Hope for new macular degeneration treatments buoys patients

The number of early-stage cases of age-related macular degeneration (AMD) for those aged 50 years and older is projected to nearly double to 17.8 million in the United States by 2050, CDC says.

The number of early-stage cases of age-related macular degeneration (AMD) for those aged 50 years and older is projected to nearly double to 17.8 million in the United States by 2050, CDC says. Patients with a more severe form of the disease can receive periodic injections of a human antibody treatment called ranibizumab (Lucentis—Genentech), which was approved by FDA in 2006. Joan Miller, MD, chief of the ophthalmology department at Massachusetts Eye and Ear Infirmary in Boston, who helped develop ranibizumab, hopes to see a new generation of treatments emerging in the next 5–10 years. Researchers worldwide are experimenting with several drug candidates, including about 20 in clinical trials. They include repurposed statins as well as gene therapies, stem cell treatments, and medicines customized to the genetic makeup of patients. Sales of current AMD medicines, mostly first-generation treatments including ranibizumab, totaled nearly $5 billion in 2016, and the expected new drugs could expand the market to $11.5 billion by 2026, estimates UK-based GlobalData. The approval of the first-ever gene therapy for any disease in December 2017 was encouraging to eye researchers. The new drug, voretigene neparvovec-rzyl (Luxturna—Spark Therapeutics), treats a rare genetic retinal disease in children. "It opened up the avenue for other gene-based treatments, and some of that might be applicable to AMD," said Miller.