Inventiva receives FDA Fast Track designation in MPS VI for its clinical-stage asset odiparci - Seite 2

The Fast Track designation for odiparcil in MPS VI follows the acceptance by the FDA in August 2020 of Inventiva’s Investigational New Drug (IND) application for odiparcil in the same indication and the publication at the end of 2019 of positive results from its iMProveS (improve MPS treatment) Phase IIa clinical trial evaluating odiparcil for the treatment of MPS VI adult patients.  

About Fast Track designation¹

Fast Track is a process designed to facilitate the development and expedite the review and potential approval of drugs candidates to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious and life-threatening conditions.

Determining whether a condition is serious is a matter of judgment, but generally is based on whether the drug will have an impact on factors such as survival, day-to-day functioning, or the likelihood that the condition, if left untreated, will progress from a less severe condition to a more serious one.

Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially better than available therapy. Any drug being developed to treat or prevent a condition with no current therapy is obviously directed at an unmet need. If there are available therapies, a fast track drug must show some advantage over available therapy, which is assessed against a pre-defined set of criteria.

About odiparcil

Odiparcil is an orally-available small molecule that acts on the underlying cause of the symptoms of mucopolysaccharidosis (“MPS”), a group of rare, progressive genetic disorders. MPS is characterized by the accumulation of glycosaminoglycans (“GAGs”), polysaccharides which are important for the modulation of cell to cell signalling and the maintenance of tissue structure and function, in the lysosomes of cells. Due to genetic mutations, lysosomes in patients with MPS contain deficient versions of the enzymes necessary to break down GAGs. As a result, GAGs accumulate within the lysosomes, causing the latter to swell and interfere with the ordinary functioning of cells, leading to the symptoms associated with MPS. MPS is categorized by subtypes, depending on the enzyme that is deficient and the corresponding GAGs that accumulate. By modifying how GAGs are synthesized, odiparcil facilitates the production of soluble GAGs that can be excreted in the urine, rather than accumulating in cells. Specifically, odiparcil acts on chondroitin sulfate (“CS”) and dermatan sulfate (“DS”), either or both of which accumulate in patients with MPS I, II, IVa, VI and VII.