The European Commission (EC) has granted marketing authorization for the first treatment for 5q spinal muscular atrophy (SMA) in the European Union, according to a statement from the manufacturer.
The injectable antisense oligonucleotide nusinersen (Spinraza, Biogen), which was approved by the US Food and Drug Administration in December 2016, was designed to increase production of survival motor neuron protein in patients with SMA.
“Based on the robust efficacy and safety profile demonstrated in the clinical trials, we believe Spinraza will have a meaningful impact on infants, children, and adults living with this devastating disease,” said Michel Vounatsos, chief executive officer of Biogen, in a statement.
The approval was based largely on results from the ENDEAR trial, which assessed infantile-onset SMA, and the CHERISH trial, which examined later-onset SMA.
“The overall clinical findings support the efficacy and safety…in a broad range of individuals with SMA, including significant improvements in motor development and reduction in risk of deaths in infants,” said Jan Kirschner, MD, professor at the Medical Center University of Freiburg, Germany, in the same press release.
“These unprecedented improvements bring new hope to a community where there previously were no approved treatments available,” added Dr Kirschner.
The drug, which is administered to cerebrospinal fluid via intrathecal injection, was reviewed under the European Medicines Agency’s (EMA;s) accelerated assessment program. Biogen announced that availability “will vary by country, per local reimbursement and access pathways.”
More Filings to Come
As reported by Medscape Medical News, interim results from the phase 3 CHERISH trial were reported at the American Academy of Neurology annual meeting in April. In 126 patients who were aged 2 to 12 years, those who received four doses of 12 mg nusinersen over 9 months had significantly greater improvements on motor function measures than those who received a sham procedure.
They also had more improvements in upper-limb function and a greater number of new motor milestones achieved.
The week before the presentation, the EMA Committee for Medicinal Products for Human Use recommended approval for the treatment based on CHERISH and the positive ENDEAR trial.
In the latter study, 51% of the infants receiving nusinersen met the primary motor milestone outcome compared with none of those receiving the sham procedure (P < .0001). In addition, the active treatment group had a significant reduction in risk for death or permanent ventilation (P = .005).
The manufacturer noted that the EC approval was also based on “open-label data in pre-symptomatic and symptomatic individuals with, or likely to develop, Types 1, 2, and 3 SMA.”
Regulatory filings have also been submitted in Canada, Japan, Australia, Brazil, and Switzerland, with plans to “initiate additional filings in other countries in 2017,” Biogen reports.
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