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Spinraza Approval History

Reviewed on Dec 23, 2016 by J.Stewart BPharm
  • FDA approved: Yes (First approved December 23rd, 2016)
  • Brand name: Spinraza
  • Generic name: nusinersen
  • Dosage form: Injection
  • Company: Biogen
  • Treatment for: Spinal Muscular Atrophy

Spinraza (nusinersen) is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

FDA approval of Spinraza was based on positive results from multiple clinical studies, including the ENDEAR clinical study of 121 patients with infantile-onset SMA. Patients were randomized 2:1 to receive Spinraza via intrathecal injection, or a placebo via a mock procedure without drug injection. Forty percent of patients treated with Spinraza achieved improvement in motor milestones (head control, sitting, ability to kick in supine position, rolling, crawling, standing and walking) compared to none of the patients who received the placebo.

Spinraza is administered via intrathecal bolus injection into the cerebrospinal fluid (CSF) by a medical professional experienced in performing lumbar punctures. Doses are administered over 1 to 3 minutes every 14 days for the first three doses, the fourth dose at 30 days after the third dose, then once every four months thereafter.

Common side effects reported during clinical trials include upper respiratory infection, lower respiratory infection and constipation. Spinraza may increase the risk of bleeding and renal toxicity, and laboratory tests are required prior to each dose to monitor safety.

Development History and FDA Approval Process for Spinraza

Dec 23, 2016Approval FDA Approves Spinraza (nusinersen) for Spinal Muscular Atrophy
Oct 28, 2016Biogen’s Regulatory Applications for Nusinersen as a Treatment for Spinal Muscular Atrophy Accepted by FDA and EMA
Sep 26, 2016Biogen Completes Rolling Submission of New Drug Application to FDA for Nusinersen as a Treatment for Spinal Muscular Atrophy

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