Breakthroughs in Post-Polio Syndrome Treatment: Grifols’ Innovative Approach

In this discussion, Dr. Jörg Schüttrumpf, Chief Scientific Innovation Officer at Grifols, delves into the promising results of their latest clinical trial for Flebogamma 5% DIF. This intravenous immunoglobulin (IVIG) treatment offers new hope for patients with post-polio syndrome (PPS), a condition with few treatment options. Schüttrumpf shares insights on trial design, patient benefits, and navigating regulatory landscapes, highlighting Grifols’ commitment to innovation and patient care.

Moe: Could you share the data showing improved physical performance in PPS patients using Flebogamma 5% DIF?

Our study underscores Grifols’ dedication to leveraging our expertise in immunoglobulins for patient benefit. Patients receiving monthly IVIG infusions of 1 gram per kilogram of Flebogamma 5% DIF showed a statistically significant improvement in the two-minute walk distance test, our primary endpoint. After 52 weeks, there was a 12.75-meter improvement compared to baseline, which was 6.07 meters more than the placebo group. Additionally, in the six-minute walk distance, a secondary endpoint, patients improved by 29.16 meters, 15.8 meters more than the placebo group. Notably, the treatment was safe and well-tolerated. This data highlights the potential efficacy of Flebogamma and underpins our commitment to exploring new applications for immunoglobulins and therapeutic antibodies.

Moe: What methodologies ensured the reliability of your results, and how do they differ from past approaches?

We recognized the limitations of previous studies, which often involved small, underpowered designs. We implemented a continuous, long-term, monthly treatment in a randomized, double-blind, placebo-controlled setting to address this. We included 191 adult patients, randomized to either Flebogamma or placebo, dosed every four weeks. This approach allowed us to draw on past experiences with IVIG in PPS while providing a robust evaluation of IVIG efficacy and safety. By doing so, we achieved a high degree of evidence, setting a new standard for clinical trials in this field and ensuring that our findings are both reliable and impactful.

Moe: What real-world benefits can PPS patients expect from this treatment?

Post-polio syndrome is characterized by muscle weakness and fatigue, significantly impacting mobility and daily activities. Our study showed that improving walking distance can greatly enhance patients’ quality of life, allowing them to maintain independence and perform normal activities of daily living. This is particularly important for a condition that emerges decades after the initial polio infection. By improving physical performance, we are addressing symptoms and empowering patients to lead fuller, more active lives, which is a significant step forward in managing this challenging condition.

Moe: How did Grifols approach regulatory discussions to address the unmet need in PPS?

In general, our strategy involves generating robust clinical evidence to make our treatments available to patients as soon as possible. To this end, we have constructive dialogue with regulatory authorities as part of our clinical trial programs. In the specific case of our treatment for PPS, we plan to consider options for patient access on a country-specific basis. By leveraging Flebogamma’s existing approval and demonstrating its efficacy in a new indication, we are poised to fill a critical gap in treatment options for PPS. This approach highlights our commitment to addressing unmet medical needs and showcases our proactive engagement with regulatory bodies to expand the reach of our therapies.

Moe: How is Grifols addressing payer skepticism regarding IVIG use in rare chronic conditions like PPS?

This study’s scope focused on providing the best possible clinical evidence on the impact of our Flebogamma IVIG on PPS. We believe that keeping patients active and independent will positively impact their quality of life while also having a beneficial impact on cost-effectiveness evaluations. We are also exploring real-world evidence that will support our case with payers in the future. By providing comprehensive data from clinical trials and real-world settings, we aim to demonstrate the long-term value of our treatment, not just in terms of patient outcomes but also in reducing healthcare costs associated with decreased mobility and increased care needs.

Moe: Can you share how real-world evidence might complement your clinical data for PPS?

Although we haven’t yet performed a real-world evidence (RWE) analysis for PPS, we recognize its potential. We are actively exploring how to integrate real-world data to support patient access and address any hurdles. The industry and regulators are still learning to use RWE effectively, but technological advancements, like electronic health records and large language models, offer promising opportunities. However, the bar for using this data is high, and in conditions like PPS, where patient numbers are limited, it can be challenging. Nonetheless, we are committed to navigating these complexities to enhance our evidence base and support regulatory and payer discussions.

Moe: Is there anything else you’d like to add about Grifols’ work in this area?

We’re excited about the potential to enhance the quality of life of PPS patients and are committed to developing immunoglobulins for other high-need indications. It’s crucial to spread awareness so patients can access these therapies. Our ongoing efforts to innovate and expand the applications of plasma-derived medicines reflect our dedication to addressing high medical needs where no treatments are available, like PPS. Thank you for highlighting this important work, as it helps ensure that patients and healthcare providers are informed about new therapeutic options.