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Biogen (BIIB) Gets Positive FDA Panel Vote for Tofersen NDA

Biogen BIIB announced that an FDA advisory committee gave a mostly favorable opinion on its new drug application (NDA) seeking approval for its pipeline candidate, tofersen for treating patients with amyotrophic lateral sclerosis (“ALS”) with superoxide dismutase 1 (“SOD1”) mutation.

The Peripheral and Central Nervous System Drugs Advisory Committee voted unanimously (9:0) that reductions of neurofilament, a marker of neurodegeneration, observed in tofersen-treated patients in the clinical studies, are reasonably likely to predict the clinical benefit of tofersen for treating SOD1-ALS. The committee supported an accelerated approval of tofersen for treating SOD1-ALS.

For the other question which was whether the clinical data submitted provides substantial evidence of the effectiveness of tofersen for treating SOD1-ALS, voting results were mixed. For this question, the committee voted 5 to 3 with one abstain, that the data submitted for the drug does not show enough evidence that tofersen is effective for treating SOD1-ALS. The committee does not believe the data supports a traditional/standard approval for tofersen.

Overall, the committee had the consensus view that the benefit-risk profile of tofersen in people with SOD1-ALS was favorable based on the review of the totality of data.


The NDA for tofersen is under priority review with the FDA, with a decision expected on Apr 25, 2023. Biogen is seeking approval of tofersen under the FDA’s accelerated approval pathway. Investors expect that the FDA should grant accelerated approval to tofersen on Apr 25.

Biogen’s stock has risen 24.6% in the past year against a decrease of 14.2% for the industry.

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The NDA filing with the FDA was based on 12-month data from the phase III VALOR study and its open-label extension and a couple of early-stage studies. Though the VALOR study did not achieve statistical significance in the primary endpoint of reducing disease progression, data from the study showed that compared to delayed initiation, the earlier initiation of treatment with tofersen showed clinical benefit in study participants. The earlier start of treatment with the candidate slowed the decline in clinical function, respiratory function, muscle strength and quality of life.

Data from the study also showed that treatment with tofersen led to robust and sustained reductions in neurofilament,

In Europe, the European Medicines Agency has also accepted the marketing authorization application for tofersen for treating SOD1-ALS.

ALS is a rare neurodegenerative disease whose progression leads to a steady decline in the ability to move, speak, eat and eventually breathe. The average life expectancy of people with this often-fatal disease ranges from three to five years. SOD1-mutated ALS is a rare genetic form of ALS that currently accounts for 2% of the ALS population, affecting about 330 people in the United States. Though medicines are approved for broad ALS, there is currently no treatment available that targets a genetic mutation associated with ALS. If approved, tofersen will be the first genetically-targeted treatment for SOD1-ALS.

Biogen had in-licensed the drug from Ionis IONS in 2018. Apart from tofersen, Biogen is also collaborating with Ionis to develop ION541 for ALS (phase II) and ION859 for Parkinson’s disease (phase I/II). Biogen and Ionis already have a collaboration deal for Spinraza for treating spinal muscular atrophy. While Biogen is responsible for Spinraza’s sales, Ionis receives royalties on the same.

Zacks Rank and Stocks to Consider

Biogen currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Some better-ranked drugmakers/biotech companies are Novo Nordisk NVO and Ligand Pharmaceuticals LGND, both with a Zacks Rank of 1 (Strong Buy).

Estimates for Novo Nordisk’s 2023 earnings per share have increased from $4.15 to $4.43. Estimates for 2024 have jumped from $4.38 per share to $5.19 in the past 60 days. The stock has surged 34.1% in the past year.

Novo Nordisk beat earnings expectations in three of the trailing four quarters. The company delivered a four-quarter earnings surprise of 3.00%, on average.

Estimates for Ligand Pharmaceuticals’ 2023 earnings per share have increased from $3.30 to $4.15 over the past 60 days while that for 2024 have jumped from $3.10 per share to $4.28 over the same timeframe. The stock has declined 38.4% in the past year.

Ligand Pharmaceuticals beat earnings expectations in one of the trailing four quarters. The company delivered a four-quarter negative earnings surprise of 10.07%, on average.

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