Clearance granted in biotech sector opens up fresh ground for development
In the dynamic world of biotechnology, recent developments have highlighted both challenges and opportunities.
Ionis Pharmaceuticals, a key player in the field, has made significant strides. The Food and Drug Administration (FDA) approved Donidalorsen for the prophylactic treatment of hereditary angioedema, and published groundbreaking Phase III results for Olezarsen in severe hypertriglyceridemia.
Vertex, another industry leader, reported promising results for its type 1 diabetes treatment, VX-880, an innovation with disruptive potential. This innovative therapy for diabetes could potentially revolutionise the treatment landscape.
Despite regulatory uncertainties, BB Biotech, a Swiss investment company, continues to generate revenue, with a drug almost exclusively sold in the US. Interestingly, the company trades at a discount of around 10% to its intrinsic value.
The biotech sector as a whole finds itself at historically low levels, with many small caps trading near or below their net asset value. This discrepancy between scientific progress and market price could signal a long-term re-evaluation.
Four key approval decisions are pending this year. Scholar Rock with Apitegromab, Agios awaits a decision on Mitapivat, Biohaven is in the Priority-Review process with Troriluzol, and Ionis plans to submit the marketing application for Olezarsen this year.
The potential implications also include the risk of restricted drug introductions in Europe and a balancing act between global margin security and fair access to innovation.
The most favoured nation pricing model in the US is currently under discussion, which could link drug prices to the lowest international comparative prices. If implemented, this could result in significant price pressure in the US, the world's most important market for pharmaceutical profits.
Argenx received approval for Vyvgart Hytrulo as a subcutaneous pre-filled syringe, a step towards simplified application and higher patient acceptance.
Alnylam Pharmaceuticals secured EU approval for Amvuttra in ATTR cardiomyopathy, expanding its market potential.
Strategic buyers like Johnson & Johnson, Sanofi, and Merck & Co. have invested billions in biotech innovations in recent months. Johnson & Johnson acquired Intra-Cellular Therapies for USD 14.6 billion, Sanofi bid for Blueprint Medicines up to USD 9.5 billion, and Merck & Co. offered for Verona Pharma USD 10 billion.
Roche made one of the largest recent investments in the biotech sector by committing a total of $50 billion for US investments announced in 2025, including a $700 million facility in North Carolina and acquiring the US biotech company 89bio for up to $3.5 billion. Amgen also invested $600 million in a new science innovation center in 2025 as part of their ongoing multi-billion dollar US commitment since 2017.
BB Biotech AG is one of the leading investors in the biotech sector, primarily investing in innovative drug development companies based in the US and Western Europe.
Clinical studies have seen milestones, including Ionis's FDA approval for Donidalorsen and the publication of promising phase III results for Olezarsen. The second quarter demonstrated that scientific innovation continues to drive growth in the biotech sector.
Regulatory uncertainty remains a risk in the biotech sector. However, the resilience and innovation shown by companies like Ionis, Vertex, and others, as well as the significant investments by strategic buyers, indicate a sector poised for continued growth and development.
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