US-based CytRx has received orphan medicinal product designation from the European Commission (EC) for its lead product candidate aldoxorubicin for treatment of advanced soft tissue sarcomas.
The orphan product status will expedite the development of drugs which may provide significant benefit to patients suffering from rare diseases.
Aldoxorubicin is a combination of chemotherapeutic agent doxorubicin and a new linker-molecule that binds specifically to albumin in the blood to allow for delivery of greater amounts of doxorubicin without several of the major dose-limiting toxicities seen with administration of doxorubicin alone.
The drug has been granted orphan drug designation by the US Food and Drug Administration (FDA’s) Office of Orphan Product Development to treat patients with soft tissue sarcomas and pancreatic cancer.
CytRx president and CEO Steven Kriegsman said there is a significant unmet medical need for drugs to treat advanced soft tissue sarcomas.
“This European designation, together with our US orphan designation, will facilitate our global development of aldoxorubicin to help improve the available treatment options for cancer patients suffering from this aggressive and difficult to treat cancer,” Kriegsman added.
Currently, aldoxorubicin is being evaluated in a major Phase III trial under a special protocol assessment (SPA) as a therapy for patients with soft tissue sarcomas whose tumours have progressed following treatment with chemotherapy.
The company has also recently started a Phase II clinical trial with aldoxorubicin in patients with late-stage glioblastoma (brain cancer), and a Phase II clinical trial in HIV-related Kaposi’s sarcoma.
Soft tissue sarcoma is a cancer which occurs in muscle, fat, blood vessels, tendons, fibrous tissues and connective tissue, and can arise anywhere in the body at any age.
Image: Undifferentiated soft tissue sarcoma in left lung of young child. Photo: courtesy of ThatPeskyCommoner.