This article is more than 5 years old. Information may no longer be current.
FDA grants orphan drug, rare pediatric disease status to entrectinib for neuroblastoma
The FDA has granted both orphan drug designation and rare pediatric disease designation to entrectinib for the treatment of neuroblastoma, according to the drug’s manufacturer.
Entrectinib (Ignyta Inc.), a selective tyrosine kinase inhibitor, is a targeted therapeutic candidate intended for patients with cancers that contain activating alterations to TrkA, TrkB, TrkC, ROS1 or ALK. Based on prior mouse xenograft models, entrectinib has demonstrated in vivo antitumor activity against multiple TrkA, ROS1 or ALK-driven cancers.
“We are pleased that the FDA has provided us these designations, which highlight the potential for entrectinib to address unmet needs of patients with rare cancers,” Jonathan Lim, MD, chairman and CEO of Ignyta, said in a press release. “Although Ignyta is intrinsically motivated to continue to aggressively pursue our clinical development program for entrectinib in solid tumors for the benefit of adult and pediatric cancer patients everywhere, we are pleased that the incentives provided by these designations – including the potential for seven years of marketing exclusivity and the potential to obtain a valuable Pediatric Disease Priority Review Voucher from the FDA – can potentially provide additional avenues for creating value for our stockholders.”
The FDA used interim efficacy and safety results from ongoing phase 1/2 clinical trials of entrectinib – the STARTRK-1 and the ALKA-372-001 trials – to base its designation.
According to study results, entrectinib demonstrated a complete response in a patient with ROS1-positive non-small cell lung cancer (NSCLC), as well as five partial responses in patients with three different cancer histologies (colorectal cancer, NSCLC and neuroblastoma) and in patients with each of TrkA, ROS1 and ALK alterations. The interim results were presented at the European Society for Medical Oncology Annual Congress in Madrid.
Ignyta has also filed orphan drug designation applications for entrectinib for multiple adult indications other than neuroblastoma.
The FDA Office of Orphan Products grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.