Ignyta's cancer drug entrectinib receives FDA orphan drug status


Biotechnology firm Ignyta has received orphan drug designation from the US Food and Drug Administration (FDA) for its investigational, orally available, CNS-active tyrosine kinase inhibitor, entrectinib, to treat NTRK fusion-positive solid tumours.

NTRK fusions are molecular alterations that occur in a wide variety of adult and pediatric solid tumour types.

Entrectinib is designed to target tumours that harbour NTRK1/2/3, ROS1, or ALK gene fusions.

The company noted that the product candidate is the only TRK inhibitor with clinically demonstrated activity against primary and metastatic CNS disease, and has not shown undesirable off-target activity.

"Entrectinib is designed to target tumours that harbour NTRK1/2/3, ROS1, or ALK gene fusions."

Entrectinib is currently being evaluated in a Phase II clinical trial called STARTRK-2, which is the second of the 'Studies of Tumour Alterations Responsive to Targeting Receptor Kinases'.

The global, multicentre, open-label, potentially registration-enabling Phase II clinical trial of entrectinib uses a basket design with screening of patient tumour samples for the relevant targets.

It is reported that such basket designs take full advantage of entrectinib's demonstrated preliminary clinical activity across a range of different tumour types and molecular targets.

The company is focused on providing cancer patients with potentially life-saving, precisely targeted therapeutics guided by companion diagnostic tests.

The integrated Rx/Dx strategy allows the company to illuminate the molecular drivers of cancer and quickly advancing treatments to address them.