study; - the small magnitude of the evolutions observed on the different functional clinical criteria, but with a homogeneous evolution of these criteria suggesting a slight improvement with TRANSLARNA®; - limitations of interpretation of these results given the absence of a control group; - the difficulty of interpreting the perspective of the results of the phase II study with those of a historical cohort; - the tolerance profile of TRANSLARNA® that appears favourable; - the poorly covered medical need identified; it was concluded that TRANSLARNA®, in combination with corticosteroids and supportive care, does not provide any improvement in the medical service rendered (ASMR V) in the management of Duchenne muscular dystrophy, resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 to 5 years. The committee proposed 15% reimbursement.