Orphan Drug designation for Borofalan (10B) (SPM-011) as treatment of Cutaneous Angiosarcoma
Angiosarcoma is a kind of cancer that is derived from vascular endothelial cells and can appear anywhere in the body, but mostly occurs in the skin.
Borofalan (10B) (SPM-011), the boron compound used for BNCT (Boron Neutron Capture Therapy), is designated as orphan drug by the Ministry of Health, Labour and Welfare for treatment of unresectable angiosarcoma in the skin.
This designation offers certain incentives such as priority review but several conditions must be met. For example, the patient number should not exceed 50,000, the indication must be regarded as serious and there is no other comparable drug or treatments. Or else, there should be a high medical need; however, it must be under the condition that the drug must exceed the efficacy and safety of similar treatments and reasonable development has to be expected.
Through the prioritization regarding marketing authorization by the Ministry, subsidies for R&D are also available from the Japanese government.
At the present day, there is no standard treatment yet for angiosarcoma. For this reason, patients undergo all sorts of treatment; such as, surgery, radiation, immunotherapy and chemotherapy.
Having stated that, the efficacy of current therapies is limited and avoiding metastasis is important for improving prognosis; therefore, there is the need of new methods for high local control. With the orphan drug designation being held our company strives to give patients with this disease the chance to get BNCT treatment, earlier than expected.
Phase II clinical trials are currently ongoing in Japan, please refer to the link below for further information:
https://jrct.niph.go.jp/latest-detail/jRCT2031220410
