AVXS-101 Managed Access Program – letter to the SMA community

Dear SMA Community,

Since its U.S. approval, AveXis has had increasing demand from families outside of the U.S. for access to AVXS-101. While we are pursuing registration in close to three dozen countries, we have heard the many requests from the SMA community for a global Managed Access Program (MAP)* to make this innovative gene therapy available to patients in immediate need.

We are pleased to take this critical step in offering a global MAP to provide AVXS-101 free of charge to eligible patients with SMA who are under the age of two and are a citizen or legal resident of a …

/by

Novartis provides update on LUSTER Phase III studies in patients with uncontrolled GINA 4/5 asthma

  • Pooled analyses of LUSTER 1 and 2 did not support further development of Fevipiprant in asthma as a primary indication
     
  • Fevipiprant was well tolerated with adverse events balanced across treatment groups

Basel, Switzerland, December 16, 2019 – Novartis today announced topline results from its pivotal global Phase III LUSTER-11 and LUSTER-22 studies exploring the efficacy and safety of the investigational oral, once-daily, DP2 receptor antagonist fevipiprant (QAW039). The …

/by

Novartis receives positive CHMP opinion for Beovu® (brolucizumab) for the treatment of wet AMD

  • In two head-to-head clinical trials, patients on Beovu achieved vision gains that were non-inferior to aflibercept at year one1
     
  • In a pre-specified secondary endpoint, fewer patients had intra-retinal fluid and/or sub-retinal fluid at week 16 and year one with Beovu1
     
  • In a key pre-specified secondary endpoint, over half of Beovu 6mg patients (56% in HAWK and 51% in HARRIER) were maintained on three-month dosing intervals immediately after the loading phase through year one1
      …
/by

Novartis Kisqali® data show superior overall survival compared to fulvestrant and consistent efficacy across advanced breast cancer patient subgroups in MONALEESA-3

  • MONALEESA-3 paper published today in The New England Journal of Medicine shows statistically significant overall survival results for Kisqali plus fulvestrant, reducing the risk of death by almost 30%
     
  • Overall survival benefit for Kisqali plus fulvestrant was consistent regardless of line of therapy or response to prior endocrine treatment
     
  • Kisqali is the only CDK4/6 inhibitor to demonstrate consistently superior overall survival in two Phase III trials, proven with multiple combination partners and in two …
/by

Novartis Kymriah® demonstrates consistent efficacy and safety outcomes in US patients when used in real-world setting

  • Efficacy in DLBCL confirmed results seen in the pivotal trial despite treatment of a broader population, including older and more heavily pretreated patients1-3
     
  • Fewer known CAR-T cell therapy adverse events for patients with DLBCL, specifically rates of high-grade cytokine release syndrome (4%) and neurologic events (5%), were observed compared with the pivotal clinical trials1-3
     
  • In children and young adults with ALL, efficacy outcomes were similar and safety outcomes appear to be …
/by

A decades-long journey in blood cancer research

It’s the dream of every medical scientist: when the years of relentless effort, minor victories, setbacks and challenges of research finally yield a breakthrough that may one day change the way patients are treated. Catherine Sabatos-Peyton, Director, Exploratory Immuno-Oncology at the Novartis Institutes for BioMedical Research (NIBR), recalls her role in the discovery of TIM-3, an immunotherapy target that may bring new hope to patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS).

…

/by

Uncovering profound and under-reported effects on people living with sickle cell disease

Novartis recently presented interim findings from the Sickle Cell World Assessment Survey (SWAY) at the 2019 American Society of Hematology (ASH) annual meeting this year, which highlighted new data around the global burden of the disease. Insights collected from over 2,100 patients and 360 health care providers evaluated the impact of the disease on patients and families, helping to inform the management of this debilitating disease.

Notably, patients reported a much higher number of vaso-occlusive crises (VOCs) than current published data, which suggests that VOCs may be …

/by

Novartis global survey uncovers profound and often under-reported effects on sickle cell disease patients

  • Findings from more than 2,000 respondents in 16 countries underscore the debilitating and under-reported effects of sickle cell disease and a pattern of not seeking care despite potentially life-threatening symptoms1
     
  • Patients reported on average more than 5 vaso-occlusive crises (VOCs) per year with serious impact on their quality of life (QoL), including emotional well-being and daily life, as well as ability to work and complete education1,2
     
  • Sickle Cell World Assessment Survey (SWAY), one of world’ …
/by

Novartis Phase III data on new inhaled dual combination QMF149 show significant improvement across key asthma outcomes versus monotherapy

  • Once-daily QMF149 met primary endpoint of lung function improvement and key secondary endpoint of asthma control improvement versus mometasone furoate1
     
  • QMF149 showed improvement in peak expiratory flow, exacerbation rates, rescue medication use versus mometasone furoate among other secondary endpoints1   
     
  • Improvement in lung function was observed in high dose QMF149 versus a high dose LABA/ICS standard-of-care in certain additional secondary endpoints1 

 

…

/by

Novartis R&D Day

Our drug discovery and development engine has the scope and scale to innovate the next generation of medicines. We’re harnessing powerful and advanced therapy platforms to take on big scientific challenges and find opportunities to develop breakthrough medicines for patients.

Learn more in our media release.

…
/by