The FDA Grants Rare Pediatric Disease Designation to Odiparcil for the Treatment of MPS VI
5 March 2019
Daix (France), March 05, 2019 – Inventiva (Euronext: IVA), a clinical-stage biopharmaceutical company developing oral small molecule therapies for the treatment of diseases in the areas of fibrosis, lysosomal storage disorders and oncology, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation (RPDD) to odiparcil, the Company’s product candidate in development for the treatment of mucopolysaccharidosis (MPS) VI, a type of rare, progressive genetic disorder characterized by a deficiency in the lysosomal enzymes responsible for the normal degradation of glycosaminoglycans (GAGs). The designation of rare pediatric disease status confirms odiparcil’s eligibility to receive a Priority Review Voucher upon FDA approval of a new drug application (NDA) to be filed for odiparcil for the treatment of MPS VI.
Pierre Broqua, Ph.D., Chief Scientific Officer and cofounder of Inventiva, declared: “This important designation from the FDA illustrates the severity of MPS VI in the pediatric population. We look forward to continued collaboration with the FDA in advancing odiparcil as a therapy in such a rare and life-threatening disease for which there is still a significant unmet medical need .”
The FDA defines “rare pediatric diseases” as diseases with serious or life-threatening manifestations that primarily affect people who are less than 18 years old and that affect fewer than 200,000 people in the United States. Under the FDA’s RPDD program, a sponsor who receives approval of an NDA or a biologics license application (BLA) for a product for the prevention or treatment of a designated rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain priority review for a future submission of an NDA or BLA. Redeemed vouchers can reduce FDA review time from twelve to six months. Priority Review Vouchers may be used by the sponsor, or sold or transferred to a third party, with prices ranging from $67.5 million to $350 million since the first one was issued in 2009. The last voucher was sold in October 2018 for $80 million.
Odiparcil is an orally-available small molecule that acts on the underlying cause of the symptoms of MPS. MPS is characterized by the accumulation of GAGs, which are important for the modulation of cell‑to‑cell signaling 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, thus 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 and dermatan sulfate, either or both of which accumulate in patients with MPS subtypes I, II, IVa, VI and VII.
Inventiva is currently evaluating odiparcil in a Phase IIa clinical study for the treatment of adult patients with MPS VI.
Odiparcil has been granted orphan drug status for the treatment of MPS VI by the FDA and the European Medicines Agency.
About the Phase IIa iMProveS clinical trial
The iMProveS (Improve MPS treatment) clinical trial is a 26-week Phase IIa clinical trial evaluating odiparcil for the treatment of adult patients with MPS VI. The primary endpoint of the trial is safety, as assessed by clinical and biological standard tests. Secondary endpoints include changes from baseline in leukocyte, skin and urinary GAG content, and improvements of activity and mobility, cardiovascular, lung and respiratory function, and vision and hearing impairments.
Inventiva expects to enrol 18 adult MPS VI patients currently receiving enzyme replacement therapy (ERT) in a double-blind placebo-controlled study and six patients not receiving ERT in an open label cohort at two sites in Europe. Results of the trial are expected in the second half of 2019.
 Source: Biocentury May 4, 2018 “Voucher Equilibrium”; Biocentury November, 1st 2018 “Lilly buys Siga’s priority review voucher gained via smallpox approval”.
Lanifibranor, its lead product, is an anti-fibrotic treatment acting on the three alpha, gamma and delta PPARs (peroxisome proliferator-activated receptors), which play key roles in controlling the fibrotic process. Its anti-fibrotic action targets two initial indications with substantial unmet medical need: NASH, a severe and increasingly prevalent liver disease already affecting over 30 million people in the United States, and systemic sclerosis, a disease with a very high mortality rate and for which there is no approved treatment to date.
Inventiva is also developing a second clinical program with odiparcil (IVA 336) for the treatment of patients with mucopolysaccaridosis type VI (or Maroteaux-Lamy syndrome), a rare and severe gene disease affecting children. Odiparcil has also the potential to address other MPS types, characterized by the accumulation of chondroitin or dermatan sulfate (MPS I or Hurler/Sheie syndrome, MPS II or Hunter syndrome, MPS IVa or Morquio syndrome and MPS VII or Sly syndrome). Inventiva is also developing a portfolio of early research projects in the field of oncology.
Inventiva benefits from partnerships with world-leading research entities such as the Institut Curie in the field of oncology. Two strategic partnerships have also been established with world-class major pharmaceutical companies AbbVie and Boehringer Ingelheim in the fields of autoimmune diseases (specifically in psoriasis) and fibrosis respectively. These partnerships provide milestone payments to Inventiva upon the achievement of pre-clinical, clinical, regulatory and commercial milestones, in addition to royalties on the products resulting from the partnerships.
Inventiva employs over 100 employees and owns R&D facilities near Dijon, acquired from the international pharmaceutical group Abbott. The Company owns, a proprietary chemical library of over 240,000 molecules as well as integrated biology, chemistry, ADME and pharmacology platforms.
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