Biogen: Higher Spinraza dose aids spinal muscular atrophy patients
Biogen has announced promising results from a clinical trial for a higher dose of its spinal muscular atrophy (SMA) drug, Spinraza. The experimental regimen, which more than doubles the current dosage, has shown significant improvements in motor function among infants participating in the trial. The company is now planning to seek FDA approval for this new, higher dose of Spinraza.
The clinical trial compared the experimental higher dose of Spinraza to a historical control group that was part of the studies leading to the drug's initial approval in 2016. According to Biogen, the higher dose regimen significantly enhanced motor function in infants, as measured by the CHOP-INTEND scale at six months (p<0.0001). This scale is a standard tool used to assess motor skills in infants with SMA.
However, the exact comparison between the experimental dose and the currently approved dose remains unclear. Biogen has stated that data trends favor the higher dose but has not confirmed whether the secondary endpoints were met in this trial. Additionally, the company reported that serious adverse events were less frequent in the higher-dose regimen compared to the 12 mg dose group (60% vs. 72%).
The trial data and comprehensive results are expected to be presented at an upcoming medical conference, where more details will likely be shared.
Spinraza, currently priced at $142,000 for a 12 mg dose/5 ml vial, represents a significant investment for patients and healthcare systems. Biogen has not provided any pricing information for the higher dose regimen. Keeping the price per milligram constant, the new regimen would be considerably more expensive, costing more than twice the current regimen in the first year and annually thereafter.
The Phase 2/3 DEVOTE study, pivotal to this advancement, included 75 children with infantile-onset SMA who had not received any prior treatment. The trial participants were randomized in a 2-to-1 ratio to receive either the experimental higher-dose regimen or the standard approved dose.
The experimental higher-dose regimen involves two initial doses of 50 mg administered 14 days apart, followed by a maintenance dose of 28 mg every four months. In contrast, the currently approved regimen consists of 12 mg doses, beginning with four loading doses before switching to maintenance doses every four months.
Biogen's announcement highlights the potential for this higher dose of Spinraza to offer improved outcomes for infants suffering from SMA, a severe and often life-threatening condition. If approved, the new regimen could represent a significant advancement in the treatment of this debilitating disease, although the cost implications for patients and healthcare providers will need to be carefully considered.
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