FDA approves Genentech’s Evrysdi tablet as first SMA treatment in tablet form

In a significant development for the treatment of spinal muscular atrophy (SMA), the U.S. Food and Drug Administration (FDA) has approved Genentech‘s Evrysdi tablet, marking a new chapter in SMA care. This approval introduces the first and only tablet designed to modify the progression of SMA, offering patients a more convenient, non-invasive treatment option. The Evrysdi tablet, which can be swallowed whole or dispersed in water, provides the same level of efficacy and safety as its previously available oral solution, making it a groundbreaking advancement for individuals living with this rare genetic disorder.

The approval is particularly important because SMA is a debilitating condition that affects motor neurons, leading to progressive muscle weakness and, in severe cases, respiratory failure. By offering a flexible SMA treatment option, the Evrysdi tablet enhances patient autonomy, simplifies administration, and reduces the dependency on healthcare facilities for drug delivery. This shift toward greater convenience and accessibility is expected to improve the quality of life for individuals with SMA and their caregivers.

How Does the Evrysdi Tablet Improve the Lives of People with SMA?

The Evrysdi tablet approval reflects Genentech’s commitment to advancing therapies that are both effective and patient-friendly. According to Dr Levi Garraway, Genentech’s Chief Medical Officer and Head of Global Product Development, the tablet formulation is designed to combine established clinical benefits with greater convenience. Dr Garraway highlighted that this new option allows individuals with SMA to manage their condition more independently without sacrificing the treatment’s proven efficacy.

One of the most significant advantages of the Evrysdi tablet is its storage flexibility. Unlike the oral solution, which often requires refrigeration, the tablet can be stored at room temperature, making it ideal for people who travel frequently or have limited access to specialized storage conditions. This new formulation also reduces the burden on caregivers, as administering a tablet is often simpler than managing liquid medication, particularly for older children and adults. The tablet’s ease of use is expected to enhance treatment adherence, which is crucial for managing chronic conditions like SMA.

What Is Evrysdi and How Does It Work to Treat Spinal Muscular Atrophy?

Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier. It is specifically designed to treat SMA caused by mutations in chromosome 5q, which result in a deficiency of survival motor neuron (SMN) protein. SMN protein plays a crucial role in maintaining healthy motor neurons, which are responsible for controlling muscle movement. Without sufficient SMN protein, motor neurons deteriorate, leading to the muscle weakness and mobility issues characteristic of SMA.

Evrysdi works by increasing and sustaining the production of SMN protein in both the central nervous system and peripheral tissues. This helps slow the progression of the disease, improving motor function and potentially extending the lifespan of individuals affected by SMA. The medication is administered daily, either in liquid form or now, thanks to the recent FDA approval, as an oral tablet. The tablet formulation provides the same bioavailability as the liquid version, ensuring that patients receive consistent therapeutic benefits regardless of the delivery method. This was confirmed through rigorous bioequivalence studies, which demonstrated that the 5 mg tablet—whether swallowed whole or dispersed in water—offers comparable exposure to risdiplam as the oral solution.

What Clinical Trials Supported the FDA’s Approval of the Evrysdi Tablet?

The FDA’s approval of the Evrysdi tablet was based on a comprehensive body of clinical evidence gathered from several global studies. These trials evaluated the drug’s efficacy, safety, and pharmacokinetics across different SMA patient populations, including infants, children, and adults. The data showed that Evrysdi consistently improved motor function and slowed disease progression in people with SMA, regardless of their age or disease severity at the start of treatment.

One of the pivotal studies, FIREFISH, focused on infants with Type 1 SMA. It demonstrated significant improvements in motor function and survival rates over a five-year follow-up period, marking a breakthrough in early intervention for spinal muscular atrophy. Another critical study, SUNFISH, was a double-blind, placebo-controlled trial that assessed the drug’s impact on individuals aged 2 to 25 with Types 2 and 3 SMA. This trial met its primary endpoint, confirming the drug’s effectiveness in improving motor function in a broad patient population. Additional trials, such as JEWELFISH and RAINBOWFISH, explored Evrysdi’s safety and pharmacokinetics in both pre-symptomatic and symptomatic patients, further reinforcing its broad applicability in SMA treatment.

Ongoing studies like MANATEE, HINALEA 1 and 2, and PUPFISH continue to evaluate Evrysdi’s potential in combination therapies and its effectiveness in younger infants. Collectively, these trials have involved thousands of patients globally, providing a robust dataset that supports the drug’s approval for widespread use. The consistency of the clinical outcomes across diverse populations underscores the reliability of Evrysdi as a cornerstone of spinal muscular atrophy management.

How Does the New Evrysdi Tablet Fit into the Broader Landscape of SMA Treatment Options?

Prior to the introduction of Evrysdi, SMA treatment options were limited, often involving invasive procedures like spinal injections or gene therapies that required complex medical infrastructure. While these treatments have significantly improved outcomes for many patients, they also come with challenges related to accessibility, cost, and administration. The Evrysdi tablet represents a major shift toward non-invasive SMA treatment options that prioritize patient convenience without compromising on clinical effectiveness.

The tablet’s approval signifies a broader trend in rare disease management, where pharmaceutical companies are increasingly focusing on therapies that enhance both clinical outcomes and quality of life. Unlike gene therapies that require a one-time administration in a controlled setting, Evrysdi offers a daily, at-home treatment regimen that can be easily integrated into a patient’s routine. This flexibility is especially important for individuals with chronic conditions, as it reduces the need for frequent hospital visits and the associated healthcare costs.

Kenneth Hobby, President of Cure SMA, emphasized the real-world impact of this new formulation. He noted that simplifying treatment regimens can reduce the emotional and logistical burdens faced by families managing chronic conditions. The room temperature stability and ease of administration make the Evrysdi tablet an attractive option for both newly diagnosed patients and those already receiving spinal muscular atrophy therapy. This development aligns with the growing demand for patient-centric treatment options that support long-term disease management.

What’s Next for Genentech’s Evrysdi and the Future of SMA Care?

With more than 16,000 people treated globally, Evrysdi has already established itself as a cornerstone of SMA management. The Evrysdi tablet approval is expected to expand its reach even further, offering a flexible solution that meets the diverse needs of the SMA community. Genentech’s collaboration with the SMA Foundation and PTC Therapeutics has been instrumental in driving the clinical development of Evrysdi. This partnership continues to support ongoing research aimed at optimizing SMA treatment protocols and exploring new therapeutic combinations.

The Evrysdi tablet is expected to be available in the United States within the coming weeks, with updated prescribing information that includes detailed guidance on administration and storage. As spinal muscular atrophy research advances, the approval of innovative therapies like Evrysdi underscores the importance of patient-centered drug development in rare disease management. Future studies are likely to explore the long-term impact of Evrysdi on disease progression, quality of life, and overall survival, providing valuable insights that could further refine SMA treatment strategies.

The continued evolution of SMA therapies highlights the potential for scientific innovation to transform the lives of individuals living with rare diseases. The FDA’s approval of the Evrysdi tablet is not just a milestone for Genentech but a beacon of hope for the global spinal muscular atrophy community, reaffirming the power of medical research to drive meaningful change.