Published On : March 2018
Juvenile macular degeneration disorder (JMD) is the term for several inherited and rare diseases that affect children and young adults. They include Stargardt disease, Best disease, and juvenile retinoschisis. They can cause central vision loss that often starts in childhood or young adulthood.
Stargardt disease is the most common form of juvenile macular degeneration. The changes in vision are usually noticed before the age of 20. This retinal condition is usually seen after the age of 6 and before the age of 20. Although they may have had it for some time, some people may not be aware that they have this disease until they are 30 or 40 years old. Both males and females are affected equally.
There are many ways to help children and young adults live active and productive lives in spite of them being affected with juvenile macular degeneration through various visual aids like large print books, electronic book readers, large button cell phones, special glasses, page magnifiers, and computer programs.
Which type of treatment option will witness most demand in the coming years?
It is seen that Stargardt disease is an inherited disease and as the disease progresses, it requires intensive treatments and drugs such as StarGen (Gene therapy). Over the time, the life expectancy of the patients with juvenile macular degeneration has increased with the medical advancements such as stem cell therapy, but so has the cost of the treatment. A company called Oxford BioMedica is working on a gene therapy treatment that replaces the defective ABCA4 gene that causes Stargardt disease by using their LentiVector technology to deliver a corrected version of the gene into the cells of the retina. Advanced Cell Technology received FDA approval to begin Phase I clinical trials using stem cells for the treatment of Stargardt's Macular Dystrophy.
As juvenile macular degeneration formation represents a hallmark in the history of eye disorders, adequate management of juvenile macular degeneration and its complications would better quality of life and escalate the treatment rate. Patients with mild juvenile macular degeneration are advised to follow a low sodium intake diet to increase mobilization of the fluid retention. As there is not even a single pharmaceutical drug approved by FDA for the management of juvenile macular degeneration. For instance, in March 2014, Santen Pharmaceuticals signed a pact with TRACON pharmaceuticals to develop and commercialize Tracon's anti-VEGF product TRC105 for the treatment of various eye related disorder
However, various complications have been observed with juvenile macular degeneration treatment approaches such as no drug treatment, no preventive measure, gradual loss of sight, difficulties in diagnosis, similar symptoms, post-procedural complexities, and others. Some of these complications are rare and life-threatening. Thus, development of new drugs may emerge as the new revenue pockets for the growth of this market.
Keen Players Bank on Novel Treatment Offering for Growth in Global Juvenile Macular Degeneration (Stargardt Disease) market
A number of players are operating in the global juvenile macular degeneration (Stargardt disease) market. Astellas Pharma is a leading player in the juvenile macular degeneration market. The major strategies adopted are mergers and acquisitions so as to develop and identify new products and technologies, and applications of existing products and therapies for use. For instance, in March 2016, Astellas Pharma acquired Ocata Therapeutics to increase its product portfolio for treatment of retinal disorders including Stargardt disease and age-related macular degeneration. The ophthalmology product portfolio of the company includes pipeline drugs mainly VAP-1 inhibitor ASP8232 (Pipeline) and ASP-7317
Carl Zeiss AG specializes in the fields of optics and optoelectronics with its business units developing, manufacturing, and distributing diagnostic and therapy systems, eyeglass, and lenses. The major strategy adopted by the company in recent years is to present their product in annual meeting and seminars to make people aware of the disease, and its treatment. In August 2017, Carl Zeiss AG has acquired Veracity Innovations, LLC to strengthen the lenses business across the markets in Europe and the Middle East and Africa.
Acucela Inc. develops an innovative portfolio of ophthalmology products. The key strategy of each program is to develop and expand the product pipeline. The collaboration strategy with other firms to sustain their position in the market. For instance, in January 2017, Acucela receives orphan drug designation for emixustat from the FDA for the treatment of Stargardt disease.
There are some pipeline drugs available for the management of juvenile macular degeneration such as emixustat by Acucela Inc. LBS-008 developed by Lin BioScience and others.
Emerging Government Policies Stoking Growth of the Juvenile Macular Degeneration (Stargardt Disease) market
According to the data suggested by the Centres for Disease Control and Prevention, juvenile macular degeneration is a chronic, genetic disease that is currently affecting the lives of around 70,000 people all across the world. The estimated prevalence in the U.S. is approximately 32,000 to 41,000 patients.
The rising burden of juvenile macular degeneration and related complications at an alarming rate are triggering various government or private associations to amend and improve their policies. For instance, the Foundation Fighting Blindness had conducted Stargardt disease Workshop to discuss the progression, pathology and therapeutic opportunities of Stargardt disease. The workshop was held in the Tinkham Veale University Center on February 2017. The Macular Degeneration Research (MDR) program by the BrightFocus Foundation has awarded more than $24 million to support research into the causes and potential preventions and treatments of macular degeneration disease. MDR is currently supporting 44 biomedical research projects after awarding 17 new grants. The Macular Disease Foundation Australia Research Grants Program has approximately $3.6 million to support world-leading Australian researchers across 18 research projects
There are very few treatment options available on the market thus, there are a number of opportunities for the manufacturers to develop new therapeutic devices and drugs for Juvenile Macular Degeneration management.