Jul 26, 2019
  • Lucentis (ranibizumab), if approved in the EU for this indication, will be the first and only pharmacological therapy for ROP in these vulnerable infants
  • Positive opinion is based on the landmark Phase III RAINBOW trial, which showed that Lucentis (ranibizumab) is an efficacious, safe and well-tolerated treatment for infants with ROP[1]
  • Standard of care treatment for ROP, laser surgery, destroys diseased retinal tissue responsible for elevated vascular endothelial growth factor (VEGF) whereas injectable Lucentis is a pharmacological therapy that directly targets and reduces VEGF[2]

Basel, July 26, 2019 - Novartis today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Lucentis® (ranibizumab 10 mg/ml) for the treatment of preterm infants with retinopathy of prematurity (ROP). ROP is a rare eye disease but a leading cause of childhood blindness [2],[3].

The European Commission will review the CHMP opinion and is expected to deliver its final decision within three months. Lucentis, if approved for this indication, will be the first and only pharmacological therapy indicated for ROP in this vulnerable patient population. The submission is based on the randomized controlled clinical study, RAINBOW, which demonstrated that Lucentis is efficacious, safe, and well-tolerated for infants with ROP [1].

Laser surgery, the current standard of care, works by destroying the tissue in the eye that is responsible for the elevation of VEGF and can be associated with significant complications such as myopia and high myopia. While it is an effective treatment, there is a clear unmet need for innovative ways to treat ROP without destroying retinal tissue. Unlike laser surgery, Lucentis pharmacologically targets and reduces the elevated intraocular level of VEGF, the underlying cause of ROP [4],[5],[6].

If approved in the EU, Lucentis (0.2 mg dose) will be indicated for the treatment of preterm infants with zone I (stage 1+, 2+, 3 or 3+), zone II (stage 3+) or AP-ROP (aggressive posterior ROP) disease.

"Given this vulnerable patient population, and the limitations of current treatments, randomized controlled clinical studies are important to ensure safe and effective use of pharmacological therapies in pediatric patient populations. If approved, ranibizumab will be a valuable alternative treatment option to laser therapy," commented Professor Andreas Stahl, Director of the Department of Ophthalmology, University Medical Center Greifswald.

"We recognize the importance of investing in clinical programs to secure marketing authorization ensuring safe use of pharmacological therapies in vulnerable pediatric patient populations," commented Dirk Sauer, Development Unit Head, Novartis Ophthalmology. "We are very excited to be one step closer to bringing this transformative treatment to preterm ROP patients across Europe. The CHMP positive opinion is a testament to our relentless dedication to innovation, addressing unmet needs and reimagining eye care across all age groups."

About Lucentis
Lucentis (ranibizumab), the first anti-vascular endothelial cell growth factor (anti-VEGF) therapy licensed for ophthalmic use, revolutionized the treatment of nAMD and has helped reduce blindness due to nAMD by 50% in several parts of the world. More than a decade of innovation and six indications (nAMD, DME, BRVO, CRVO, mCNV and other CNV) later, Lucentis continues to preserve and resolve vision for patients. We continue to investigate the possibility of Lucentis to transform the treatment of even the youngest, most vulnerable patients.

Lucentis is available in more than 110 countries and is supported by a portfolio of 251 sponsored clinical studies and extensive real-world experience. The Lucentis clinical development program has enrolled more than 130,000 patients globally across indications with 5.5 million patient-treatment years of exposure since the therapy's launch in the United States in 2006. Lucentis was developed by Genentech and Novartis. Genentech has the rights to Lucentis in the United States. Novartis has rights in the rest of the world. Lucentis is a registered trademark of Genentech Inc.

About ROP
Retinopathy of prematurity affects premature infants in both developed and developing countries. Caused by the abnormal development of retinal blood vessels in premature infants, disease progression is due to high levels of a growth factor called VEGF [6]. After premature birth, high VEGF levels can cause an infant's blood vessels in the retina to develop abnormally, which may lead to structural abnormalities such as retinal detachment, resulting in vision loss or blindness [4],[6]. The retina is the inner layer of the eye that receives light and turns it into visual messages that are sent to the brain [7]. The retina develops late in the womb and very premature babies may have incomplete development of blood vessels needed to provide oxygen [4],[6]. VEGF is an important regulator of the development of new blood vessels (known as angiogenesis) and plays a key role in the progression of ROP. The blood vessels that can develop abnormally during ROP can exert traction on the retina and lead to macular dragging, retinal detachment or other structural abnormalities resulting in vision loss or potentially blindness [6].

The RAINBOW study, which was conducted in 26 countries, is a randomized, open-label, controlled, multicenter study designed to compare the efficacy and safety of intravitreal Lucentis (ranibizumab 10 mg/ml) with laser surgery in 225 patients with ROP. The trial compared two different concentrations of Lucentis, 0.1 mg and 0.2 mg, to the current standard of care, laser surgery. The outcomes of the study were measured at 24 weeks after starting the trial. A long-term extension trial of 5 years is currently underway and expected to conclude by Q4 of 2022 [1].

About Novartis in ophthalmology
Novartis is reimagining the treatment and prevention of visual impairment and blindness. By working to push the boundaries of medicine and technology, we aim to develop life-changing gene therapies, next-generation pharmaceuticals, and transformative technologies for diseases and conditions spanning every area of eye disease, including front and back of the eye.

This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political and economic conditions; safety, quality or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

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 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 108,000 people of more than 140 nationalities work at Novartis around the world. Find out more at www.novartis.com.

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[1] Clinicaltrials.gov. (2018). RAINBOW Study: Ranibizumab Compared With Laser Therapy for the Treatment of INfants BOrn Prematurely With Retinopathy of Prematurity - Study Results - ClinicalTrials.gov. [online] Available at: https://clinicaltrials.gov/ct2/show/results/NCT02375971 [last accessed 31/05/19]
[2] Mintz-Hittner, H., Kennedy, K. and Chuang, A. (2011). Efficacy of Intravitreal Bevacizumab for Stage 3+ Retinopathy of Prematurity. New England Journal of Medicine, 364(7), pp.603-615.
[3] Blencowe, H., Lawn, J., Vazquez, T., Fielder, A. and Gilbert, C. (2013). Preterm-associated visual impairment and estimates of retinopathy of prematurity at regional and global levels for 2010. Nature, 74(s1), pp.35-49.
[4] Nei.nih.gov. (2018). Facts About Retinopathy of Prematurity (ROP) | National Eye Institute. [online] Available at: https://nei.nih.gov/health/rop/rop [last accessed 31/05/19]
[5] Medicines.org.uk. (2018). Lucentis 10 mg/ml solution for injection - Summary of Product Characteristics (SmPC) - (eMC). [online] Available at: https://www.medicines.org.uk/emc/product/307/smpc [last accessed 31/05/19]
[6] Keshet, E. (2003). Preventing pathological regression of blood vessels. Journal of Clinical Investigation, 112(1), pp.27-29.
[7] Moorfields.nhs.uk. (2018). Anatomy of the eye - Moorfields Eye Hospital. [online] Available at: https://www.moorfields.nhs.uk/content/anatomy-eye [last accessed 31/05/19]

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