Amira Therapeutics Receives Orphan Drug Designation for AMI463 in Soft Tissue Sarcoma in the United States
- Amira therapeutics has received orphan drug designation (ODD) for its compound AMI463 by the FDA.
- The compound had previously received the designation of rare pediatric disease, also by the FDA, and ODD itself by the EMA.
- Amira is associated with the Fundació Mutua General de Catalunya (MGC) and collaborates closely with the Vall d’Hebron University Hospital Research Institute Foundation (VHIR) in the development of this compound.
AMIRA THERAPEUTICS (Amira), 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. This crucial milestone underlines our commitment to address unmet needs in pediatric oncology.
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.
About Amira Therapeutics
Amira Therapeutics, a preclinical stage biotechnology company developing two lead compounds, AMI463 and AMI605, is at the forefront of the development of therapies for children with cancer. As a spin-off of the Leitat Technological Center, based in Barcelona, Spain, Amira embraces a rapid development model, collaborating with prominent European research institutions, such as VHIR, to accelerate the path from the laboratory to the clinic.
Amira Therapeutics is also deeply committed to collaborative efforts with society in general, as exemplified in our partnership with the Fundació Mutua General de Catalunya (MGC), a non-profit foundation that shares our dedication to combating pediatric cancer.
About Leitat:
Founded in 1906, Leitat is one of the reference entities at national and European level in technological management. It has a team of more than 500 professionals, experts in applied research, technical services and management of technological initiatives and innovation. Leitat provides social, industrial, economic and sustainable value, offering comprehensive solutions in multiple sectors and areas: development of new materials, eco-sustainable production, occupational health prevention systems, revaluation of waste and utilization of natural resources, interconnectivity and digitization of the industry, green energy and maximizing energy efficiency. Leitat is recognized by the Ministry of Economy, Industry and Competitiveness and is one of the main start-up entities in the European Union’s Horizon2020 program.