Retinitis pigmentosa (RP) is a group of hereditary disorders that lead to the breakdown and loss of cells in the retina. Mutation in genes encoding the proteins required by retinal cells for their proper functioning, results in occurrence of RP. The disease results in partial vision loss during the earlier stage of the disease, which may lead to complete blindness with the progression of the disease. Some of patient-reported side effects include night visual impairment, loss of the visual field, and trouble in differentiating colors.
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The aim of diagnosing RP is to determine the type of mutation that has occurred in genes and can be detected by performing tests, including electroretinogram (ERG), visual field testing, and genetic testing.
The available treatment options include retinal implant, increased intake of vitamin A palmitate, and acetazolamide medication. Furthermore, Luxturna (Spark Therapeutics Inc.) is the only approved therapy by the U.S. Food and Drug Administration (USFDA) for the treatment of RP.
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According to the research findings, majority of the pipeline drug candidates are being developed for subretinal administration. It has been observed that this route of administration has high bioavailability and ensures direct effect on targeted cells.
Positive Clinical Results are Expected to Drive the RP Therapeutics Pipeline
Companies that are involved in developing therapeutics for RP have shown positive clinical results in various phases of drug development. For instance, in December 2018, Applied Genetic Technologies Corporation announced positive top-line results from the phase I trial of XLRS gene therapy for the treatment of patients with RP. The results of the study exhibited that 2 of 13 adult patients treated at high dose showed some improvements in retinal structure as measured by optical coherence tomography (OCT), an imaging technology that provides a side view of retina’s layers.
Companies are actively seeking designation grants to accelerate the process of development. For instance, HORA – PDE6B of Horama, was granted Orphan Drug Designation by the USFDA for the treatment of RP in July 2017.
Luxturna is the only approved therapy for the treatment of RP. Therefore, with the emergence of late- and mid-stage pipeline products in the market, the overall RP therapeutic market is expected to rise significantly in the upcoming years.
Acucela Inc., HORAMA S.A., GenSight Biologics S.A., Novartis AG, Allergan Inc., Spark Therapeutics Inc., Sanofi-aventis Groupe, Ocugen Inc., Applied Genetic Technologies Corporation, Eyevensys, MeiraGTx Holdings plc, and Biogen Inc. are some of the key players involved in the development of drugs indicated for the treatment of RP.
RP Therapeutics Pipeline Analysis
The report comprises detailed pipeline analysis of therapeutics being developed for the treatment of RP. Comprehensive insights on the pipeline products have been provided, with special focus on strategic developments of key players, information on drug licensing, designations, financing, and grants, technological advancements, patents, and upcoming conferences. In addition, the report highlights the winning strategies of companies involved in the RP therapeutics development. Detailed regulatory approval procedures in the U.S., Europe, and Japan are also provided in this report. Furthermore, the report contains competitive analysis and extensive information on monotherapies, combination therapies, targets and mechanisms of action, and drug origin with respect to RP.
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