Description du projet
Prise en charge nutritionnelle de la dystrophie myotonique de type 1
La dystrophie myotonique de type 1 (DM1) est une maladie rare neuromusculaire multisystémique incurable pour laquelle il n’existe aucun traitement. Une approche de prise en charge diététique pourrait offrir une option unique aux patients atteints de maladies rares par le biais d’aliments destinés à des fins médicales spéciales (FSMP). MYODM est une formulation unique de caféine et de théobromine spécialement développée pour la prise en charge nutritionnelle de la DM1. Cependant, en tant que complément alimentaire, MYODM est confronté à un problème: la communauté DM1 exige des données humaines pour justifier l’utilisation du produit alimentaire. Par le biais d’un essai clinique interventionnel pilote, le projet MYODM-FSMP, financé par l’UE, rassemblera des preuves humaines que MYODM est une approche unique pour améliorer la qualité de vie des patients atteints de DM1.
Objectif
Myotonic Dystrophy type 1 (DM1) is the most common and multisystemic neuromuscular rare disease affecting 1 million people worldwide, 100,000 EU citizens, who still have no cure or treatment available.
In the light of the ´Leave No-one Behind´ commitment behind Sustainable Development Goals and particularly, the dietary management approach may provide a unique option for rare disease patients through Foods for special medical purposes (FSMP), foods (not drugs) intended for the dietary management of individuals who suffer from certain diseases. The main goal of the present project is to compile in human evidences that MYODM’s food for special medical purposes is a unique approach to improve DM1 patients’ Quality of Life through a pilot clinical trial for foods and supplements sponsored by Myogem and leaded by Dr. Adolfo López de Munain, a worldwide neurologist specialized in DM1.
MYOGEM is the current licensee of the patents WO2016075288 A1 and WO2016075285 A1, which disclosed the effects of some methylxanthines on DM1. Owning the Intellectual Property Rights over the present solution is one of the main competitive advantages of MYOGEM. MYOGEM is the first and unique company worldwide who launched a food supplement for a rare disease in May 2017. However, as food supplement MYODM faces the problem that DM1 community, either neurologists and patient associations, claim for in human data in order to broadly prescribe and consume the product. To overcome this barrier, and gain market confidence, MYOGEM aims to perform a Feasibility pilot clinical trial to demonstrate MYODM’s effect on a small cohort of 10-15 DM1 patients in order to commercially upgrade our current product “food supplement” to “food for special medical purpose”, from now on referred as MYODM-FSMP, that will reach the market in less than one year after the present project ends.
By launching the new MYODM-FSMP product, a 5.5 M€ SOM is expected in 5 years after the project. Two SoE have been awarded.
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SME-1 - SME instrument phase 1Coordinateur
08100 Mollet Del Valles
Espagne
L’entreprise s’est définie comme une PME (petite et moyenne entreprise) au moment de la signature de la convention de subvention.