CAMBRIDGE, Mass.–(BUSINESS WIRE)–Biogen (NASDAQ: BIIB) today announced that its New Drug Application (NDA) for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), has been accepted by the U.S. Food and Drug Administration (FDA) for Priority Review, and that the company’s Marketing Authorization Application (MAA) has been validated by the European Medicines Agency (EMA). Nusinersen had previously been granted Accelerated Assessment status by the EMA’s Committee for Medicinal Products for Human Use (CHMP). The regulatory review process for these applications has now been initiated in the U.S. and EU. Both the Priority Review and Accelerated Assessment designations can reduce the standard review time. If approved, nusinersen would be the first therapy for SMA, a leading genetic cause of infant mortality.

Biogen intends to market nusinersen under the brand name SPINRAZA.This name has been conditionally accepted by the FDA and the CHMP and will be confirmed upon approval.

“The FDA and EMA have acknowledged the potential for nusinersen to address the urgent need for an effective SMA treatment by granting special status to the applications, and FDA has shared that they plan to act early on our NDA under an expedited review,” said Michael Ehlers, M.D., Ph.D., executive vice president, head of Research and Development at Biogen. “We are now focused on working with the agencies to hopefully bring this investigational treatment to the SMA community as quickly as possible.”

The regulatory filing packages in the U.S. and EU are based on data that demonstrate the clinically meaningful efficacy and favorable safety profile of nusinersen from multiple studies. These include the results from the interim analysis of ENDEAR, the Phase 3 study evaluating nusinersen in infantile-onset (most likely to develop Type 1) SMA, as well as open-label data in other patient populations. The ENDEAR interim analysis demonstrated that infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones compared to those who did not receive nusinersen. Data from the other endpoints analyzed were also consistently in favor of the treated infants. Nusinersen was generally well-tolerated, with a favorable safety profile. No adverse events (AEs) were considered related to nusinersen.

Infants benefit from Nusinersen/Spinraza in Spinal Muscular Atrophy

– Initial Phase 3 Safety Data Presented in Infantile-onset Spinal Muscular Atrophy Demonstrated Favorable Safety Profile with no Treatment Related Adverse Events

CAMBRIDGE, Mass. & CARLSBAD, Calif.–(BUSINESS WIRE)–New data from the clinical program for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), were presented by Biogen (NASDAQ: BIIB) and Ionis Pharmaceuticals (NASDAQ: IONS) in the late-breaking session at the 2016 World Muscle Society Congress in Granada, Spain. The presentations included safety results from the interim analysis of the Phase 3 ENDEAR study in infantile-onset SMA (most likely to develop Type 1), encouraging preliminary results from NURTURE, a Phase 2 open-label study in pre-symptomatic infants, and a recent analysis of the ongoing Phase 2 open-label study in patients with later-onset SMA (consistent with Types 2 or 3).

“We continue to be encouraged by the consistently positive results with nusinersen across our clinical program, including our first data in infants treated before they show signs of the disease,” said Wildon Farwell, senior director SMA clinical development at Biogen. “NURTURE is the first study to evaluate an investigational therapy in pre-symptomatic infants genetically at risk for SMA. In this analysis, infants treated for up to one year achieved motor milestones in timelines more consistent with normal development than what is observed in the natural history of patients with Type 1 SMA.”

Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen. Submission of the Marketing Authorization Application to the European Medicines Agency is planned in the next week. Biogen will initiate regulatory filings in other countries in the coming months.

Interim Data from Phase 3 ENDEAR Trial
In the interim analysis of the controlled, Phase 3 ENDEAR study in infantile-onset SMA, infants treated with nusinersen demonstrated a statistically and clinically significant improvement in the primary endpoint (p<0.0001), defined as the proportion of motor milestone responders as measured by the Hammersmith Infant Neurological Examination (HINE). A responder was defined as a patient who improved in more motor milestone categories (kicking, head control, rolling, sitting, crawling, standing, walking) than worsened.

Nusinersen was generally well-tolerated, with an acceptable safety profile. No adverse events (AEs) were considered related to treatment. Based on the results of the positive interim analysis, patients who elect to do so will be transitioned to SHINE, an open-label extension study, in which they will be able to receive nusinersen. Following the transition of patients, the ENDEAR study will close. Detailed efficacy data will be presented at a future medical conference once participants complete their final study visit in ENDEAR.