Friedreich’s ataxia (FRDA) is an inherited autosomal recessive genetic condition that progressively damages the nervous system, with a typical age of onset between 10 years and 15 years. The risk factors associated with FRDA are diabetes mellitus and heart diseases, including cardiomyopathy and cardiac arrhythmias. FRDA occurs due to mutation in the frataxin (FXN) gene, which is inherited from parents, leading to the presence of mutations in both copies of FXN gene. Initial symptoms of FRDA include frequent falling, difficulty in walking owing to impairment in the coordination ability voluntary movements (ataxia), and unsteady posture. People with FRDA also develop characteristic foot deformities, unbalanced sideways or lateral curvature of the spine (scoliosis), degeneration in the sensory nerve fibers at dorsal root ganglia, impaired swallowing, and slurred speech (dysarthria).
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FRDA is usually diagnosed through genetic testing to search the levels of FXN in spinal cord and brain. Medications and therapies are available only to treat the symptoms associated with FRDA. For instance, angiotensin-converting enzyme (ACE) inhibitors, beta blockers, and diuretics are used to control cardiac abnormalities that occurs due to FRDA.
According to the research findings, majority of the drug candidates in the pipeline are being developed to be administered by the oral route. It has been observed that the oral route of medication is convenient, improved the patient’s compliance, less risk of systemic infections, and inexpensive in nature. Administration of therapeutics for FRDA through oral route has shown promising results in the clinical studies.
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Companies that are involved in developing therapeutics for FRDA have shown positive clinical results in the various phases of drug development. For instance, in June 2017, Reata Pharmaceuticals Inc. announced the positive data of phase II clinical study of omaveloxolone for the treatment of FRDA. The drug candidate induced nuclear factor erythroid 2–related factor 2 (Nrf2) which is suppressed in FRDA patients; and improved mitochondrial and neurological function.
In November 2017, JOTROL, a unique formulation of trans-resveratrol developed by Jupiter Orphan Therapeutics Inc. to treat patients with FRDA, received Orphan Drug Designation by the U.S. Food and Drug Administration (USFDA). The Orphan Drug Designation may help to facilitate drug development of JOTROL for the treatment of FRDA that accelerates the process of development.
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AbbVie Inc., Reata Pharmaceuticals Inc., Takeda Pharmaceutical Company Limited, and Jupiter Orphan Therapeutics Inc. are some of the key companies involved in the development of FRDA therapeutics.
The report comprises detailed pipeline analysis of therapeutics being developed for the treatment of FRDA. 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 FRDA therapeutics development. Detailed regulatory approval procedures in the U.S., Europe, and Japan are also provided in the 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 FRDA.