- The designation is intended to accelerate the regulatory review and facilitate the overall development of odiparcil for the treatment of MPS VI patients
Daix (France), October 19, 2020 – Inventiva (Euronext Paris and Nasdaq: IVA), a clinical-stage biopharmaceutical company focused on the development of oral small molecule therapies for the treatment of non-alcoholic steatohepatitis (NASH), mucopolysaccharidoses (MPS) and other diseases with significant unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to odiparcil, the Company’s clinical-stage drug candidate for the treatment of MPS type VI (MPS VI), a rare and progressive genetic disorder.
The Fast Track program of the FDA is designed to facilitate the development and expedite the regulatory review and potential approval of drug candidates. Its overall objective is to improve patient access to therapies aimed at treating serious conditions and filling significant unmet medical needs.
Pierre Broqua, Ph.D., Chief Scientific Officer and cofounder of Inventiva, commented: “We are very pleased by the FDA’s decision to grant Fast Track designation to odiparcil for the treatment of MPS VI. It is a clear recognition of the existing unmet medical need for this rare and progressive genetic disorder. We believe that odiparcil has the potential to become a treatment of choice for MPS VI patients, especially due to its oral formulation and ability to reach organs and tissues which are poorly treated by the current standard of care. This designation represents an important step in the development of odiparcil towards becoming an additional treatment for MPS VI patients, who are looking for more efficacious and convenient therapies.”
Fast Track designation offers the possibility of more frequent communication with the FDA to discuss the drug candidate’s development plan, the design of the proposed clinical trials, the use of biomarkers, and the collection of appropriate data required to support drug approval. It also offers access to accelerated approval and priority review, as well as to rolling review of its Biologic License Application (BLA) or New Drug Application (NDA) on a section by section basis, rather than waiting for the entire BLA or NDA to be completed.
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.
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.
Odiparcil has been evaluated for the treatment of MPS VI adult patients during the iMProveS (improve MPS treatment) Phase IIa clinical trial with positive results published at the end of 2019.
Odiparcil has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and has also obtained Rare Pediatric Disease Designation (RPDD) in the U.S. for the treatment of MPS VI.
Inventiva is a clinical-stage biopharmaceutical company focused on the development of oral small molecule therapies for the treatment of NASH, MPS and other diseases with significant unmet medical need.
Leveraging its expertise and experience in the domain of compounds targeting nuclear receptors, transcription factors and epigenetic modulation, Inventiva is currently advancing two clinical candidates, as well as a deep pipeline of earlier stage programs.
Lanifibranor, its lead product candidate, is being developed for the treatment of patients with NASH, a common and progressive chronic liver disease for which there are currently no approved therapies. Inventiva recently announced positive topline data from its Phase IIb clinical trial evaluating lanifibranor for the treatment of patients with NASH.
Inventiva is also developing odiparcil, a second clinical-stage asset, for the treatment of patients with subtypes of MPS, a group of rare genetic disorders. At the end of 2019, Inventiva published positive results from its Phase IIa clinical trial evaluating odiparcil for the treatment of MPS VI adult patients.
In parallel, Inventiva is in the process of selecting an oncology development candidate for its Hippo signalling pathway program. Furthermore, the Company has established a strategic collaboration with AbbVie in the area of autoimmune diseases. AbbVie has started the clinical development of ABBV‑157, a drug candidate for the treatment of moderate to severe psoriasis resulting from its collaboration with Inventiva. This collaboration enables Inventiva to receive milestone payments upon the achievement of pre-clinical, clinical, regulatory and commercial milestones, in addition to royalties on any approved products resulting from the collaboration.
The Company has a scientific team of approximately 70 people with deep expertise in the fields of biology, medicinal and computational chemistry, pharmacokinetics and pharmacology, as well as in clinical development. It also owns an extensive library of approximately 240,000 pharmacologically relevant molecules, approximately 60% of which are proprietary, as well as a wholly‑owned research and development facility.
Inventiva is a public company listed on compartment C of the regulated market of Euronext Paris (Euronext: IVA – ISIN: FR0013233012) and on the Nasdaq Global Market in the United States (ticker: IVA). www.inventivapharma.com
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