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Letter to the Editor: Gene therapies are reaching regulatory approvals! But are they reaching the patients?


Miguel Forte, M.D., Ph.D. and Bruce Levine, Ph.D.

The field of C&GT is experiencing great growth, and we are witnessing a growing number of products being approved.  The hope would be that these products following regulatory approvals and marketing efforts will be reaching patients in need. C&GT represents a significant paradigm shift because of the incredible technological and scientific innovation coupled with long-lasting therapeutic effects that are potentially curative. These products rest on the genetic modification or gene editing of cells for a corrected or targeted improved function and tolerability. C&GT is becoming more and more a field of cell engineering both ex-vivo and in-vivo.

August 2022 witnessed gene therapy approvals both in the US by the FDA as well as by EMA in Europe. On August 17th, the FDA approved Zynteglo (betibeglogene autotemcel), a cell-based gene therapy to treat adult and pediatric patients with beta-thalassemia who require regular blood transfusions.  ISCT noted the significance of this approval for patients in need by stressing the good news for the field with this therapeutic valuable innovation, yet balanced with the need to provide patient access worldwide.  Two days later, August 24th marked the conditional approval of BioMarin’s Roctavian (valoctocogene roxaparvovec) by EMA in Europe for severe haemophilia A, making it the first gene therapy cleared for this patient population.  Within a month the FDA approved a second bluebird bio gene therapy product, Skysona, which had been approved by the European Commission in 2021 for With Early Cerebral Adrenoleukodystrophy, received FDA approval on September 19, 2022.

While we celebrate these landmark approvals, as a field we must recognize the challenges to patient access presented by the price tags of these drugs. The FDA approval of Zynteglo benefits to be put in context with its previous conditional approval in Europe by the EMA for the same patient group in May 2019 and the challenges for patient access. The product, as required for conditional approval, went through several annual renewals until a company requested withdrawal just a year ago in August 2021. This voluntary withdrawal was decided by Bluebird Bio in view of the inability to reach agreement with European Health Authorities on the treatment price. The company intended a price of around €1.6 million ($1.8 million) with Health Authorities aiming at reductions in the order of 50%. In the end this discussion and strategic decision leave European patients without access to this innovative gene therapy. For the approval now in the US, Bluebird Bio aims at a price of $2.8 million. With some apparent support from potential payers in the US, it will be interesting and very important to watch and learn from the launch history and uptake of this therapy with US patients.  For the time being, beta-thalassemia patients elsewhere in the world will not have access to Zynteglo. The Institute for Clinical and Economic review assessed the value of Roctavian at an assumed price of $2.5  .     Skysona broke a drug pricing record at $3 million upon approval.

Gene therapies, mostly for rare and low patient-number indications so far, are reaching the market validating the science and clinical efficacy of these transformative therapies that appear to be of long-lasting, potentially corrective, therapeutic value. It is important to understand the investment that has taken us here and still the manufacturing challenges that these approaches represent.  A lifesaving therapy is only lifesaving if patients are able to access it and afford it. With time, like with most technologies, we hope to see improved access while we must always bear in mind the balance between the real added value to patients versus the price of these therapies, together with the avoided costs and individual and societal implications.

As long time members of ISCT, with its mission to promote the translation of C&GT products towards the patients and strive for patient access to them globally, we recognize the innovation and therapeutic value of these product approvals. However, we believe it is important to continue to work with all stakeholders to help facilitate the product development process in a way that promotes and maximizes patient access to C&GT products globally.

This is Letter to the Editors article. The opinions expressed in this article are the author’s own. ISCT does not endorse nor support views, opinions or conclusions drawn the Letter to the Editors articles and we are not responsible or liable for any content, accuracy or quality within the article or for any damage or loss to be caused by and in connection to it.