FDA approves $2.1M gene therapy for spinal muscular atrophy
The Food and Drug Administration on Friday approved a $2.125 million treatment for spinal muscular atrophy, a genetic illness known as muscle-wasting disease that kills many babies before they turn 2. (Source: Health News - UPI.com)
Source: Health News - UPI.com - May 24, 2019 Category: Consumer Health News Source Type: news
FDA OKs First Gene Therapy for Spinal Muscular Atrophy FDA OKs First Gene Therapy for Spinal Muscular Atrophy
A single, one-time infusion of onasemnogene abeparvovec-xioi (Zolgensma) can minimize the progression of SMA and improve survival in children younger than age 2 years — and will cost over $2 million.FDA Approvals (Source: Medscape Pharmacist Headlines)
Source: Medscape Pharmacist Headlines - May 24, 2019 Category: Drugs & Pharmacology Tags: Neurology & Neurosurgery News Alert Source Type: news
FDA approves $2M medicine, most expensive ever
U.S. regulators approve gene therapy priced at $2.125 million US to treat a rare condition called spinal muscular atrophy. (Source: CBC | Health)
Source: CBC | Health - May 24, 2019 Category: Consumer Health News Tags: News/Health Source Type: news
Novartis ’ Spinal Drug Gets FDA Approval, $2 Million Price Tag
(CNN) — The US Food and Drug Administration approved a treatment Friday for a genetic disease called spinal muscular atrophy that causes infants’ muscles to waste away, potentially killing them before age 2.
And then came the price tag: $2.125 million for a one-time treatment.
The gene therapy, called Zolgensma, will be marketed by AveXis, whose parent company is Novartis.
“Today’s approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” Dr. Ned Sharpless, the FDA’s acting commissioner, said in a statement Friday. ̶...
Source: WBZ-TV - Breaking News, Weather and Sports for Boston, Worcester and New Hampshire - May 24, 2019 Category: Consumer Health News Authors: Health – CBS Boston Tags: Health News CNN Novartis Source Type: news
Gene therapy gets FDA approval -- and a $2 million price tag
The US Food and Drug Administration approved a treatment Friday for a genetic disease called spinal muscular atrophy that causes infants' muscles to waste away, potentially killing them before age 2. (Source: CNN.com - Health)
Source: CNN.com - Health - May 24, 2019 Category: Consumer Health News Source Type: news
Novartis wins approval for world’s most expensive drug
US FDA gives green light for $2.1m treatment of spinal muscular atrophy (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - May 24, 2019 Category: Pharmaceuticals Source Type: news
Novartis CEO dances around price tag talks for breakthrough gene therapy
Days before Novartis is expected to win approval for its breakthrough spinal muscular atrophy treatment, the drugmaker ' s CEO said he could justify charging up to $5 million for the revolutionary treatment — but that the goal is to set the cost at " a fraction of what is the current standard of care. " (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - May 24, 2019 Category: Pharmaceuticals Source Type: news
FDA Approves Zolgensma, Landmark AAV-Delivered Gene Therapy to Treat...
Zolgensma (Novartis, AveXis), an AAV-delivered gene therapy used to treat spinal muscular atrophy (SMA) also known as AVXS-101, was approved for clinical use in the United States by the Food and Drug...(PRWeb May 24, 2019)Read the full story at https://www.prweb.com/releases/fda_approves_zolgensma_landmark_aav_delivered_gene_therapy_to_treat_spinal_muscular_atrophy/prweb16337373.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - May 24, 2019 Category: Pharmaceuticals Source Type: news
Novartis CEO plans gene therapy price 'far lower' than $4 million to $5 million range
Novartis AG's top executive said on Wednesday it expects to price its gene therapy for spinal muscular atrophy “far lower” than the $4 million to $5 million figure the Swiss drugmaker has said it could be worth. (Source: Reuters: Health)
Source: Reuters: Health - May 22, 2019 Category: Consumer Health News Tags: healthNews Source Type: news
FDA poised to approve $5 MILLION gene therapy to treat muscular atrophy
The FDA is expected to approve Zolgensma, the world's second gene therapy for spinal muscular atrophy. Expert's worry that the two drugs for the disease are setting an expensive precedent. (Source: the Mail online | Health)
Source: the Mail online | Health - May 17, 2019 Category: Consumer Health News Source Type: news
NHS finally agrees a deal with pharmaceutical giant for a £450,000 'wonder drug'
NHS England has announced spinal muscular atrophy patients, like Finley Newell (pictured) will now be able to get access to Spinraza, which has been proven to improve strength. (Source: the Mail online | Health)
Source: the Mail online | Health - May 15, 2019 Category: Consumer Health News Source Type: news
NHS to fund drug that prolongs lives of children with muscle-wasting disease
Spinraza to be made available to spinal muscular atrophy patientsA drug that could prolong the lives of children with a rare muscle-wasting disease has been approved by the NHS in England after lengthy negotiations with the manufacturer over the high price.Spinraza could help between 600 and 1,200 children and adults in England and Wales who have the genetic condition spinal muscular atrophy (SMA). It affects the nerves in the spinal cord, making muscles weaker and causing problems with movement, breathing and swallowing. It can shorten the life expectancy of babies and toddlers.Continue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - May 15, 2019 Category: Science Authors: Sarah Boseley Health editor Tags: NHS Health Society UK news Pharmaceuticals industry Business Drugs Science Source Type: news
Spinal muscular atrophy: Spinraza approval delights family
A drug that can slow the effects of spinal muscular atrophy is approved for use on the NHS. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - May 15, 2019 Category: Consumer Health News Source Type: news
First treatment available for spinal muscular atrophy
NHS England negotiates deal on drug cost Related items fromOnMedica Clinicians need better options if we are to stop over-medication Call for NHS England to ‘think again’ on limiting treatment Green light for primary progressive MS drug Stroke survivors need more help taking medicine Resources wasted on low-risk blood pressure patients (Source: OnMedica Latest News)
Source: OnMedica Latest News - May 14, 2019 Category: UK Health Source Type: news
Exclusive: Novartis pitches discounts on pricey gene therapy for deadly muscle disorder
Novartis AG is offering price discounts in negotiations with U.S. health insurers on its gene therapy for spinal muscular atrophy (SMA), a treatment that could cost more than a million dollars, but the gesture comes with strings attached. (Source: Reuters: Health)
Source: Reuters: Health - May 10, 2019 Category: Consumer Health News Tags: healthNews Source Type: news
