Kleinkinder erkranken an Krebs: Novartis erleidet mit der Gentherapie Zolgensma einen weiteren Rückschlag

Novartis, a global pharmaceutical company, has faced backlash over its expensive gene therapy, Zolgensma, which is suspected of causing cancer in young patients. Despite the public outcry and concerns from medical professionals, Novartis' stockholders remained calm on Monday. The drug, which cost $2.1 million per treatment, was launched in 2019 as the world's most expensive medication.

Since its introduction, over 6,000 infants with spinale Muskelatrophie (SMA), an inherited muscle-wasting disease, have received the therapy. However, cancer cases have raised alarms among parents of treated children and healthcare workers. At least nine cancer cases have been reported since the drug's launch in 2019, with two patients showing the integration of AAV-Vector DNA into their genetic material. This integration could potentially lead to tumor formation.

The Zurich Cantonal Bank analysts confirmed that the tumor risk has now become a clinical reality. They also noted that the risk of cancer development was already mentioned in the drug's package insert. However, market observers believe that affected parents will now more carefully consider the therapy for their children.

Novartis stated that they will analyze all new information regarding the drug's safety. The American SMA patients' organization pointed out that there is currently limited information about the timing and type of cancer cases. They also questioned whether patients should be more closely monitored after receiving the therapy.

Gentherapies, or gene therapies, are still a relatively new field in the pharmaceutical industry. While there have been some impressive successes, such as the treatment of SMA, there have also been numerous reports of severe side effects and even deaths. Novartis acquired Zolgensma from US biotech company Avexis for $8.7 billion just four years ago.

In the past year, Zolgensma's sales stagnated at $1.2 billion, with a 13% increase in the first nine months of the current year to $1.1 billion. However, the therapy still only ranks 11th among Novartis' top-selling products. US bank J. P. Morgan analysts had previously predicted that Zolgensma sales would reach $2.3 billion by 2030. However, recent estimates suggest that the figure may be closer to $3.2 billion.

Despite the issues with Zolgensma, Novartis' stockholders remained unimpressed on Monday. The company's stock price rose by nearly 1%, reaching just above its recent all-time high of $135.22. Investors seem confident that Novartis' pipeline of new drugs will help mitigate any losses from the Zolgensma debacle. However, some industry observers are skeptical, fearing that more research failures could lead to unpleasant consequences for the company's investors.