Novartis Receives Positive CHMP Opinion for Lucentis as First Pharmacological Treatment for Retinopathy of Prematurity in Preterm Infants
Jul 28, 2019 15:21
CST Updated
15:21
Novartis
Drug Development and Manufacturing
July 28, 2019 /BioValleyBIOON/ -- Swiss pharmaceutical giantNovartisNovartis’ ophthalmic drug Lucentis (ranibizumab) has recently received positive regulatory news in the European Union. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion, recommending the approval of Lucentis (10 mg/mL) for the treatment of retinopathy of prematurity (ROP), a rare eye disease and a leading cause of blindness in children.
The CHMP’s assessment opinion will now be submitted to the European Commission (EC), which will consider the CHMP’s opinion and issue a final review decision within the next 2–3 months. If approved, Lucentis will become the first and only medicine authorized in the European Union for the treatment of retinopathy of prematurity (ROP). The drug (0.2 mg dose) will be indicated for the treatment of Zone I ROP (stage 1+, 2+, 3, or 3+), Zone II ROP (stage 1+, 2+, 3, or 3+), and aggressive posterior ROP (AP-ROP) in premature infants.
Currently, the standard of care for retinopathy of prematurity (ROP) is laser surgery, which works by ablating ocular tissues that drive elevated levels of vascular endothelial growth factor (VEGF), but may lead to serious complications such as myopia and high myopia. Although laser surgery is an effective treatment, there is a clear need for innovative approaches to treat ROP without destroying retinal tissue. Unlike laser surgery, Lucenti directly targets and reduces elevated intraocular VEGF levels, addressing the underlying cause of ROP.
The Marketing Authorization Application (MAA) for the new indication of Lucentis in the treatment of retinopathy of prematurity (ROP) is based on data from the Phase III RAINBOW clinical trial (NCT02375971). This was a randomized, open-label, controlled, multicenter clinical study conducted across 26 countries, enrolling a total of 225 patients with ROP to compare the efficacy and safety of intravitreal injections of Lucentis versus laser photocoagulation. The study compared two different concentrations of Lucentis (0.1 mg and 0.2 mg) against laser surgery, the current standard of care, with efficacy evaluated at 24 weeks after study initiation. The results demonstrated that Lucentis is an effective, safe, and well-tolerated medication for infants with ROP. Although the study slightly missed statistical significance for the primary endpoint (p=0.0254; note: p≤0.025 is considered statistically significant), the differences in treatment success rates between the Lucentis groups (0.2 mg and 0.1 mg) and the laser surgery group (80%, 75%, and 66.2%, respectively) were clinically meaningful.
Professor Andreas Stahl, Director of the Department of Ophthalmology at the University Medicine Greifswald, stated, “Given the vulnerabilities of these patient populations and the limitations of current treatments, randomized controlled Phase III clinical trials are crucial to ensure the safe and effective use of pharmacotherapy in pediatric patients. If approved, Lucentis will represent a valuable alternative to laser therapy.”
NovartisDirk Sauer, Head of Ophthalmology Development, stated: “We recognize the importance of investing in clinical programs to secure marketing authorization, ensuring the safe use of pharmacological treatments in the vulnerable pediatric patient population. We are pleased to take another step forward in bringing this transformative therapy to premature infants with retinopathy of prematurity (ROP) in Europe. The CHMP’s positive opinion reflects our unwavering commitment to innovation, addressing high unmet medical needs, and reimagining eye care for patients of all ages.”
Lucentis is a humanized therapeutic antibody fragment designed to block all biologically active forms of vascular endothelial growth factor A (VEGF-A), whose levels are elevated in wet AMD and various other ophthalmic diseases, such asDiabetes(diabetic macular edema [DME], retinal vein occlusion [RVO]). Lucentis was launched in 2006 and was co-developed by Genentech, a subsidiary of Roche, and Novartis. Roche holds the commercialization rights for Lucentis in the United States, while Novartis holds exclusive rights for the drug in countries and regions outside the United States. In 2018, Lucentis respectively contributed to Roche’s andNovartisGenerated sales of CHF 1.659 billion and USD 2.046 billion.
Lucentis is the first anti-VEGF therapy approved for ophthalmic use, which has revolutionized the treatment of neovascular age-related macular degeneration (nAMD) and helped reduce nAMD-related blindness by 50% worldwide. After more than a decade of innovation, Lucentis has been approved for up to six indications. Currently,NovartisRoche is continuing to explore the potential of Lucentis for treating the youngest and most vulnerable patient populations.
To date, Lucentis has been marketed in more than 110 countries worldwide for the treatment of up to six indications: neovascular age-related macular degeneration (nAMD),DiabetesDiabetic Macular Edema (DME), Branch Retinal Vein Occlusion (BRVO), Central Retinal Vein Occlusion (CRVO), Myopic Choroidal Neovascularization (mCNV), and CNV associated with other etiologies. (Bioon.com)