Amira Therapeutics Receives Orphan Drug Designation for AMI463 in Soft Tissue Sarcoma in the United States

On April 9, 2024 Amira Therapeutics, an innovative biotechnology company dedicated to improving the lives of pediatric patients with cancer and their families, proudly announces that the Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AMI463 for the treatment of soft tissue sarcoma (Press release, Amira Therapeutics, APR 9, 2024, View Source [SID1234643099]). This crucial milestone underlines our commitment to address unmet needs in pediatric oncology.

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Soft tissue sarcoma (STS), a rare and diverse group of tumors arising from embryologically derived mesenchymal connective tissues, often leads to a poor prognosis, with limited treatment options beyond chemotherapy. Amira is at the forefront of the development of AMI463, particularly for Rhabdomyosarcoma (RMS), the most common STS in children. The RMS, with an annual incidence of about 500 new cases in the United States and about 400 in children under 18 years of age in Europe, is a muscle-related cancer characterized by an activation of the Hedgehog signaling pathway, primarily impacting muscular tissue and hollow organs.

AMI463, a first-in-class inhibitor that blocks the cell adhesion molecule (CAM)-related down-regulated by oncogenes (CDON), is very promising in the treatment of RMS and other sarcomas, as well as various solid tumors. This innovative compound, which targets Hedgehog’s CDON co-receptor, has demonstrated exceptional efficacy in preclinical studies, especially against the most aggressive subtypes of RMS. These studies have been carried out in collaboration with the Vall d’Hebron University Hospital Research Institute Foundation (VHIR), with whom Amira has been collaborating since the beginning of the project. Within the framework of this collaboration, a family of shared ownership patents has been developed that protect the use of the compound for different indications. These patents have recently been granted in Europe, the USA and Japan.

The ODD is granted to promising treatments for rare diseases, offering benefits such as protocol assistance and market exclusivity, further supporting our efforts to bring AMI463 to patients who need it.

In addition to our current designation as an orphan drug from FDA, the compound AMI463 has recently been recognized, also by the FDA, with the Rare Pediatric Disease Designation (RPDD), as well as had previously received the ODD from the European Medicines Agency (EMA), which will mean support for development and protection during marketing also in Europe. These recognitions by the regulatory agencies reinforce our commitment to expand the scope of our innovative treatments and address the global challenge of pediatric cancer.