Publisher's Note: With Age Comes Wisdom

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On June 20, 2024, FDA announced that it had expanded its approval of delandistrogene moxeparvovec-rokl (Elevidys), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD), to cover both ambulatory and non-ambulatory DMD patients of four years and older with a confirmed mutation in the DMD gene (1). This expanded approval will allow for a greater number of patients suffering from DMD, which is a rare and serious genetic condition that progresses over time, to be treated. In addition, the expanded approval will help to meet the urgent unmet medical need of this patient population.

The area of rare, difficult-to-treat diseases is where cell and gene therapies truly shine, offering treatment options where there have previously been none and in some cases, even the potential to eliminate the disease altogether. Given the promise of these advanced therapies, it is no wonder that the global market is expected to witness significant double-digit growth over the next few years (2).

However, as the cell and gene therapy sector continues to mature, greater attention is being paid to increasing therapeutic efficacy while also improving patient safety. This particular topic has been explored in this issue’s cover story on pages 10–12 (3), where experts reveal some of the strategic approaches being used to overcome constraints and challenges related to emerging therapies, such as reducing vector tropism to reduce liver damage or regulating the immunoreceptor complex with a dimerizing agent.

As researchers from all over the industry and regulatory bodies continue to focus on their investigations into approaches and strategies to improve not only the therapies themselves, but also methods to test these novel drugs, their efficacy and safety for patients will be optimized, and more unmet medical needs will be fulfilled.

References

1. FDA. FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy. Press Release, June 20, 2024.
2. Research and Markets. Cell and Gene Therapy Global Market Report 2024. Market Report, February 2024.
3. Mirasol, F. Taking Stock of a Maturing CGT Sector. Pharm. Tech. 2024 48 (7) 10–12.

About the author

Mike Hennessy Jr is president and CEO of MJH Life Sciences®

Article details

Pharmaceutical Technology®
Vol. 48, No. 7
July 2024
Page: 8

Citation

When referring to this article, please cite it as Hennessy, M. With Age Comes Wisdom. Pharmaceutical Technology 2024 48 (7).