Roche ’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy
Basel, 06 February 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from the pivotal Part 2 of SUNFISH, a global placebo-controlled study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study showed that change from baseline in the primary endpoint of the Motor Function Measure scale (MFM-32)1 was significantly greater in people treated with risdiplam, compared to placebo (1.55 point mean difference; p=0.0156). The Revised Upper Limb Module (RULM),2 a key secondary endpoint, also showed an improvement (1.59 point difference; p=0.0028). Safety for risdi...
Source: Roche Media News - February 6, 2020 Category: Pharmaceuticals Source Type: news

Roche ’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy
Basel, 6 February 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from the pivotal Part 2 of SUNFISH, a global placebo-controlled study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study showed that change from baseline in the primary endpoint of the Motor Function Measure scale (MFM-32)[1]was significantly greater in people treated with risdiplam, compared to placebo (1.55 point mean difference; p=0.0156). The Revised Upper Limb Module (RULM),[2] a key secondary endpoint, also showed an improvement (1.59 point difference; p=0.0028). Safety for ris...
Source: Roche Investor Update - February 6, 2020 Category: Pharmaceuticals Source Type: news

Reminder: Invitation to Roche ’s live audio webcast on efficacy and safety of risdiplam in patients with type 2/3 spinal muscular atrophy presented at SMA Europe 2020
  (Source: Roche Investor Update)
Source: Roche Investor Update - January 31, 2020 Category: Pharmaceuticals Source Type: news

Roche's SMA drug trial win bolsters $2 billion sales prospects
Roche's spinal muscular atrophy (SMA) drug risdiplam hit another clinical trial target, the Swiss drugmaker said on Thursday, which analysts said bolsters its prospects of reaching $2 billion in annual sales. (Source: Reuters: Health)
Source: Reuters: Health - January 23, 2020 Category: Consumer Health News Tags: healthNews Source Type: news

Roche ’s Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with type 1 spinal muscular atrophy
             Basel, 23 January 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA). The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12-months of treatment, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Safety for risdiplam in the FIREFISH study was con...
Source: Roche Investor Update - January 23, 2020 Category: Pharmaceuticals Source Type: news

Roche ’s Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with type 1 spinal muscular atrophy
             Basel, 23 January 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA). The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12-months of treatment, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Safety for risdiplam in the FIREFISH study was con...
Source: Roche Media News - January 23, 2020 Category: Pharmaceuticals Source Type: news

Invitation to Roche ’s live audio webcast on efficacy and safety of risdiplam in patients with type 2/3 spinal muscular atrophy presented at SMA Europe 2020
  (Source: Roche Investor Update)
Source: Roche Investor Update - January 17, 2020 Category: Pharmaceuticals Source Type: news

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)
Source: Reuters: Health - January 14, 2020 Category: Consumer Health News Tags: healthNews Source Type: news

Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community. (Source: The Scientist)
Source: The Scientist - January 7, 2020 Category: Science Tags: News & Opinion Source Type: news

Spinraza funding recommended for spinal muscular atrophy in NZ
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - January 1, 2020 Category: Drugs & Pharmacology Source Type: news

Novartis in talks with patients upset about lottery-like gene therapy giveaway
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)
Source: Reuters: Health - December 20, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Novartis plans to give away world's costliest therapy to some patients
Novartis aims to give away 100 doses of its $2.1 million-per-patient Zolgensma for spinal muscular atrophy (SMA) in 2020 in a free-drug program that one patient group worried was a "health lottery" that could neglect some babies. (Source: Reuters: Health)
Source: Reuters: Health - December 19, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
The animals lived longer and showed milder symptoms than untreated mice, although they didn't survive as long as wildtype mice. (Source: The Scientist)
Source: The Scientist - December 11, 2019 Category: Science Tags: News & Opinion Source Type: news

Novartis R & D boss says doesn't see big opportunity in oral SMA therapy
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)
Source: Reuters: Health - December 5, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Novartis's $90 million Swiss factory to help solve cell therapy bottleneck
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)
Source: Reuters: Health - November 28, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

FDA grants priority review to Roche ’s risdiplam for spinal muscular atrophy
Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for SMA. Risdiplam is designed to increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body. (Source: World Pharma News)
Source: World Pharma News - November 27, 2019 Category: Pharmaceuticals Tags: Featured Roche Business and Industry Source Type: news

Roche eyes quick FDA approval in SMA race with Biogen, Novartis
Roche expects its risdiplam medicine for spinal muscular atrophy (SMA) to get U.S. approval by May, the Swiss drugmaker said on Monday, as it takes on Novartis and Biogen in the lucrative rare disease area. (Source: Reuters: Health)
Source: Reuters: Health - November 25, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

FDA grants priority review to Roche ’s risdiplam for spinal muscular atrophy
Roche today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for SMA. (Source: Roche Media News)
Source: Roche Media News - November 25, 2019 Category: Pharmaceuticals Source Type: news

FDA grants priority review to Roche ’s risdiplam for spinal muscular atrophy
Roche today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for SMA. (Source: Roche Investor Update)
Source: Roche Investor Update - November 25, 2019 Category: Pharmaceuticals Source Type: news

SUNFISH: Risdiplam Meets Primary Endpoint in SMA SUNFISH: Risdiplam Meets Primary Endpoint in SMA
Risdiplam, an oral investigational agent designed to increase and sustain SMN protein levels of patients with spinal muscular atrophy (SMA), met the primary endpoint in the pivotal part 2 of the SUNFISH trial.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - November 13, 2019 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news

Roche closer to breaking into lucrative SMA market with positive trial results
Positive trial results for an oral drug to treat spinal muscular atrophy (SMA) are bringing Roche one step closer to breaking into the high-cost treatment area. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - November 11, 2019 Category: Pharmaceuticals Source Type: news

Roche drug for spinal muscular atrophy hits target in late-stage trial
Roche's bid to rival Biogen and Novartis in treating spinal muscular atrophy (SMA) got a lift on Monday when the Swiss drugmaker said its drug risdiplam improved motor function in a key study. (Source: Reuters: Health)
Source: Reuters: Health - November 11, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Roche sets sights on Novartis, Biogen as SMA trial hits target
Roche's bid to rival Biogen and Novartis in treating spinal muscular atrophy (SMA) got a lift on Monday when the Swiss drugmaker said its drug risdiplam improved motor function of patients in a key study. (Source: Reuters: Health)
Source: Reuters: Health - November 11, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Roche ’s risdiplam meets primary endpoint in pivotal SUNFISH trial in people with type 2 or 3 spinal muscular atrophy
Roche today announced positive data from the pivotal Part 2 of SUNFISH, a study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). (Source: Roche Media News)
Source: Roche Media News - November 11, 2019 Category: Pharmaceuticals Source Type: news

Roche ’s risdiplam meets primary endpoint in pivotal SUNFISH trial in people with type 2 or 3 spinal muscular atrophy
Roche today announced positive data from the pivotal Part 2 of SUNFISH, a study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). (Source: Roche Investor Update)
Source: Roche Investor Update - November 11, 2019 Category: Pharmaceuticals Source Type: news

Nusinersen improves motor function in adults with spinal muscular atrophy
(IOS Press) A study published in the Journal of Neuromuscular Diseases presents the first evidence of mild improvement or stabilization of motor and respiratory function in adults with spinal muscular atrophy type 3 (SMA3) treated with Nusinersen, which was the case even in patients who have had the disease for 20 years or more. These findings prove the efficacy of Nusinersen beyond types and age groups, paving the way for adult treatment. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 11, 2019 Category: International Medicine & Public Health Source Type: news

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 ...
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 - November 1, 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 7, 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

Medical News Today: Medical conditions that cause muscle wasting
Conditions that can cause muscle wasting include muscular dystrophy, multiple sclerosis, and spinal muscular atrophy. Treatment may include dietary changes and exercise. Learn more here. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - June 11, 2019 Category: Consumer Health News Tags: Muscular Dystrophy / ALS Source Type: news

New Gene Therapy Poised to Transform Care for Spinal Muscular Atrophy
The University of Rochester Medical Center has been tapped as one of the first institutions in the U.S. to offer a new gene replacement therapy to treat spinal muscular atrophy. The new treatment, which is being marketed by Novartis under the brand name Zolgensma, can be delivered within weeks of birth and clinical trials have shown that it dramatically changes the course of the disease. (Source: University of Rochester Medical Center Press Releases)
Source: University of Rochester Medical Center Press Releases - June 6, 2019 Category: Universities & Medical Training Authors: University of Rochester Medical Center Source Type: news

Monthly News Roundup - May 2019
Zolgensma: First Gene Therapy for Pediatric Spinal Muscular Atrophy (SMA) Zolgensma (onasemnogene abeparvovec-xioi) from AveXis (a Novartis company), is now approved for children less than 2 years of age with spinal muscular atrophy (SMA). SMA... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - June 1, 2019 Category: Pharmaceuticals Source Type: news

Newly Approved Spinal Muscular Atrophy Gene Therapy, Zolgensma ® ,...
AskBio continues to lead the path for gene therapy development and cost-effective manufacturing methods based on the contributions of its co-founder, Dr. Jude Samulski.(PRWeb May 30, 2019)Read the full story at https://www.prweb.com/releases/newly_approved_spinal_muscular_atrophy_gene_therapy_zolgensma_validates_askbio_gene_therapy_platform/prweb16344912.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - May 30, 2019 Category: Pharmaceuticals Source Type: news

FDA approves most expensive gene therapy ever — and here is its connection to Penn
The Food and Drug Administration last week approved a gene therapy product — which immediately became the country's most expensive drug at $2.125 million for the one-time treatment — that incorporates a gene delivery technology developed at the the University of Pennsylvania. Novartis received FDA approval for Zolgensma, a one-time infusion for pediatric patients with spinal muscular atrophy (SMA) who are less than two years of age. SMA is caused by a mutation in a gene, known as the survival… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - May 28, 2019 Category: Biotechnology Authors: John George Source Type: news

FDA Approves First Gene Therapy Zolgensma (Onasemnogene Abeparvovec-xioi) for Spinal Muscular Atrophy
TUESDAY, May 28, 2019 -- The first gene therapy has been approved to treat children younger than 2 years with spinal muscular atrophy (SMA), the U.S. Food and Drug Administration announced Friday. Zolgensma (onasemnogene abeparvovec-xioi), an... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - May 28, 2019 Category: General Medicine Source Type: news

FDA Approves First Gene Therapy for Spinal Muscular Atrophy
TUESDAY, May 28, 2019 -- The first gene therapy has been approved to treat children younger than 2 years with spinal muscular atrophy (SMA), the U.S. Food and Drug Administration announced Friday. Zolgensma (onasemnogene abeparvovec-xioi), an... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - May 28, 2019 Category: Pharmaceuticals Source Type: news

FDA Approves Gene Therapy for Spinal Muscular Atrophy
At $2 million for a single dose, Novartis ’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease. (Source: The Scientist)
Source: The Scientist - May 27, 2019 Category: Science Tags: News & Opinion Source Type: news

Girl, 15, delighted because NHS made a life-changing drug available
Abbie Bolt, 15, from Minster in Kent, suffers from spinal muscular atrophy (SMA) and has to be hooked up to a machine throughout the night to help her breathe. Pictured with mother Mel. (Source: the Mail online | Health)
Source: the Mail online | Health - May 27, 2019 Category: Consumer Health News Source Type: news