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

• In two head-to-head clinical trials, patients on brolucizumab 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 brolucizumab¹ 

• In a key pre-specified secondary endpoint, over half of brolucizumab 6 mg patients (56% in HAWK and 51% in HARRIER) were maintained on three-month dosing intervals immediately after the loading phase through year one¹

• Frequent injections is one common reason patients drop off treatment for wet age-related macular degeneration, a leading cause of blindness in the UK² with approximately 40,000 people developing wet AMD in the UK each year³

London, UK, 13 December, 2019 — Novartis today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for Beovu^® (brolucizumab 6 mg), also known as RTH258, an investigational product for the treatment of wet age-related macular degeneration (AMD). The European Commission will consider the CHMP opinion as it makes its final decision on the marketing authorisation for brolucizumab. The decision is expected within three months.

“Age-related macular degeneration is the most common cause of blindness in the UK. Wet AMD, a chronic, degenerative disease which causes deterioration in central vision can lead to difficulties in everyday activities, such as reading and driving, and subsequently a loss of independence,” said Dr Mark Toms, Medical Director and Chief Scientific Officer, Novartis UK. “Today’s decision marks a significant step towards delivering a new treatment option for people living with wet AMD in the UK. Brolucizumab is the first anti-VEGF to maintain a majority of patients on a 12-weekly injection interval immediately after loading whilst maintaining its effectiveness, which will mean fewer hospital visits and encourage more independence for people living with wet AMD in the UK.”

In wet AMD, uncontrolled vascular endothelial growth factor (VEGF) proteins cause faulty blood vessels to grow and leak fluid and blood in the back of the eye. This fluid and blood accumulates, disrupting the normal retinal architecture and ultimately causing damage to the macula^4,5. The macula is the part of the eye that is responsible for central vision, most of our colour vision and the fine detail of what we see. By inhibiting VEGF, brolucizumab is designed to suppress the growth of abnormal blood vessels and reduce the potential for fluid and blood leaking into the retina¹.

The positive CHMP opinion is based on findings from the Phase III, global, head-to-head HAWK and HARRIER clinical trials, in which brolucizumab demonstrated non-inferiority versus aflibercept in mean change in best-corrected visual acuity from baseline to year one¹. In both trials, approximately 30% of patients treated with brolucizumab gained at least 15 letters at year one¹.*

In pre-specified secondary endpoints, fewer patients treated with brolucizumab 6 mg versus aflibercept had intra-retinal and/or sub-retinal fluid at week 16 (35% fewer patients in both HAWK and HARRIER) and at year one (30% fewer patients in HAWK and 41% fewer patients in HARRIER)¹. Significant reductions in central subfield thickness were also seen with brolucizumab¹.

Additionally, over half (56% in HAWK and 51% in HARRIER) of patients treated with brolucizumab 6mg maintained a three-month dosing interval immediately after the loading phase through year one¹. Brolucizumab patients who started on three-month dosing intervals after the loading phase had an 85% (HAWK) and 82% (HARRIER) probability of remaining on this interval through year one¹.

Brolucizumab exhibited an overall safety profile comparable to aflibercept. The most common adverse events (≥5% of patients) with brolucizumab were vision blurred, cataract, conjunctival hemorrhage, vitreous floaters and eye pain¹.

“Wet AMD can progress rapidly and cause significant visual loss in as little as 3 months. Wet AMD can be managed with effective, consistent treatment and regular monitoring of fluid in the back of the eye,” said Mr Robin Hamilton, Consultant Ophthalmic Surgeon at Moorfields Eye Hospital and UK Chief Investigator for the HARRIER study. “Results from HAWK and HARRIER clinical studies showed that brolucizumab offers greater fluid resolution with long lasting effects and better outcomes. I look forward to offering brolucizumab to my patients to potentially reduce future hospital visits for patients and their families. This should enable them more time to do the things that matter most to them.”

About Beovu (brolucizumab)

Beovu (brolucizumab) is the most clinically advanced humanized single-chain antibody fragment (scFv)^1,6. Single-chain antibody fragments are highly sought after in drug development due to their small size, enhanced tissue penetration, rapid clearance from systemic circulation and drug delivery characteristics^6,7,8.

The proprietary innovative structure results in a small molecule (26 kDa) with potent inhibition of, and high affinity to, all VEGF-A isoforms⁷. Brolucizumab is engineered to deliver the highest concentration of drug, providing more active binding agents than other anti-VEGFs^1,6. In preclinical studies, brolucizumab inhibited activation of VEGF receptors through prevention of the ligand-receptor interaction^7,8,9. Increased signaling through the VEGF pathway is associated with pathologic ocular angiogenesis and retinal edema⁴. Inhibition of the VEGF pathway has been shown to inhibit the growth of neovascular lesions and suppress endothelial cell proliferation and vascular permeability⁴.

In October 2019, Novartis received the first, and so far the only, approval for brolucizumab from the U.S. Food and Drug Administration in the treatment of wet AMD.

About the HAWK and HARRIER studies

With more than 1,800 patients across nearly 400 centers worldwide, HAWK (NCT02307682) and HARRIER (NCT02434328) are the first and only global head-to-head trials in patients with wet AMD that prospectively demonstrated efficacy at week 48 using an innovative q12w/q8w regimen, with a majority of patients on q12w immediately following the loading phase¹. Both studies are 96-week prospective, randomized, double-masked multi-center studies and part of the Phase III clinical development of brolucizumab¹. The studies were designed to compare the efficacy and safety of intravitreal injections of brolucizumab 6 mg (HAWK and HARRIER) and 3 mg (HAWK only) versus aflibercept 2 mg in patients with wet AMD¹.

About wet age-related macular degeneration

Macular disease is the biggest cause of blindness in the UK² and age-related macular degeneration (AMD) is the most common type. There are two types of AMD, known as ‘dry’ and ‘wet’. Approximately 39,800 people develop wet AMD in the UK each year³. Wet AMD occurs when abnormal blood vessels form underneath the macula, the area of the retina responsible for sharp, central vision^4,10. These blood vessels are fragile and leak fluid, disrupting the normal retinal architecture and ultimately causing damage to the macula^4,10.

Early symptoms of wet AMD include distorted vision (or metamorphopsia) and difficulties seeing objects clearly^5,11. Prompt diagnosis and intervention are essential^4,10. As the disease progresses, cell damage increases, further reducing vision quality. This progression can lead to a complete loss of central vision, leaving the patient unable to read, drive or recognize familiar faces and potentially depriving them of their independence^4,12. Without treatment, vision can rapidly deteriorate¹³.

About Novartis in ophthalmology

At Novartis, our mission is to discover new ways to improve and extend people's lives. In ophthalmology, we develop and deliver life-changing medicines and therapies for diseases and conditions from front to back of the eye, enabled by data and transformative technologies. Our ophthalmic solutions reach more than 150M people per year, from premature infants to the elderly.

About Novartis

Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach more than 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of nearly 140 nationalities work at Novartis around the world. Find out more at www.novartis.com.

In the UK, we employ approximately 1,500 people to serve healthcare needs across the whole of the UK, as well as supporting the global operations of Novartis. Since 2014, Novartis has invested over £200 million in R&D and is a leading sponsor of clinical trials, in the UK. For more information, please visit www.novartis.co.uk

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