TransMolecular Receives Orphan Drug Designation for 131I-TM601 for the Treatment of Melanoma
News Dec 29, 2008
TransMolecular, Inc. has announced that the U.S. Food and Drug Administration (FDA) have granted Orphan Drug Designation for its anti-cancer compound 131I-TM601 for the treatment of Stage IIb-IV melanoma. The drug candidate is currently in a Phase 1/2 clinical trial for the treatment of recurrent malignant melanoma.
The company had previously received Orphan designation for 131I-TM601 in malignant glioma, as well as for the non-radiolabeled version of TM601 for the treatment of malignant glioma.
“The receipt of our third Orphan Drug Designation is a result of our robust regulatory and clinical development strategy for the TM601 tumor-targeting platform,” said Michael Egan, President and Chief Executive Officer of TransMolecular. “We are pleased that this designation represents another step toward our goal of delivering a new therapy to patients battling this difficult disease. Further, it demonstrates recognition of the broad applicability and promise of this platform across multiple oncology applications.”
TM601 is capable of delivering small and large complex molecules to tumors, an attribute that can increase tumor uptake of therapeutic agents while reducing accumulation in normal tissues. In a completed Phase 1 trial it was confirmed that intravenously-delivered 131I-TM601 has the ability to target and bind to tumor tissue in multiple solid tumor types, as well as cross the blood-brain barrier to target tumors in the brain.
TransMolecular has obtained an agreement with the FDA through a Special Protocol Assessment (SPA) for conduct of a Phase 3 trial of 131I-TM601 in patients with newly diagnosed Glioblastoma Multiforme (GBM). TransMolecular’s tumor-targeting platform is currently being pursued by the company and is the subject of ongoing discussions with several potential partners.
In addition to its tumor-targeting capability, non-radiolabeled TM601 has demonstrated anti-angiogenic activity at higher doses. The anti-angiogenic method-of-action of TM601 is now being investigated in a Phase 1 clinical trial in patients with recurrent glioma.
The FDA grants Orphan Drug Designation to promising products that address rare diseases affecting fewer than 200,000 Americans annually. If 131I-TM601 receives FDA approval for melanoma, this designation will entitle TransMolecular to exclusive marketing rights for the compound for the treatment of melanoma for seven years following an NDA approval. Orphan Drug Designation provides financial and regulatory incentives for companies pursuing less common diseases.