Investing.com — Shares of Ionis Pharmaceuticals (NASDAQ:) climbed 7% today after the company announced that regulatory agencies in the United States and Europe have accepted applications for a higher dose regimen of its spinal muscular atrophy (SMA) drug, nusinersen.
The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are now reviewing the supplemental New Drug Application (sNDA) for nusinersen, which includes a more rapid loading regimen and a higher maintenance dose than the currently approved treatment. The announcement marks a potential advancement for SMA care and has been positively received by the market.
Biogen Inc. (NASDAQ:), which licensed the rights to nusinersen from Ionis Pharmaceuticals, stated that the new regimen could offer meaningful benefits to patients. The higher dose comprises two 50 mg doses administered 14 days apart, followed by a 28 mg maintenance dose every four months. This is in contrast to the approved 12 mg regimen of SPINRAZA, the brand name under which nusinersen is currently commercialized in over 71 countries.
The applications for the updated dosing regimen are based on results from the DEVOTE study, a Phase 2/3 trial designed to evaluate the safety, tolerability, and efficacy of the higher dose of nusinersen. The study involved 145 participants across various age groups and types of SMA at approximately 42 sites worldwide. The pivotal part of the study showed that the higher dose regimen enabled meaningful clinical benefits while maintaining a safety profile consistent with the approved 12 mg regimen.
SPINRAZA has been a foundational treatment in SMA, with over 14,000 individuals treated worldwide. The drug works by increasing the amount of full-length survival motor neuron (SMN) protein, which is crucial for maintaining motor neuron health. Given directly into the central nervous system, SPINRAZA has demonstrated sustained efficacy and a well-established safety profile.
The positive market response to the regulatory acceptance indicates investor confidence in the potential for the higher dose regimen to expand the drug’s impact on SMA treatment. As the review process unfolds, stakeholders in the SMA community and Ionis Pharmaceuticals’ investors will be closely monitoring the progress.
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