Putting a Price on Life: Is the Answer Outside Pharma?
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 innovation is Neovasculgen (...
Source: EyeForPharma - November 4, 2019 Category: Pharmaceuticals Authors: Nicola Davies Source Type: news
Intrathecal Trials of AVXS-101 (Zolgensma) in SMA Halted Intrathecal Trials of AVXS-101 (Zolgensma) in SMA Halted
The partial hold on trials of intrathecal administration stem from safety concerns in animals and does not affect Zolgensma intravenous infusion, which is already approved for spinal muscular atrophy.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - November 1, 2019 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news
QOL burden substantial in spinal muscular atrophy
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - October 31, 2019 Category: Drugs & Pharmacology Source Type: news
Entresto Sales See Slow and Steady Upturn Entresto Sales See Slow and Steady Upturn
The heart failure drug is one of the key growth drivers for Novartis, along with the spinal muscular atrophy treatment, Zolgensma, which got off to a rocky start.Medscape Medical News (Source: Medscape Cardiology Headlines)
Source: Medscape Cardiology Headlines - October 25, 2019 Category: Cardiology Tags: Cardiology News Source Type: news
FDA Calls Out Drug Company for'Manipulated' Data FDA Calls Out Drug Company for'Manipulated' Data
AveXis knowingly submitted manipulated data in its application for Zolgensma, the first gene therapy approved for spinal muscular atrophy, the agency says; the product will remain on market for now.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - August 9, 2019 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news
Novartis C.E.O. Defends Company ’s Decision to Withhold False Data From the F.D.A.
Responding to the agency ’s stern rebuke, Vas Narasimhan, the company’s executive, tried to reassure investors that Novartis did not intentionally deceive the F.D.A. while seeking approval for its $2.1 million gene therapy. (Source: NYT Health)
Source: NYT Health - August 8, 2019 Category: Consumer Health News Authors: Katie Thomas Tags: Novartis AG Narasimhan, Vasant Food and Drug Administration Zolgensma Falsification of Data Drugs (Pharmaceuticals) Genetic Engineering Spinal Muscular Atrophy Source Type: news
FDA: Pricey Drug Approved on Manipulated Data
The FDA approved the gene therapy Zolgensma to treat children with a severe form of spinal muscular atrophy. The drug costs $2.125 million for a one-time treatment, CBS News reported. (Source: WebMD Health)
Source: WebMD Health - August 7, 2019 Category: Consumer Health News Source Type: news
Novartis Hid Manipulated Data While Seeking Approval for $2.1 Million Treatment
The failure to report the issue has not put patients at risk, the F.D.A. said, but the drugmaker could face criminal and civil penalties. (Source: NYT Health)
Source: NYT Health - August 6, 2019 Category: Consumer Health News Authors: Katie Thomas Tags: Novartis AG Food and Drug Administration Drugs (Pharmaceuticals) Spinal Muscular Atrophy Genetics and Heredity gene therapy your-feed-healthcare Source Type: news
FDA says Novartis gene therapy drug application contains manipulated data
On May 24, the FDA approved Zolgensma, a gene therapy product intended to treat children less than two years of age with spinal muscular atrophy, but was informed recently by the product ’s manufacturer about a data manipulation issue (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - August 6, 2019 Category: Pharmaceuticals Source Type: news
Lifesaving Drug for Infants Costs & #36;2.1 Million a Dose
MONDAY, July 29, 2019 -- Its extremely high price tag means that a lifesaving medication to treat young children with spinal muscular atrophy is simply too expensive for most families.
A single dose of Zolgensma costs $2.1 million, the highest... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - July 29, 2019 Category: Pharmaceuticals Source Type: news
Biogen downplays ‘premature’ talk of competition for SMA drug Spinraza
During an earnings call Tuesday morning, executives at Biogen Inc. brushed off investor concerns about competitors closing in on its key drugs, particularly its treatment approved less than three years ago for spinal muscular atrophy.
Much of the focus of the quarterly earnings report for the three-month period that ended in June was on Spinraza, Biogen’s (Nasdaq: BIIB) SMA drug. The company’s $3.6 billion in second quarter revenue (an 8 percent increase from the same quarter last year) was… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - July 23, 2019 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news
Biotech group Biogen boosted by sales of key drugs
Treatments for multiple sclerosis and spinal muscular atrophy help Biogen beat forecasts (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - July 23, 2019 Category: Pharmaceuticals Source Type: news
Parents of son diagnosed with crippling disease fight for mandatory genetic screening
Shane Philipps, 17 months, from Haddon Heights, New Jersey, was diagnosed at 10 months, with Spinal Muscular Atrophy, a disease which weakens the physical muscles. (Source: the Mail online | Health)
Source: the Mail online | Health - July 10, 2019 Category: Consumer Health News Source Type: news
Campaign girl 'ineligible' for muscle wasting drug
The 11-year-old is thought to be in 15% of spinal muscular atrophy patients who do not qualify. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - June 19, 2019 Category: Consumer Health News Source Type: news
Do You Consider Cost When Prescribing a Drug? Do You Consider Cost When Prescribing a Drug?
A newly approved gene therapy for children with spinal muscular atrophy will cost more than $2 million for a one-time infusion.Medscape Reader Polls (Source: Medscape Business of Medicine Headlines)
Source: Medscape Business of Medicine Headlines - June 12, 2019 Category: Pharmaceuticals Tags: Internal Medicine News Source Type: news
