Stargardt disease is an inherited juvenile macular degeneration that causes vision loss. The signs and symptoms of the disease typically appear in late childhood or early adulthood and worsen with time. The most common cause of the disease is mutations in the ABCA4 gene. People suffering from stargardt disease might notice grey or black color in the middle of eye, or eyes may be more sensitive to bright light. Others may even have symptoms of color blindness. The prevalence for the disease is low, reported at around 1 in 8,000-10,000 people globally, as stated by National Eye Institute in April 2015. Hence, the disease falls into rare disease category. Till date, there is no treatment available, though there are multiple therapies in pipeline.
Robust pipeline for each treatment type that may get regulatory approval over the forecast period is expected to boost the growth of stargardt disease therapeutics market
Treatment options for Stargardt disease include drug, gene therapy and stem cell therapy. There are products in each of these category currently in clinical trials. For instance, Nightstar Therapeutics expanded its pipeline with gene therapy program for Stargardt Disease treatment in November 2017. In drug category, certain companies have received orphan drug designation from U.S. FDA. For instance, in January 2017, Acucela Inc. received orphan drug designation for its leading product emixustat for the treatment of stargardt disease. In October 2017, Lin BioScience also received orphan n drug designation for its leading product LBS-008 for the treatment of Stargardt Disease. Furthermore, companies such as Astellas Pharma Inc. is working on stem cell therapy for Stargardt disease. With the approval of some of the therapy that are currently in pipeline, the global stargardt disease therapeutics market is expected to witness rapid growth over the forecast period.
Stargardt Disease Therapeutics Market Taxonomy:
By Treatment Type:
- Gene Therapy
- Stem Cell Therapy
By Stages of Development:
North America is expected to hold a dominant position in the global stargardt disease therapeutics market
Acucela Inc. and Lin Bioscience have received orphan drug designation for their respective products, and are headquartered in U.S. Other companies such as Astellas Pharma Inc, Copernicus Therapeutics Inc. and Sanofi S.A. are either U.S.-based or have considerable presence in U.S. This positions North America as a major market for stargardt disease. Europe is expected to hold second position in stargardt disease therapeutics market as companies like Nightstar therapeutics has shown interest in developing treatment for stargardt disease. In Asia Pacific region, Japan is home to companies such as Astellas Pharma Inc. Also, major Japan-based pharmaceutical companies are working with small-size Company operating in this area. For instance, Otsuka Pharmaceutical Co., Ltd., a Japan-based company, has continued its collaboration agreement with Acucela Inc., a clinical stage company working on stargardt disease, since 2008.
Key players operating in stargardt disease therapeutics market include Nemus Bioscience Inc., Acucela Inc., Katairo, Ocata Therapeutics Inc., Sanofi, Alkeus Pharmaceuticals Inc., Astellas Pharma Inc., Copernicus Therapeutics Inc., Grupo Ferrer Internacional SA, Iris Pharma, Ophthotech Corp, ProQR Therapeutics NV.