Zolgensma: The Hurdles to Bring it to Market

In early 2026, the stories of two Singaporean infants, Baby Ginny and Baby Faziq, captured the public’s hearts as their families raced to raise S$2.4 million for a dose of the one-time cure drug, Zolgensma. Both children suffer from Spinal Muscular Atrophy (SMA), a condition caused by a missing or defective SMN1 gene that leads to the progressive death of motor neurons. Without treatment, the most severe form is typically fatal before the age of two.

Thankfully, the fundraisers for Baby Ginny and Baby Faziq were successful, and both babies now have the best possible chance to rewrite their futures as they will receive treatment within the optimal clinical timeframe. 

The publicity surrounding Zolgensma made us look into the IP surrounding it, and clearing the IP hurdles was not straightforward. Indeed, there was a possibility that the drug could have been pulled from the market.

The Technology: A Viral Delivery Service[3]

The magic of Zolgensma lies in its delivery system. This system uses Adeno-Associated Virus serotype 9 (AAV9) as a delivery vehicle for crossing the blood-brain barrier, allowing the gene therapy to be delivered to the correct cells so that they can produce the essential proteins that prevent the death of motor neurons.

As of early 2026, over 5,000 patients worldwide have received this life-changing therapy[4], with data showing that children treated early can go on to achieve milestones such as sitting, standing, and even walking—outcomes previously considered impossible.

The IP Challenges

Bringing Zolgensma to market (and keeping it there) was not straightforward. Novartis needed to navigate a complex IP landscape with many different companies having rights that could block the sale of this life-changing drug.

The foundational patents for Zolgensma were based on Dr. James Wilson’s work in developing AAV vectors, including AAV9, at the University of Pennsylvania (UPenn). REGENXBIO obtained an exclusive license to this technology in 2009, they then sublicensed rights to a second company, AveXis, to develop treatments for SMA using these AAV vectors[5]. When AveXis announced successful initial clinical trial results in 2018, Novartis decided to acquire them in a US$8.7 billion deal[6].

At the same time that UPenn was developing the AAV vector technology, scientists at Genethon developed specific delivery routes necessary to enable the successful delivery of drugs like Zolgensma to the spinal cord, which is a difficult challenge due to the blood-spinal cord barrier – a membrane that seeks to prevent foreign materials (like Zolgensma) gaining access to the central nervous system. Therefore, Novartis also obtained a license from Genethon in 2018 to make use of this technology for Zolgensma[7].

Zolgensma obtained marketing approval and was launched in the U.S. in 2019[8]. However, perhaps the most significant IP hurdle surfaced in 2021, when Genzyme (a subsidiary of Sanofi) sued Novartis for infringing patents relating to the purification, drug stability and quality control for drugs like Zolgensma. The stakes were high; if Novartis were found to infringe and/or a settlement could not be reached, there was a very real risk that Zolgensma could be withdrawn from the market.

Thankfully, the litigation was settled in 2024, and Genzyme granted Novartis rights to use the patents in exchange for an undisclosed payment[9]. Given the success of Zolgensma, and the fact that it was already on the market when the litigation launched, the payment may have been substantial, given the amount Novartis paid for AveXis and also provided that the litigated patents were likely to be considered valid and infringed by a court. 

We can only speculate on the underlying facts of this case, and what the settlement was. However, the situation Novartis found themselves in serves to illustrate the importance of conducting due diligence across all aspects needed to bring a product or service to market.  This is particularly important in a fragmented and complex field where multiple parties may hold IP that could block the launch of a product. 

Subscribe to receive more articles like this here.

The importance of conducting due diligence across all aspects needed to bring a product or service to market is particularly important in a fragmented and complex field where multiple parties may hold IP that could block the launch of a product.