Genta Incorporated has received notice from the US Food and Drug Administration (FDA) that tesetaxel, the company's oral taxane in clinical development, has been granted designation as an 'Orphan Drug' for treatment of patients with advanced melanoma. Orphan drug status provides for a period of marketing exclusivity, certain tax benefits, and an exemption from certain fees upon submission of a New Drug Application.

"The clinical development plan for tesetaxel includes an early determination of its activity in melanoma," said Dr Loretta M Itri, president, Pharmaceutical Development, and chief medical officer. "Prototype taxanes such as paclitaxel have activity in melanoma and have comprised a standard of care in currently ongoing phase-3 studies. We plan to leverage our extensive knowledge gained from late-stage trials of Genasense in melanoma, as well as our worldwide relationships with clinical experts in this disease, to accelerate the tesetaxel programme. Orphan Drug designation is an important milestone in that clinical development process."

Tesetaxel is a novel, orally absorbed, semi-synthetic taxane that is in the same class of drugs as paclitaxel and docetaxel. However, both prototype agents suffer from serious safety issues, particularly hypersensitivity reactions related to intravenous infusions that are occasionally fatal and that require careful pre-medication and observation. Other prominent side-effects of this drug class include myelosuppression (low blood counts) and peripheral neuropathy (disabling nerve damage).

With administration as an oral capsule, tesetaxel was developed to maintain the high anti-tumour activity of the taxane drug class while eliminating infusion reactions, reducing neuropathy, and increasing patient convenience. The oral route also enables development of novel schedules that may expand dosing options when tesetaxel is used alone or in combination with other anticancer drugs. Preclinically, tesetaxel has demonstrated substantially higher activity against cell lines that were resistant to paclitaxel and docetaxel, since acquired resistance is not mediated by the multidrug-resistant p-glycoprotein.

As a late phase-2 oncology product, tesetaxel has demonstrated anticancer activity in its initial clinical trials, and the drug has not been associated with the severe infusion reactions that are linked with other taxanes. Moreover, unlike other oral taxanes, nerve damage has not been a prominent side effect of tesetaxel. Thus, the drug offers substantial opportunities to improve patient convenience, safety, and anticancer activity. More than 250 patients worldwide have been treated with oral tesetaxel in phase-1 and phase-2 clinical trials.

Genta Incorporated is a biopharmaceutical company with a diversified product portfolio that is focused on delivering innovative products for the treatment of patients with cancer.