Biogen Plans Resubmission After FDA Declines High-Dose Spinraza for SMA, Requests More Production Information

Biogen received a Complete Response Letter (CRL) from the FDA for its supplemental New Drug Application (sNDA) seeking approval of a high-dose regimen of nusinersen (Spinraza) for treating spinal muscular atrophy (SMA)12.

The FDA did not cite any deficiencies in the clinical data submitted for the high-dose regimen, but requested updated technical information in the Chemistry Manufacturing and Controls (CMC) module126.

The requested information is related to production and manufacturing, rather than efficacy or safety of the drug236.

Biogen stated the outcome was unexpected and plans to resubmit the application as soon as the required information is ready12.

The higher-dose regimen includes a faster loading phase (two 50 mg doses 14 days apart) and higher maintenance dosing (28 mg every 4 months) compared to the currently approved regimen (12 mg/5 mL per injection)1.

The high-dose regimen of Spinraza is already approved in Japan and is under review by the European Medicines Agency (EMA) and other regulatory bodies2.

Biogen remains committed to advancing the high-dose regimen globally and is actively working to address the FDA's requests12.

Sources:

1. https://www.neurologylive.com/view/fda-gives-complete-response-letter-higher-dose-sma-therapy-nusinersen

2. https://www.quiverquant.com/news/Biogen+Inc.+Receives+Complete+Response+Letter+from+FDA+for+Nusinersen+High+Dose+Regimen+in+Spinal+Muscular+Atrophy

3. https://firstwordpharma.com/story/6095275

6. https://www.stocktitan.net/news/BIIB/biogen-provides-regulatory-update-on-high-dose-regimen-of-7uig0vff5rfz.html