Retrotope is a clinical-stage company, within the area of degenerative disease. Their first first lead candidate, RT001, today became an orphan designated product for PLA2G6 associated neurodegeneration (PLAN). After asking to expand their clinical trials into Infantile Neuroaxonal Dystrophy (INAD) and severe childhood PLA2G6-induced neurodegeneration, already in trial, the FDA granted a much larger scope for the company to target.
But this isn't just good news for INAD patients, many patients suffering from PLAN conditions have no treatments. PLA2G6 can also be associated with young onset Parkinson's. By seeing the link between all of these conditions it could be that this drug could go on to be re-purposed or re-designed so it can be used within other neurodegenerative indications. And with a much larger patient population, thanks to the wider indication list, this could also benefit patients as the drug does not need to be as expensive to regain R&D costs. However, the outcome of the clinical trials will determine whether this huge decision from the FDA will come to fruition for the PLAN patients.
Retrotope announced today that the U.S. Food and Drug Administration’s (FDA’s) Office of Orphan Products Development (OOPD) granted orphan drug designation for its chemically-modified polyunsaturated fatty acid drug (RT001) for the treatment of PLA2G6 associated neurodegeneration (PLAN).