Roche aims to 'underwhelm' on SMA drug price to challenge rivals

Swiss drugmaker Roche plans to price its oral spinal muscular atrophy drug (SMA) risdiplam aggressively to challenge two of the world's most expensive medicines, Biogen's Spinraza and Novartis's gene therapy Zolgensma.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news

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CONCLUSIONS: This proof-of-concept study is the first attempt to explore SMA in a comprehensive manner from the perspective of the ICF-CY using a selected set of codes. These codes define essential child dimensions that can make up an ICF-CY core set, as identified by a trained multidisciplinary team, to guide assessment, treatment and rehabilitation. CLINICAL REHABILITATION IMPACT: Although limited to a single patient, this study makes nonetheless a strong point: we suggest using the ICF-CY as an essential tool in SMA management at a time when gene therapy with nusinersen is changing the phenotypes of activity and fu...
Source: European Journal of Physical and Rehabilitation Medicine - Category: Rehabilitation Authors: Tags: Eur J Phys Rehabil Med Source Type: research
The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community.
Source: The Scientist - Category: Science Tags: News & Opinion Source Type: news
Novartis is in discussion with patient groups over its lottery-style free drug program for its multi-million-dollar gene therapy for spinal muscular atrophy (SMA) after criticism that the process could be unfair to some babies with the deadly disease.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Publication date: Available online 11 December 2019Source: Pharmacology &TherapeuticsAuthor(s): Natalie F. Nidetz, Michael C. McGee, Longping V. Tse, Chengwen Li, Le Cong, Yunxing Li, Weishan HuangAbstractAdeno-associated viral (AAV) vectors have emerged as the leading gene delivery platform for gene therapy and vaccination. Three AAV-based gene therapy drugs, Glybera, LUXTURNA, and ZOLGENSMA were approved between 2012 and 2019 by the European Medicines Agency and the United States Food and Drug Administration as treatments for genetic diseases hereditary lipoprotein lipase deficiency (LPLD), inherited retinal disease ...
Source: Pharmacology and Therapeutics - Category: Drugs & Pharmacology Source Type: research
The animals lived longer and showed milder symptoms than untreated mice, although they didn't survive as long as wildtype mice.
Source: The Scientist - Category: Science Tags: News & Opinion Source Type: news
Novartis research head Jay Bradner said on Thursday the Swiss drugmaker is focusing drug development efforts against spinal muscular atrophy on gene therapy Zolgensma and retreating from oral therapy like its molecule LMI070, also called branaplam.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Abstract Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is a rare autosomal recessive neuromuscular disorder caused by mutations in the IGHMBP2 gene, which encodes immunoglobulin μ-binding protein 2, leading to progressive spinal motor neuron degeneration. We review the data available in the literature about SMARD1. The vast majority of patients show an onset of typical symptoms in the first year of life. The main clinical features are distal muscular atrophy and diaphragmatic palsy, for which permanent supportive ventilation is required. No effective treatment is available yet, but novel the...
Source: J Cell Mol Med - Category: Molecular Biology Authors: Tags: J Cell Mol Med Source Type: research
Swiss drugmaker Novartis's new $90 million cell and gene therapy factory in northern Switzerland is on track to begin commercial production of treatments, including Kymriah for cancer and Zolgensma for spinal muscular atrophy, in 2020.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
 To cure the previously incurable we must take two giant steps: the first towards better understanding the science behind a cure, and the second towards ensuring accessibility for those people in need of the treatment.   Although there is still much to discover, we got the science right some time ago. The first gene replacement therapy – Gendicine (Shenzhen SiBiono GeneTech) – was approved in China in 2003 and has been successfully administered to more than 30,000 patients with head and neck squamous cell carcinoma. A single dose of this medication costs $400.  Another example of early ...
Source: EyeForPharma - Category: Pharmaceuticals Authors: Source Type: news
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder due to mutations in the SMN1 gene, causing degeneration of motor neurons in the spinal cord, with subsequent muscle wasting and weakness. In the last few years there have been exciting advances with revised standards of care and clinical trials using different therapeutic approaches such as antisense oligonucleotides [1,2], small molecules or gene therapy [3], showing increased life expectancy and functional and respiratory improvements.
Source: Neuromuscular Disorders - Category: Neurology Authors: Tags: Review Source Type: research
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