As gene therapies advance through clinical trials, more and more of these treatments are receiving approval from the U.S. Food and Drug Administration and its European counterpart, the European Medicines Agency. The latest in line to gain approval by the FDA was CSL Behring’s hemophilia B gene therapy Hemgenix. With a price tag of 3.5 MUSD per dose, Hemgenix will be the most expensive medicine in the world. BioStock reached out to Jan Nilsson, CEO of the Swedish gene therapy company CombiGene, for a comment.
In short, gene therapies are treatments that add a new gene, or replace or repair a defected gene inside the body’s cells to help prevent or treat certain diseases. The idea of genetic engineering is not new. The first theories arose half a century ago, but it wasn’t until 2003 that the first gene therapy was approved, in China. Since the first wave of gene therapy approvals, between 2003 and 2012, the pace has picked up speed.
Scientists gradually developed better viral vectors to deliver genetic therapies, and, in 2019, the US Food and Drug Administration (FDA) observed a massive increase in the number of cell and gene therapy products entering early development, thus expecting to receive more than 200 investigational new drug applications per year, and, by 2025, to approve 10 to 20 cell and gene therapies per year.
Today, according to the latest report from The Alliance for Regenerative Medicine, there are 1369 cell, gene, and tissue-engineering therapeutic developers worldwide, while 1215 clinical studies registered in ClinicalTrials are recruiting patients.
Regulatory approvals during the last months
In the latter part of 2022, the gene therapy sector gained momentum. In July, PTC Therapeutics was granted marketing authorisation by the European Commission for the gene therapy Upstaza for the treatment of a rare genetic disease. In August, The European Medicines Agency (EMA) approved BioMarin’s gene therapy for the treatment of hemophilia A, Roctavian.
Also during Q3, FDA approved two of Bluebird bio’s gene therapy treatments. Zynteglo broke the record as the priciest therapy in the world with 2.8 MUSD per treatment. However, that changed when their second gene therapy Skysona soon after won FDA approval, with a whopping price tag of 3 MUSD per treatment.
The latest development in the sector came this week when US drug regulators approved CSL Behring’s one-off gene-therapy treatment Hemgenix for adults with the genetic blood disorder hemophilia B. The treatment costs 3.5 MUSD a dose, now making it the new leader as the most expensive medicine in the world.
CSL licensed the exclusive global rights to Hemgenix from uniQure. UniQure received 450 MUSD cash payment from CSL Behring and is eligible to receive up to 1.6 billion USD in payments based on regulatory and commercial milestones.
Swedish gene therapy on the rise
Swedish biotech company CombiGene has two gene therapy projects in pipeline, CG01 and CGT02. The main candidate CG01 is being developed for the treatment of drug-resistant epilepsy, while CGT2 is being developed for the treatment of lipodystrophy.
Last year, CombiGene signed an exclusive collaboration and licensing agreement worth up to 328.5 MUSD with US-based Spark Therapeutics for the development of CG01. The project is now in the final phase of preclinical development, with the hope of being able to initiate the first study in humans under the leadership of Spark.
CombiGene’s CEO comments on the recent developments
BioStock spoke to CombiGene’s CEO Jan Nilsson to get his take on the recent development in the field of gene therapy.
Jan, to start out with, what does the approval of Hemgenix mean for the gene therapy area and the general acceptance compared to conventional, often life long, drug treatments?
– For each new approval, gene therapy becomes more and more accepted as an efficient treatment option. Each new approval also serves as inspiration for the entire industry to keep working towards our goal of improving the life for patients who today suffer from diseases with no effective treatment options. I also believe that this is the first approval of a gene therapy for a disease that is commonly known, i.e. hemophilia.
The Hemgenix treatment will be the most expensive ever, which could act as a deterrent for reimbursement systems and the willingness to prescribe it to patients. What is your view of the pricing of gene therapies?
– I think this is one very important question when it comes to gene therapy, but it is not an easy one. There are several factors that need to be considered. Health economics is of course one. I think that the industry will benefit enormously if we can demonstrate positive health economic effects with our treatments. Another factor is the payment systems. How shall we get paid? For each treatment or for achieved results, and over which time period. It is in the context important to remember that a gene therapy treatment typically is given only once or a few times, whereas traditional treatments often tend to be lifelong. Naturally you also need to consider what options the patient has, and what the effect of a declined treatment would be. I spend quite a lot of time on these issues and follow new developments closely. My opinion is that we as an industry together with the payers still have some issues that need to be discussed and resolved.
On the 24th of November, the Alliance for Regenerative Medicine (ARM) concluded that the EU should modernise its policy and regulatory framework to reflect the distinct promise of cell and gene therapies as the future of medicine. What is your general view on this?
– I think this is a very good proposal. Our industry needs to have a regulatory framework that stimulates innovation and progress. The potential of cell and gene therapy is great, and if we could have regulatory framework which embraces the fantastic opportunities, we could see a development that would be beneficial for patients suffering from severe diseases as well as for the industry itself. As discussed in the previous question, we also need to include the payers to form a truly cohesive framework.
Finally, as we have concluded in previous interviews with you, an important next step for CombiGene, in addition to advancing CG01, is to in-license a third gene therapy project. Can you give us a hint on how this work is progressing?
– The very nature of this process is of course that it is conducted under full confidentiality. However, it is no secret that finding new gene therapy projects for inlicensing is at the very top of our agenda. We have recently strengthened our organization to make sure that we have the internal resources we need. Furthermore, we have mapped a wide range of potentially interesting projects both from academia and industry. Based on this work, we have singled out a smaller number of projects that we currently are evaluating both from a medical and commercial perspective.The content of BioStock’s news and analyses is independent but the work of BioStock is to a certain degree financed by life science companies. The above article concerns a company from which BioStock has received financing.