Biogen has submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for nusinersen as a treatment for SMA.
New data from the clinical program for nusinersen were presented at the World Muscle Society Congress.
A marketing authorisation application to the European Medicines Agency for nusinersen
In a statement sent to associations of people with proximal SMA linked to SMN1, Biogen has announced that on 7 October 2016 it submitted an MAA to the EMA for nusinersen as a treatment for SMA.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) had already agreed to an accelerated assessment, which reduces the time taken to examine the dossier from 7 to 5 months.
Meanwhile, the nusinersen expanded access program (EAP) in France is via a nominative temporary use authorisation (TUA).
Presently, early access concerns Type 1 SMA. The MAA does not correspond to a precise indication to date; It will be decided by the regulatory agencies of the countries concerned.
Nusinersen results presented at the World Muscle Society Congress
Several preliminary results concerning nusinersen were presented at the World Muscle Society (WMS) International Congress which took place from 4 to 8 October 2016 in Granada, Spain.
Interim data from the phase 3 ENDEAR study in infantile-onset SMA (less than 7 months of age) showed a statistically significant improvement in motor function in nusinersen-treated infants compared with placebo-treated infants.
Preliminary data from the NURTURE trial, which included pre-symptomatic newborns under 6 weeks of age, demonstrated an improvement in motor function in participants treated with nusinersen. No participant has required respiratory assistance at this stage of the study.
Additional data on the ongoing nusinersen open-label phase II trial in 28 patients with Type 2 or 3 SMA showed increased motor function measurements during follow-up of approximately 3 years.
> See the Biogen press release sent to associations of people with SMA
> See the press release on the results presented at the World Muscle Society Congress
