“She was the picture of health,” recalls Andrea Myers, a cancer physician and scientist at Novartis. Seven years ago, Myers’ young and fit former medical school classmate was diagnosed – inexplicably – with lung cancer. She died two years later. 

A lung cancer diagnosis often comes as a shock. The disease tends to progress silently, with few symptoms. For most people it is not discovered until it has already advanced. At this point, the disease is very difficult to treat. Fewer than 20% of patients survive more than five years.

Innovative medicines have helped, but lung cancer …

Your body has a built-in sensor that self-assembles inside cells in response to a threat. This danger-sensing system is a large piece of cellular machinery, but until 20 years ago, no one knew it existed.

Today, however, scientists have learned that this system can get stuck in the “on” position. When it does, it could fuel the flames of diseases such as cancer. 

What if we could inhibit an overactive danger-sensing system? Medicines that target this system have the potential to help stop damaging …

  • All children (100%) treated presymptomatically in the SPR1NT two-copy cohort survived without respiratory or nutritional support, and sat independently for ≥30 seconds, most (11/14) within the WHO window of expected normal development
  • The majority of children (82%) treated in STR1VE-EU achieved developmental motor milestones not observed in the natural history of SMA Type 1, including patients with more severe disease
  • More than 1,200 patients have now been treated with Zolgensma globally across …

Novartis announced today that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) to 177Lu-PSMA-617, an investigational radioligand therapy for the treatment of metastatic castration-resistant prostate cancer (mCRPC).  Breakthrough Therapy designation is granted to medicines being evaluated for serious conditions where early clinical evidence indicates the potential for substantial improvement over available therapy1.

  • Breakthrough therapy designation granted based on positive data from the pivotal, Phase III VISION …
  • First-in-class, oral, targeted factor B inhibitor iptacopan substantially reduced both intra- and extravascular hemolysis when given as monotherapy in a Phase II study of anti-C5 naïve paroxysmal nocturnal hemoglobinuria (PNH) patients1

  • New results are promising for potential use of iptacopan as monotherapy in PNH, a rare and life-threatening blood disorder2,3; results from a previous Phase II study showed iptacopan substantially improved hematological response as add-on to standard-of-care (eculizumab …

Novartis today announced that Entresto® (sacubitril/valsartan) has received a new indication in China for the treatment of patients with essential hypertension, a disease affecting approximately 245 million adults in the country1. This National Medical Products Administration (NMPA) decision follows the 2017 approval of Entresto for the treatment of heart failure with reduced ejection fraction (HFrEF). 

  • The prevalence of hypertension in China is expected to rise to approximately 300 million2 by 2025 with control rates generally lower than in other parts of …
  • Data shows investigational iptacopan improved estimated glomerular filtration rate (eGFR) slope and stabilized kidney function in patients with C3G1; Phase III clinical trial to start in 2021
  • Results add to body of information on iptacopan, with previous interim analysis data from same trial showing significant reduction in proteinuria, improved plasma C3 levels, and favorable safety and tolerability profile over 12 weeks2
  • There …
  • Phase II primary endpoint results for investigational iptacopan in IgAN demonstrated effective and clinically meaningful reduction of proteinuria1 – a key risk predictor in kidney disease progression2

  • Iptacopan also showed a trend toward stabilization of kidney function1;
  • Tabrecta® (capmatinib) showed a median overall survival (OS) of 20.8 months in treatment-naïve patients and 13.6 months in previously-treated patients in first published mature data1,2
  • Tabrecta achieved 65.6% overall response rate (ORR) in first-line and 51.6% in second-line settings in new expansion cohort analysis of additional patients1,2
  • Patient-reported outcomes on quality-of-life symptoms were also presented
  • Tislelizumab demonstrated a 30% reduction in the risk of death and extended median overall survival by 2.3 months compared to chemotherapy in advanced or metastatic esophageal squamous cell carcinoma after prior systemic therapy1
  • Additional Phase II data presented at ASCO showed tislelizumab demonstrated durable anti-tumor activity in patients with microsatellite instability-high, or mismatch-repair-deficient, solid tumors2

Basel, June 4, 2021 — Novartis announced today results from …