US-based biopharmaceutical company Biogen (NASDAQ: BIIB) has submitted supplemental New Drug Applications (sNDAs) to the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for a higher-dose regimen of its spinal muscular atrophy (SMA) treatment, Spinraza (nusinersen). The applications are currently under review by both regulatory bodies, with the aim of expanding treatment options for patients with SMA.
Higher-Dose Regimen Details
The proposed higher-dose regimen of nusinersen includes a more rapid loading phase, with two 50 mg doses administered 14 days apart, followed by a higher maintenance dose of 28 mg every four months. This contrasts with the currently approved regimen, which features a lower maintenance dose of 12 mg. The new dosing schedule is designed to provide more rapid therapeutic effects and sustained benefits for patients with SMA.
Clinical Study Results
A Phase II/III study evaluating the safety, tolerability, pharmacokinetics, and efficacy of the higher-dose nusinersen regimen demonstrated promising results. The study showed that the higher-dose treatment rapidly reduced neurofilament levels, a biomarker associated with nerve damage, in patients with different phenotypes of SMA. Importantly, the safety profile of the higher-dose regimen was found to be comparable to that of the currently approved 12 mg treatment regimen, indicating no significant additional risks.
Implications for SMA Treatment
If approved, the higher-dose regimen of Spinraza could offer enhanced therapeutic benefits for patients with spinal muscular atrophy, potentially improving outcomes and quality of life. The submission to both the FDA and EMA underscores Biogen’s commitment to advancing SMA treatments globally and addressing the evolving needs of patients and healthcare providers.-Fineline Info & Tech
Leave a Reply