The Food and Drug Administration has granted Orphan Drug status to the chemotherapy drug Cantrixil for the treatment of patients with ovarian cancer and to the MEK inhibitor selumetinib for the treatment of patients with uveal melanoma.
Cantrixil, being developed by Novogen and CanTx, Inc., is a cyclodextrin envelope containing the active ingredient of TRXE-002. Preclinical data were presented at the American Association for Cancer Research Annual Meeting in April. A Phase I study is currently enrolling patients, and a Phase IIa study is being planned.
Selumetinib is being studied in an ongoing Phase III study of use in combination with chemotherapy to treat patients with first-line metastatic uveal melanoma. Results are expected to be available later this year, according to a news release from the drug's manufacturer, AstraZeneca.
Selumetinib is also being investigated in Phase III studies in KRAS mutation positive lung cancer and thyroid cancer; and a Phase II study is ongoing evaluating the drug for the treatment of children with neurofibromatosis Type 1.
Initial data from a combination study of selumetinib with other pipeline molecules including AZD9291 (a T790M-directed EGFR inhibitor) and MEDI4736 (an anti-PD-L1 drug) for the treatment of non-small cell lung cancer are scheduled to be presented at the ASCO Annual Meeting, AstraZeneca noted.
The Orphan Drug designation-to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.-grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.