Shots:The EC has approved label extension of Evrysdi (5mg oral tablet) for pts with spinal muscular atrophy (SMA)Approval was based on P-I study assessing Evrysdi 5mg tablet vs oral solution, showing bioequivalence with comparable efficacy & safety; data was presented at SMA Europe 2024Evrysdi, an SMN2 splicing modifier, treats SMA caused by…
Shots: Biogen recently shared findings from the P-II/III DEVOTE study, which investigated the safety and efficacy of a higher dose regimen of Spinraza for the treatment of spinal muscular atrophy (SMA) Stephanie Fradette, Head of Neuromuscular Development Unit at Biogen, joins PharmaShots for a stimulating conversation on Biogen's groundbreaking presentation at World Muscle Society 2024. …
Shots:The US FDA received BLA, plus a request for a priority review of apitegromab to improve motor function in pts with SMA who are on SMN-targeted therapy. The EMA’s MAA to be filed in Q1’25
The BLA was supported by the P-III (SAPPHIRE) & P-II (TOPAZ) trial, where SAPPHIRE met its 1EP of…
Shots:To keep our readers acquainted with several disease conditions, ongoing trials, and available treatment options, PharmaShots brings every month a detailed take on a particular disease after thorough research Continuing the series for the disease of the month, PharmaShots brings this month a summary of Spinal Muscular Atrophy, a genetic neuromuscular disease that affects…
Shots:Kathleen spoke about new long-term SPINRAZA (nusinersen) data presented at the 3rd International Scientific CongressShe then explained the new findings from individuals with infantile-onset SMA who were treated with SPINRAZA in the pivotal ENDEAR study followed by the long-term extension SHINE study for up to 7 yearsThe interview also discusses Biogen’s advances…
Shots:Kathleen spoke about the results of a new study published in Advances in Therapy evaluating real-world adherence to SPINRAZA (nusinersen) with its unique dosing schedule among people with spinal muscular atrophy (SMA)She also talked about the overall adherence rates to nusinersen. She highlighted that adherence was higher during the maintenance phase than the loading phase…
In an interview with PharmaShots, Toby Ferguson, Vice President & Head of the Neuromuscular Development Unit at Biogen shared his views on new data on Spinraza and improves clinical outcomes for the treatment of SMA patientsShots:The first patient has been treated in the P-IIIb (ASCEND) study evaluating the higher dose of nusinersen in 135 children, teens & adults with…

