Gene genies

The story of gene therapy has taken many twists and turns, but with 14 gene therapies currently approved by the European Medicines Agency (EMA) and a further 2025 gene therapies expected to reach market approval by 2030, there’s no denying that the much-anticipated gene therapy revolution is about to become a reality.

The gene therapy landscape, however, is dynamic at best and turbulent at times. Over the last few years, there has been a near constant stream of news around gene therapy, from treatments receiving regulatory approval, clinical trial successes and failures, and regulatory and commercialisation triumphs and setbacks.

As with any innovative new approach, some challenges are unpredictable; however, medical researchers, institutions and regulatory agencies have been working to ensure that gene therapy research and clinical trials yield approved treatments for conditions with high unmet need. To move forward and realise the full potential of gene therapies, we need to be bold and willing to adapt, even innovate, known structures and old pathways.

There are many disease areas that can benefit from gene therapy research. My personal dream is that one day, there will be a one-time treatment for most genetic diseases available to all eligible patients.

A different lens

One subset of diseases that are prime candidates for gene therapy are those known as ‘monogenic’. This simply means that a disease is caused by a singular faulty or missing gene, so gene therapy can be used to correct or replace that singular gene causing a condition – usually via a one-time dose.

Many monogenic conditions are rare diseases, meaning there are only small patient populations for clinical studies, leading to limited data available at the time of approval. It is not always feasible, however, to wait for long-term data before making a therapy available to patients in need – due to the usually progressive and detrimental nature of these diseases. Conditional approval could be considered to allow for this if early positive benefit/risk is established with the new therapy.

Echoing the value of one-time gene therapies, the EMA’s Committee for Medicinal Products for Human Use recently adopted a positive opinion for our SMA gene therapy in its second annual renewal of the conditional marketing authorisation.

Furthermore, the medicine is no longer subject to conditional marketing authorisation based on its quality, safety and efficacy having been sufficiently demonstrated, translating to the treatment of over 2,000 patients to date.
The current approach to data requirements and sample sizes for marketing authorisation is only one area in which we must work with regulators to find a more agile path forward.

Agility ability

Many innovative treatments are designed to improve patients’ lives significantly, some of whom may have no current treatment options available. Thus, we must challenge the status quo of healthcare systems and traditional access pathways to accommodate new therapies.

Only then can we share the potential benefits of medicine innovation among patients, industry and wider society. Stakeholders must be willing to collaborate and find new solutions to lessen the burden treatment costs may place on society.
We need to work together to find innovative ways to share the risk of the uncertainties.

Mike Fraser is General Manager, EMEA at Novartis Gene Therapies.
Go to novartis.com