orphan-designation

177Lu-FAP-VG11 is an experimental radioactive medicine from VitsGen Therapeutics that targets FAP, a protein on the support cells around many tumours. It is in an early hospital-led trial in China, and the FDA has given it orphan drug status for soft tissue sarcoma.

BAL0891 is an experimental medicine from SillaJen that blocks two enzymes cancer cells use to divide. It is in an early trial, and the FDA has given it orphan drug status for acute myeloid leukaemia.

Cantrixil is an experimental medicine from Vivesto being developed for blood cancers. It is at the laboratory stage for acute myeloid leukaemia, where the FDA has given it orphan drug status, after an earlier phase 1 study.

CAR001 is an off-the-shelf immune cell therapy from Ever Supreme Bio Technology, made from donor gamma delta T cells. It is in an early trial in solid tumours, and the FDA has given it orphan drug status for malignant glioma.

CPI-008 (cRGD-ZW800-1) is an experimental dye that sticks to tumour tissue so surgeons can see it under near-infrared light. It has been tested in phase 2 trials and has European orphan status for head and neck cancer.

This is indocyanine green, a glowing dye, as developed by Renew Pharmaceuticals for liver cancer. The FDA has given it orphan drug status as a diagnostic for managing hepatocellular carcinoma, the commonest liver cancer.

PACAB-002 is an experimental nanoparticle form of the chemotherapy cabazitaxel from NaDeNo Nanoscience, given into the abdomen after ovarian cancer surgery to clear small deposits left behind. Its first trial in people has been approved, and it has orphan status in the European Union for ovarian cancer.

SKL35501 is an experimental radioactive medicine from SK Life Science that seeks out tumour cells carrying the NTSR1 receptor and irradiates them from close range. It is in a first trial in people, and the FDA has given it orphan drug status for pancreatic cancer.

TE-1182 is an experimental antibody-drug conjugate from T-E Meds that carries two different cell-killing drugs to cancer cells with HER2 on their surface. It has been tested only in the laboratory, and the FDA has given it orphan drug status for gastric cancer.

Ulixertinib is an experimental tablet from Biomed Valley Discoveries that blocks ERK, the last switch in a growth signalling chain that drives some cancers. It is being tested in histiocytosis and in gliomas.

UTRxMYCN M1-14 is an experimental RNA medicine from UTR Therapeutics that makes the message of the cancer gene MYCN break down. It has been tested only in the laboratory, and the FDA has given it orphan drug status for soft tissue sarcoma.