Monday, December 22, 2008

Transmolecular’s melanoma treatment gets FDA orphan drug status

By Marc Songini

Cambridge-based biotech company TransMolecular Inc. has won an orphan drug designation from the U.S. Food and Drug Administration for its 131I-TM601 melanoma treatment.

TransMolecular, which focuses on cancer treatments, announced that the 131I-TM601 drug candidate is currently in a Phase 1/2 clinical trial for recurrent malignant melanoma. This is TransMolecular’s third orphan drug designation. It already has an orphan designation for its 131I-TM601 treatment for malignant glioma, and for the non-radiolabeled version of TM601 malignant glioma.

In a statement, Michael Egan, president and CEO of TransMolecular, said the company had won the three orphan designations through a “robust regulatory and clinical development strategy” for the TM601 tumor-targeting platform.

TM601 is a new synthetic peptide originally derived from scorpion venom.  It can deliver small and large complex molecules to tumors, increasing the tumor uptake of therapeutic agents, while diminishing agent accumulation in healthy tissue. The Phase 1 trial confirmed that the intravenously delivered 131I-TM601 can target and bind to tumor tissue in different solid tumors, as well as cross the blood-brain barrier to target brain tumors.

The FDA grants orphan drug designation to therapies and technologies that address rare diseases (affecting under 200,000 Americans each year). The designation offers financial and regulatory incentives to companies to attempt to treat these diseases. About 60,000 people in the U.S. are diagnosed with melanoma annually, and it’s the deadliest form of skin cancer, explained TransMolecular. By the end of 2008, an estimated 8,000 people will have died from the disease.

Founded in 1997, TransMolecular moved to Cambridge in 2005 from its original home in Birmingham, Ala.