FDA and EMA review Biogen's higher dose SMA drug

Published 23/01/2025, 11:38 pm

CAMBRIDGE, Mass. - Biogen Inc. (NASDAQ:BIIB), a $20.8 billion biotechnology company with a robust 75.6% gross profit margin, has reached a pivotal phase as regulatory agencies in the United States and Europe are now reviewing a higher dose regimen of nusinersen, a drug designed to treat spinal muscular atrophy (SMA). The U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA), and the European Medicines Agency (EMA) has validated the same application.

This new regimen includes a more rapid loading dose and an increased maintenance dose compared to the currently approved SPINRAZA® treatment, which is available in over 71 countries. The announcement was made by Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen, who expressed gratitude to the SMA community for their support.

The DEVOTE study, a Phase 2/3 trial, has been instrumental in evaluating the efficacy and safety of the higher dose regimen. It involved 145 participants of various ages and SMA types across approximately 42 sites globally. The study showed that the new regimen could provide meaningful clinical benefits while maintaining a safety profile similar to the approved 12 mg dose of SPINRAZA. With the stock currently trading near its 52-week low, InvestingPro analysis indicates the company remains undervalued based on its Fair Value calculations.

SPINRAZA, the brand name for nusinersen, is an antisense oligonucleotide (ASO) that increases the production of full-length survival motor neuron (SMN) protein, essential for motor neuron health. The drug is administered directly into the central nervous system, targeting the disease at its source.

Biogen, a pioneer in neuroscience, has a rich history of bringing innovative treatments to market. According to InvestingPro, which offers detailed analysis through its comprehensive Pro Research Report, the company shows strong financial health and is expected to grow its net income this year. The company has emphasized its commitment to advancing care for individuals with SMA through this latest development.

The review of the higher dose regimen by the FDA and EMA marks a significant advancement for SMA treatment options, with investors anticipating the company's next earnings report on January 30, 2025. The information reported here is based on a press release statement from Biogen Inc.

In other recent news, Biogen Inc. continues to make headlines with a series of significant developments. The biotech company's disciplined growth strategy has earned it an Outperform rating from Oppenheimer, further highlighting Biogen's leadership in Alzheimer's Disease treatment. On the other hand, the company's revenue outlook has been deemed challenging by Piper Sandler, which downgraded Biogen from "Overweight" to "Neutral" and reduced its price target to $138.

In the realm of analyst adjustments, Biogen experienced a series of changes. RBC Capital Markets reduced its price target for Biogen to $231 but maintained an Outperform rating. Baird increased its price target to $300 following a positive recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use for lecanemab, an Alzheimer's treatment. Jefferies downgraded Biogen from Buy to Hold, reducing the price target to $180 due to concerns over Leqembi and a potential decline in Ocrevus royalties.

Recent news also highlights a strategic shift in finance leadership within Biogen with the appointment of Sean Godbout as Vice President, Chief Accounting Officer & Global Corporate Controller, effective March 1, 2025. This comes as current CFO Michael McDonnell is set to retire on the same date.

Despite facing several challenges, Biogen's strong fundamentals, including a gross profit margin of 75.6% and a robust financial health score, are expected to contribute to near-term revenue growth. The company's strategic business development strategy and disciplined approach to capital allocation are also expected to support future growth.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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