Scholar Rock Reports the US FDA Approval of Isembyld (Apitegromab-mstn) to Treat Spinal Muscular Atrophy

Shots: The US FDA has approved Isembyld for the treatment of spinal muscular atrophy (SMA) in pts (≥2yrs.) who are currently receiving a SMN2-targeted treatment Approval was backed by the P-III (SAPPHIRE), which met its 1EP of 2.2-point improvement in HFMSE with Isembyld + SMN2-targeted therapy vs PBO at 1yr. in main efficacy population (n=103);…

ByRidhi RastogiSeptember 14, 2026

INSIGHTS+

EMA Marketing Authorization of New Drugs in July 2026

Shots: The European Commission (EC) approved multiple new medicines in July 2026 across rare diseases, respiratory disorders, aesthetics, and oncology, involving companies including Novartis, Boehringer Ingelheim, AbbVie’s Allergan Aesthetics, and AstraZeneca. EC approvals included Itvisma (onasemnogene abeparvovec) for patients aged ≥2 years with 5q spinal muscular atrophy (SMA) and confirmed bi-allelic SMN1 mutations; Jascayd (nerandomilast) for adults with idiopathic pulmonary…

ByDipanshu DixitAugust 18, 2026

NEWS

Novartis’ Itvisma Receives the EC Approval for Spinal Muscular Atrophy

Shots: The EC has approved Novartis' Itvisma (onasemnogene abeparvovec) for the treatment of pts (≥2yrs.) living with 5q spinal muscular atrophy (SMA) harboring a confirmed bi-allelic mutation in the SMN1 gene Approval was based on the P-III (STEER) trial & supportive P-IIIb (STRENGTH) & P-I/II (STRONG) studies, where STEER showed a 2.39-point improvement in the…

ByRidhi RastogiJuly 2, 2026

NEWS

Novartis’ Itvisma Receives the CHMP Positive Opinion for Spinal Muscular Atrophy

Shots: The CHMP has recommended Novartis' Itvisma (onasemnogene abeparvovec) for the treatment of pts (≥2yrs.) living with 5q spinal muscular atrophy (SMA) harboring a confirmed bi-allelic mutation in the SMN1 gene Opinion was based on the P-III (STEER) trial & P-IIIb (STRENGTH) study, where STEER showed a 2.39-point improvement in the HFMSE with effects sustained…

ByRidhi RastogiApril 27, 2026

NEWS

Novartis’ Itvisma Receives the US FDA Approval for Spinal Muscular Atrophy

Shots: The US FDA has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of pts (≥2yrs.) living with spinal muscular atrophy (SMA) harboring a confirmed mutation in the SMN1 gene; US availability expected in Dec 2025 Approval was based on the P-III (STEER) trial & open-label P-IIIb (STRENGTH) study, showing Itvisma improved motor function & stabilized…

ByRidhi RastogiNovember 25, 2025

NEWS

The EC Approves Roche’s Evrysdi Tablet for Spinal Muscular Atrophy

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 2024 Evrysdi, an SMN2 splicing modifier, treats SMA caused by…

ByRidhi RastogiJune 5, 2025

VIEWPOINTS

Biogen at WMS 2024: Stephanie Fradette from Biogen in an Illuminating Dialogue Exchange with PharmaShots

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. …

BySaurabh ChaubeyFebruary 19, 2025

NEWS

Scholar Rock Reports the US FDA’s BLA Submission for Apitegromab to Treat Spinal Muscular Atrophy (SMA)

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…

ByDipanshu DixitJanuary 30, 2025

INSIGHTS+

Disease of the Month – Spinal Muscular Atrophy

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…

BySaurabh ChaubeyAugust 17, 2023

VIEWPOINTS

Kathleen Somera-Molina, Head of SMA Global Medical Affairs at Biogen Shares Insights on New SPINRAZA (nusinersen) Long-Term Data in SMA

Shots: Kathleen spoke about new long-term SPINRAZA (nusinersen) data presented at the 3rd International Scientific Congress She 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 years The interview also discusses Biogen’s advances…

BySenior EditorAugust 7, 2023

Scholar Rock Reports the US FDA Approval of Isembyld (Apitegromab-mstn) to Treat Spinal Muscular Atrophy

EMA Marketing Authorization of New Drugs in July 2026

Novartis’ Itvisma Receives the EC Approval for Spinal Muscular Atrophy

Novartis’ Itvisma Receives the CHMP Positive Opinion for Spinal Muscular Atrophy

Novartis’ Itvisma Receives the US FDA Approval for Spinal Muscular Atrophy

The EC Approves Roche’s Evrysdi Tablet for Spinal Muscular Atrophy

Biogen at WMS 2024: Stephanie Fradette from Biogen in an Illuminating Dialogue Exchange with PharmaShots

Scholar Rock Reports the US FDA’s BLA Submission for Apitegromab to Treat Spinal Muscular Atrophy (SMA)

Disease of the Month – Spinal Muscular Atrophy

Kathleen Somera-Molina, Head of SMA Global Medical Affairs at Biogen Shares Insights on New SPINRAZA (nusinersen) Long-Term Data in SMA

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