SMA – BiotechReality – Information Intelligence System for Biopharma

Novartis today announced that the U.S. Food and Drug Administration (FDA) has approved Itvisma® (onasemnogene abeparvovec-brve), a novel gene replacement therapy for the treatment of spinal muscular atrophy (SMA). The approval covers children two years of age and older, teenagers, and adults with a confirmed mutation in the SMN1 gene. This historic approval marks a significant milestone in the treatment of SMA, making Itvisma the first and only gene replacement therapy available for this broader patient population. Previously, gene therapy options were limited to infants under the age of two. A "Game-Changing" Advancement Itvisma is an intrathecal formulation (delivered directly into the spinal fluid) of the company's pioneering gene therapy, Zolgensma. While Zolgensma is administered intravenously and dosed by weight for infants, Itvisma is a one-time, fixed-dose injection designed to address the genetic root cause of SMA in larger, older patients. The FDA’s approval of intrathecal onasemnogene abeparvovec is a game-changing advance, expanding the use of transformational gene replacement therapy for SMA across age groups, This achievement is not only a significant step forward for SMA – it also signals new possibilities for the broader field of neurological disorders and genetic medicine. John W. Day Read More: Positive Outcomes From the Initial Prenatal Therapy for Spinal Muscular Atrophy Clinical Efficacy The FDA’s decision was based on robust data from two Phase 3 studies: STEER and STRENGTH. The STEER study evaluated treatment-naïve patients (those who had not received prior SMA therapy). Results showed statistically significant improvements in motor function compared to a sham control group. The STRENGTH study focused on patients who switched from other SMA therapies (such as Spinraza or Evrysdi). Data demonstrated that Itvisma stabilized motor abilities, preserving function in a disease typically characterized by progressive decline. Addressing the Root Cause SMA is a rare, genetic neuromuscular disease caused by a lack of the survival motor neuron (SMN) protein, which is essential for muscle function. Without it, patients experience progressive muscle weakness, affecting movement, breathing, and swallowing. Itvisma works by delivering a functional copy of the SMN1 gene to stop the progression of the disease and sustain SMN protein production. Safety and Availability Itvisma demonstrated a consistent safety profile across clinical trials. The most common adverse events included upper respiratory tract infection, pyrexia (fever), and headache. The label includes a Boxed Warning for hepatotoxicity (liver injury) and cardiotoxicity, consistent with the safety profile of Zolgensma. Novartis expects Itvisma to be available in the U.S. in the coming weeks. The wholesale acquisition cost is reported to be approximately $2.59 million for the one-time dose, a pricing strategy Novartis states is cost-effective compared to the cumulative cost of chronic, lifelong therapies. Information: Novartis

Tag: SMA

Novartis Receives FDA Approval for Itvisma, Expanding Gene Therapy to Older Children and Adults with SMA

Areas Covered

Discover More

GSK Secures Japan Orphan Drug Designation for Risvutatug Rezetecan in Small-Cell Lung Cancer

Pfizer’s Talzenna + Xtandi Delivers Proven rPFS Breakthrough in HRR-Mutated mCSPC

Eli Lilly Declares Powerful 2% A1C Reduction and 17% Weight Loss in Landmark Phase 3 Trial Results of Retatrutide

ICOTYDE FDA Approval: J&J’s First-Line Oral Peptide Breakthrough for Plaque Psoriasis Treatment

Atirmociclib Phase 2 Breakthrough: Pfizer Declares Powerful Results for Metastatic Breast Cancer Treatment

EBGLYSS (lebrikizumab-lbkz) Delivers Remarkable Phase 3 Results for Pediatric Atopic Dermatitis Treatment