Tag Archives: spinal muscular atrophy

December, 2018

5 December
FDA Grants Priority Review to Novartis’ AVXS-101, a One-Time Treatment Designed to Address the Genetic Root Cause of SMA Type 1

Basel, December 3, 2018 – Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA® (onasemnogene abeparvovec-xxxx)[1], an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the …
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20 December
Biogen and Ionis Collaborate to Identify Novel Therapies for Treatment of Spinal Muscular Atrophy

CAMBRIDGE, Mass. & CARLSBAD, Calif.–(BUSINESS WIRE)–Biogen (Nasdaq: BIIB) and Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that they have entered into a new collaboration agreement to identify new antisense oligonucleotide drug candidates for the treatment of spinal muscular atrophy (SMA). Biogen will have the option to license therapies arising out …
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6 October
Biogen’s Spinraza Data Show Initiating Treatment Earlier May Improve Motor Function in SMA

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Biogen (NASDAQ: BIIB) presented new data demonstrating that earlier initiation of treatment with SPINRAZA® (nusinersen) may improve motor function outcomes in infants and children with spinal muscular atrophy (SMA). Results continued to reinforce the favorable efficacy and safety profile of SPINRAZA. The data were shared at the 22nd International Annual Congress …
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17 March
AveXis’ Gene Therapy for SMA Demonstrates Positive Results

CHICAGO, March 16, 2017 (GLOBE NEWSWIRE) — AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today reported topline results from the Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1. The company also reported financial …
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16 January
Biogen’s Spinraza Significantly Reduces Risk of Death or Permanent Ventilation in Infantile-Onset SMA

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Biogen presented new data from the Phase 3 ENDEAR study of SPINRAZA™ (nusinersen), which demonstrated a statistically significant reduction in the risk of death or permanent ventilation in SPINRAZA-treated infants with spinal muscular atrophy (SMA) compared to untreated infants. The data were presented at the British Paediatric Neurology …
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28 December
FDA Approves Biogen’s Spinraza, the First Treatment for Spinal Muscular Atrophy

CAMBRIDGE, Mass.–(BUSINESS WIRE)–The U.S. Food and Drug Administration (FDA) approved Biogen’s (NASDAQ: BIIB) SPINRAZA™ (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is the first and only treatment approved in the U.S. for SMA, a leading genetic cause of death in infants …
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22 July
FDA Grants Breakthrough Therapy Designation to AveXis’ Gene Therapy

CHICAGO, July 20, 2016 (GLOBE NEWSWIRE) — AveXis, Inc., (Nasdaq:AVXS) a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS-101, the company’s lead development candidate for the …
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16 January
Roche Expands its Rare Disease Portfolio with Trophos Acquisition

Swiss drugmaker Roche has signed an agreement to acquire privately-held Trophos for up to $545 million. The acquisition will provide Roche with Trophos’ candidate for the rare and debilitating spinal muscular atrophy (SMA). Trophos’ proprietary screening platform generated olesoxime (TRO19622), which is in mid-stage development for SMA. SMA is a …
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