Biogen BIIB announced that the European Commission (EC) has granted marketing approval to Qalsody for treating patients with amyotrophic lateral sclerosis (“ALS”) with superoxide dismutase 1 (“SOD1”) mutation.
The EC has granted marketing authorization to Qalsody under exceptional circumstances. When a treatment is deemed to have a positive benefit/risk assessment but gathering comprehensive data is difficult due to the rarity of the condition, regulatory agencies like the European Medicines Agency (EMA) may recommend granting marketing authorization under such circumstances. The EC has also maintained an orphan designation for Qalsody.
The approval was based on data from the phase III VALOR study. The approval was expected as, in February, the Committee for Medicinal Products for Human Use (CHMP) of the EMA rendered a positive opinion recommending a marketing authorization under exceptional circumstances for Qalsody for SOD1-ALS.
Biogen’s stock was up 2.6% on Thursday in response to Qalsody’s approval in the EU. Biogen’s stock has declined 15% so far this year compared with a decline of 7.7% for the industry.
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ALS is a rare neurodegenerative disease that leads to the loss of motor neurons in the brain and the spinal cord. Progression of the disease leads to muscle weakness and atrophy, which causes 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 genetic form of the disease that currently accounts for 2% of the ALS population. Qalsody is the first therapy approved to treat the rare genetic form of ALS in Europe. SOD1-ALS affects less than 1,000 people in Europe
Qalsody/tofersen was granted accelerated approval to treat ALS with SOD1 mutations in the United States in April 2023. Qalsody recorded sales of $4.6 million in the first quarter of 2024 and $3.3 million in the fourth quarter of 2023. On the Q1 conference call, Biogen said that the uptake of Qalsody was encouraging.
Biogen in-licensed rights to tofersen from Ionis Pharmaceuticals IONS under a collaborative development and license agreement inked in 2018.
Biogen and Ionis already have an existing partnered drug, Spinraza, which is the standard-of-care treatment for treating spinal muscular atrophy (SMA). While Biogen records Spinraza’s sales, Ionis receives royalties on the same. Biogen is also collaborating with Ionis to develop ION859 for Parkinson’s disease (phase I/II).
Qalsody is Biogen’s third rare disease therapy to be approved in the EU. The other two rare disease drugs in its portfolio are Spinraza and Skyclarys for Friedreich’s ataxia, added with the September 2023 acquisition of Reata Pharmaceuticals.
Biogen believes its new products, such as Qalsody, Skyclarys, Leqembi for Alzheimer’s disease and Zurzuvae for depression, have the potential to revive growth in the long term as sales of most of its key drugs are declining due to intense competitive pressure
Zacks Rank and Stocks to Consider
Biogen currently has a Zacks Rank #3 (Hold).
Biogen Inc. Price and Consensus
Biogen Inc. price-consensus-chart | Biogen Inc. Quote
Some better-ranked stocks in the healthcare sector are Bioventus BVS and Heron Therapeutics HRTX. While Bioventus has a Zacks Rank #1 (Strong Buy), Heron Therapeutics has a Zacks Rank of 2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, 2024 estimates for Bioventus have improved from 16 cents per share to 27 cents per share. For 2025, earnings estimates have improved from 40 cents to 43 cents per share in the past 60 days. So far this year, shares of BVS have risen 28.6%.
Earnings of Bioventus beat estimates in three of the last four quarters while missing in one, delivering a four-quarter average earnings surprise of 151.67%.
In the past 60 days, loss estimates for Heron Therapeutics for 2024 have narrowed from 22 cents per share to 10 cents per share over the past 60 days. For 2025, estimates have improved from a loss of 9 cents per share to earnings of 1 cent per share over the same timeframe. Year to date, shares of HRTX have risen 102.9%.
Earnings of Heron Therapeutics beat estimates in three of the last four quarters while missing in one, delivering a four-quarter average earnings surprise of 30.33%.
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