Roche has announced a significant development in the treatment of
spinal muscular atrophy (SMA) with the approval of a tablet form of its drug,
Evrysdi (risdiplam), by the US Food and Drug Administration (FDA). Originally greenlit by the FDA in 2020 for patients aged two months and older, Evrysdi’s initial approval was expanded in 2022 to include infants under two months old. Traditionally available only as an oral solution taken daily at home, either by mouth or through a feeding tube, the introduction of this new tablet formulation represents a substantial advancement for patients and caregivers.
The new 5mg tablet is set to become available in the coming weeks and is suitable for patients aged two years and older who weigh over 20 kilograms.
Roche has stated that the original liquid form will still be accessible for those on different dosages or for patients who prefer the oral solution. This novel tablet form is expected to offer greater convenience by allowing storage at room temperature, and patients have the option to swallow the tablet whole or disperse it in water, providing more autonomy and flexibility in managing their condition.
SMA is a genetic disorder that affects approximately one in every 10,000 newborns globally. It is characterized by a mutation in the
SMN1 gene, leading to a deficiency in the SMN protein. This deficiency significantly impacts patients' physical abilities, including walking, eating, and breathing, depending on the severity of the disease. Evrysdi is engineered to address SMA by enhancing and maintaining the production of the SMN protein in both the central nervous system and peripheral tissues.
The FDA's recent decision marks Evrysdi as the first and only approved tablet for SMA, based on findings from a bioequivalence study. This study confirmed that the new tablet form provides a level of drug exposure comparable to that of the original oral solution, ensuring its effectiveness and safety.
Levi Garraway, Roche’s chief medical officer and head of global product development, emphasized the significance of this approval. He stated, “Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date. This approval marks another significant step forward. The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management.”
This advancement underscores Roche’s commitment to improving the quality of life for SMA patients through innovative treatment options. By offering a more convenient form of administration, the tablet version of Evrysdi is poised to enhance patient compliance and comfort, ultimately contributing to better health outcomes. As the medical community continues to strive for breakthroughs in genetic disorders, the approval of this tablet form of Evrysdi represents a noteworthy milestone in the ongoing battle against SMA.
How to obtain the latest research advancements in the field of biopharmaceuticals?
In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!
