HOUSTON — IPS HEART has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for its investigational therapy, GIVI-MPCs, aimed at treating Becker Muscular Dystrophy (BMD). The designation recognizes GIVI-MPCs’ unique ability to generate new muscle tissue containing 100% full-length dystrophin, a key protein missing in patients with BMD.
The company has demonstrated the regenerative potential of GIVI-MPCs across several preclinical models. These include BMD dystrophic pigs, both young and aged Duchenne Muscular Dystrophy (DMD) mice, and mice with sarcopenia — a condition linked to age-related muscle loss. In all models, GIVI-MPCs successfully formed new human muscle tissue containing full-length dystrophin.
The U.S. Food and Drug Administration (FDA) has already granted Orphan Drug Designation to GIVI-MPCs for both BMD and DMD. This dual recognition underscores the therapy’s broad potential to treat two of the most debilitating muscle disorders.
Currently, FDA-approved treatments for DMD — such as exon skipping drugs and gene therapies — are unable to produce full-length dystrophin or regenerate new muscle. In contrast, IPS HEART’s platform may offer a more comprehensive therapeutic solution.
While many BMD patients have contacted the company seeking information about clinical trials, IPS HEART emphasized that its initial clinical focus is on DMD. The company is preparing to launch a Phase I/II clinical trial for DMD using GIVI-MPCs.
“We are deeply appreciative of the support from the BMD community,” a company spokesperson stated. “Our goal is to expand clinical development into BMD as soon as we secure the right partners.”
IPS HEART confirmed it is currently engaged in confidential discussions with several major pharmaceutical companies. These partnerships could help accelerate clinical trials for BMD. The company also noted that it has successfully scaled up production of GIVI-MPCs using suspension-based bioreactor manufacturing, a method capable of meeting the demands of large patient populations.
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