John Brandsema, MD, on Navigating the New Landscape of Care for DMD

“If you receive gene transfer with Elevidys, for example, would we continue exon skipping for those who are eligible for it? There's a scientific argument for me why that might be valid, but that may not be approved by the third-party payer of that patient.”

On June 20, 2024, the FDA approved Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), an adeno-associated virus (AAV) vector-based gene therapy for patients with Duchenne muscular dystrophy (DMD), for an expanded indication, making it an option for ambulatory patients (via traditional approval) and nonambulatory patients (via accelerated approval) with a confirmed mutation in the DMD gene who are 4 years of age or older and who do not have any deletion in exon 8 or exon 9 in the gene. As a result of this decision, many more patients became eligible for the gene therapy than were previously, thus adding to the number of FDA-approved treatment options many individual patients can choose between.

CGTLive® recently interviewed John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, to learn more about how doctors, patients, and their families are now navigating the treatment landscape for DMD in the real world setting. Brandsema discussed several aspects of the treatment landscape that continue to make the decision-making process challenging, such as the need to balance the benefits and risks of a diverse array of approaches to treatment; the lack of data on gene therapy in older patients; and the difficulties of implementing combination treatments, both from the perspective of insurance coverage and from the scientific perspective of monitoring for potential adverse outcomes. With regard to the latter, Brandsema emphasized the friction between a staggered approach to combination treatments, for the sake of scientific rigor, and the desire to give patients everything that might help them as soon as possible in the face of a disease that causes degeneration in muscles over time.