FDA Approves Genentech's Evrysdi as First Tablet for Spinal Muscular Atrophy

Genentech, a part of the Roche Group, has announced that the U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of Evrysdi® (risdiplam) for treating spinal muscular atrophy (SMA). This new 5 mg tablet formulation offers a non-invasive, disease-modifying treatment option for individuals living with SMA and can be taken either as a whole tablet or dispersed in water. This advancement marks a significant milestone in the treatment of SMA, providing a convenient administration method without compromising the established efficacy and safety of the original oral solution.

Dr. Levi Garraway, Genentech's chief medical officer, emphasized the potential of Evrysdi to significantly alter the course of SMA. He noted that the introduction of this tablet form represents a meaningful step forward, offering a flexible and convenient option for managing the condition. The approval is based on a bioequivalence study demonstrating that the tablet form provides similar exposure to risdiplam as the oral solution, ensuring that patients receive consistent treatment outcomes regardless of the formulation they choose.

Kenneth Hobby, President of Cure SMA, highlighted the importance of simplifying treatment administration for those affected by SMA. He pointed out that the room temperature-stable tablet formulation could seamlessly integrate into daily activities, such as work, travel, and education, enhancing the quality of life for patients and their caregivers.

The updated prescribing information for Evrysdi now includes guidance on administering and storing the tablet form. The new tablet is expected to be available shortly and is suitable for individuals aged 2 years and older who weigh more than 44 pounds (20 kilograms).

Evrysdi, a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier, is designed to increase and sustain SMN protein production, crucial for the health of motor neurons. This medication addresses SMA, which is caused by mutations in the SMN1 gene, leading to a deficiency in the SMN protein. This protein is essential for the proper functioning of nerves controlling muscle movement, and its absence results in progressive muscle weakness.

Evrysdi's efficacy and safety have been demonstrated in multiple clinical trials, including FIREFISH, SUNFISH, JEWELFISH, RAINBOWFISH, and MANATEE, among others. These trials have shown positive outcomes across various age groups and types of SMA, underscoring the drug's potential to improve the lives of those affected by this debilitating condition. The medication has received approvals in over 100 countries and has been administered to more than 16,000 individuals worldwide.

Spinal muscular atrophy is a severe neuromuscular disease that can lead to significant disability or even death. Affecting approximately one in 10,000 babies, SMA is one of the leading genetic causes of infant mortality. The introduction of Evrysdi's tablet formulation provides a promising new treatment avenue, making disease management more accessible and manageable for patients and families dealing with SMA.

Genentech remains at the forefront of neuroscience research and development, committed to pioneering therapies for neurological disorders. In collaboration with various partners, the company is actively pursuing innovative solutions to address the complex challenges posed by diseases such as SMA, multiple sclerosis, Alzheimer's, and Parkinson's disease.

With over 40 years of experience, Genentech continues to be a leader in biotechnology, dedicated to advancing medical science and improving patient outcomes across a range of serious and life-threatening conditions.

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