Novartis receives US Food and Drug Administration (FDA) Orphan Drug Designation for branaplam (LMI070) in Huntington’s disease (HD)

  • Huntington’s disease is a rare, inherited neurodegenerative disease that leads to progressive disability and death

  • There are no approved disease modifying therapies that delay disease onset or slow progression of the disease

  • Branaplam (LMI070) is an orally administered, small molecule RNA splicing modulator that could potentially reduce the levels of mutant huntingtin protein

Basel, October …

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Novartis receives positive CHMP opinion for Leqvio®* (inclisiran), a potential first-in-class siRNA for the treatment of high cholesterol

  • If approved, inclisiran will be the first and only small interfering RNA (siRNA) in Europe for patients with hypercholesterolemia or mixed dyslipidemia1
  • Cardiovascular disease (CVD) claims 3.9 million lives annually in Europe2, and 80% of high-risk patients do not reach guideline-recommended low-density lipoprotein cholesterol (LDL-C) targets despite the widespread …
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Novartis received European Medicines Agency (EMA) PRIME designation for iptacopan (LNP) in C3 glomerulopathy (C3G)

  • The European Medicines Agency has granted iptacopan a priority medicines (PRIME) designation in C3 glomerulopathy (C3G). 
  • PRIME is granted for medicines that may offer major therapeutic advance or benefit patients without treatment options.
  • C3 glomerulopathy (C3G) is a rare renal disease, affecting young patients with a poor prognosis and significant unmet need.1 …
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Meet a visionary who beat the odds to realize his vision of inclusive eye care

In Rwanda, it is estimated that more than 65,000 people (0.6% of the population) are blind in both eyes and that 12% of the population have a correctable refractive error or blurred vision, requiring corrective lenses.1

In this country of more than 12 million residents, there are only 18 ophthalmologists.

As one of those ophthalmologists, and the only one within a catchment area of 1.2 million people from Rwanda and nearby countries, Dr. Theophile Tuyisabe has an enormous responsibility. Each day he commutes an hour to the Eye Unit at the Kabgayi Hospital, treats …

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Meet a visionary who beat the odds to realize his vision of inclusive eye care

In Rwanda, it is estimated that more than 65,000 people (0.6% of the population) are blind in both eyes and that 12% of the population have a correctable refractive error or blurred vision, requiring corrective lenses.1

In this country of more than 12 million residents, there are only 18 ophthalmologists.

As one of those ophthalmologists, and the only one within a catchment area of 1.2 million people from Rwanda and nearby countries, Dr. Theophile Tuyisabe has an enormous responsibility. Each day he commutes an hour to the Eye Unit at the Kabgayi Hospital, treats …

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New Phase III analysis demonstrates Novartis Beovu® showed improvement in best-corrected visual acuity in wet AMD patients with early persistent fluid

  • In a post-hoc analysis of HAWK and HARRIER, fewer Beovu (brolucizumab) patients had early persistent fluid (12.5% vs. 20.4% of aflibercept patients), defined as the presence of intra-retinal fluid and/or sub-retinal fluid through week 12 of treatment1
  • Patients with early persistent fluid treated with Beovu experienced greater gains in best-corrected visual acuity (BCVA) at week 96 …
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Zolgensma® data including patients with more severe SMA at baseline further demonstrate therapeutic benefit, including prolonged event-free survival, increased motor function and milestone achievement

  • Nearly two-thirds of patients (65.6%) in STR1VE-EU have already achieved developmental motor milestones not observed in the natural history of SMA Type 1 at a mean duration of follow-up of 10.6 months, including patients with a more severe phenotype compared to previous studies
  • Two-thirds of patients (66.7%) were free of feeding support, an important indicator of stabilization/halting of disease progression
  • New interim Phase 3 STR1VE-EU data presented at WMS support the robust clinical evidence that …
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Life Science Companies and the Bill & Melinda Gates Foundation: Joint Communique

COVID-19’s existence anywhere poses a threat to communities everywhere. The health, social, and economic impacts can only be addressed through the collective actions of stakeholders across private, public, and philanthropic sectors in partnership with civil society. As organizations dedicated to improving and protecting global health, with our varied skills, roles, and resources, we remain committed to doing our part in ending this pandemic worldwide. Earlier this year AstraZeneca; Bayer; bioMérieux; Boehringer Ingelheim; Bristol Myers Squibb; Eisai; Eli Lilly; Gilead; GSK; Johnson & …

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Chagas disease: Breaking the silence

Although Chagas disease was discovered more than a century ago, it remains a major public health challenge. Chagas disease affects approximately six million people, mainly in Latin America. Yet, less than 1% of affected individuals receive proper anti-parasitic treatment, and current tools to fight the disease are outdated and inadequate.

Like other neglected tropical diseases, Chagas disease often affects poor and marginalized communities.

The disease can be silent for decades until it may become fatal, leading to severe cardiovascular and gastrointestinal complications in up …

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Speaking Up in CML

Lisa, who lives with her family in Adelaide, Australia, began experiencing bouts of extreme fatigue about 18 years ago, and remembers being nearly asleep at odd hours of the day. Having two young children at the time, she shrugged it off as being a normal part of motherhood.

When Lisa’s fatigue worsened, she visited her doctor’s office. Her general practitioner diagnosed her with chronic fatigue syndrome, but Lisa still felt something was off and decided to get bloodwork done.

After Lisa’s blood tests revealed an abnormally high white blood cell count, she was referred to an …

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