The Future of Targeted Therapy in CIDP

Body text: The treatment landscape for chronic inflammatory demyelinating polyneuropathy has changed considerably over the past several years, with targeted therapies expanding the options available to clinicians beyond traditional immunomodulatory approaches. As these therapies become integrated into routine practice, the focus is increasingly turning toward optimizing treatment selection and defining long-term therapeutic goals for individual patients.

In this NeurologyLive® Special Report, Karissa Gable, MD, FAAN, professor of neurology at Duke University, joins Jeffrey Allen, MD, associate professor of neurology at the University of Minnesota, to discuss the clinical implications of a post hoc analysis from the ADHERE and ADHERE+ studies evaluating subcutaneous efgartigimod PH20 (Vyvgart Hytrulo). Throughout the series, the two experts have explored how achieving lower disability may reshape expectations for patients living with CIDP and influence future treatment strategies.

In this concluding episode, Gable and Allen reflect on the broader implications of the analysis, discussing how targeted therapies are changing the treatment paradigm, why shared decision-making remains central to patient care, and the growing need for biomarkers that can help identify which patients are most likely to experience durable responses with therapies such as efgartigimod.

Karissa Gable, MD, FAAN:

From my perspective, this therapy represents an important addition to our armamentarium and expands the tools we have available to help patients return to their lives and function as close as possible to where they want to be.

As we continue to better understand the underlying pathophysiology of this heterogeneous disease, we'll also gain a better understanding of which therapies work best for which patients. I think that's really where this discussion begins. Historically, we've relied largely on broader immunomodulatory therapies that were used across many different patients, yet some individuals remained refractory and had limited treatment options. Efgartigimod provides another targeted option, and it will be important to better understand which patients derive the greatest and most durable benefit from it. Those are my thoughts. Jeff, is there anything you'd add?

Jeffrey Allen, MD:

I'd agree with all of that. I also think it highlights the importance of shared decision-making whenever we're selecting treatment for patients with CIDP. We need to establish realistic expectations together and agree on what success looks like, but we also need to choose therapies that fit each patient's lifestyle while minimizing treatment burden and adverse effects. At the same time, we have to consider factors such as cost and other comorbidities.

It's encouraging to have new treatment options that can achieve meaningful clinical benefit while potentially reducing treatment burden for some patients.

Looking ahead, one of the biggest challenges will be determining which patients are the best candidates for targeted therapies such as FcRn antagonists. We need to better identify those who are least likely to relapse after transitioning therapy and those who are most likely to experience the durable benefits observed in ADHERE and ADHERE+.

Ideally, we'll develop biomarkers that allow us to identify those patients before treatment begins. Right now, we don't have that capability, but I think developing those tools will be one of the most important priorities for the field moving forward, and I'm excited to see where that research leads.