11 September 2019
Sydney, Australia – Clarity Pharmaceuticals, a radiopharmaceutical company focused on the treatment of serious disease, is pleased to announce that it has submitted an Orphan Drug Designation (ODD) request with the U.S. Food and Drug Administration (FDA) for 64Cu-SARTATE™, a diagnostic for the clinical management of neuroendocrine tumours (NETs).
NETs are a group of cancers that originates in the diffuse neuroendocrine system. They most commonly occur in the gastrointestinal tract (48%), lung (25%), and pancreas (9%), but may also occur in other areas including the breast, prostate, thymus and skin. NET patients commonly present at an advanced (metastatic) disease stage.
Due to the rarity of NETs and nonspecific presentation of symptoms, a delay in diagnosis or misdiagnosis is common. Mean patient-reported time from first symptom onset to diagnosis was 52 months; 29% of patients required ≥ 5 years for a NET diagnosis, and 58% of patients had metastases at the time of diagnosis. As a result, the median survival for patients can be as short as 10 months.
FDA grants ODDs to facilitate the development of investigational therapies intended to treat, diagnose or prevent rare diseases affecting fewer than 200,000 people in the United States. An ODD provides a number of benefits to pharmaceutical development companies, including potential tax credits for clinical costs, exemptions from certain administrative FDA fees, eligibility for grants to fund future clinical work and seven years of marketing exclusivity if a marketing application is approved.
Dr Alan Taylor, Clarity’s Executive Chairman, said “At Clarity, we are continuing to progress our products to market and this is an important regulatory step for the development of 64Cu-SARTATE™ in NETs.
“Despite the recent advances in diagnostic methods with gallium-68 (68Ga) labelled somatostatin analogs becoming a preferred diagnostic imaging agent for well-differentiated NETs, most NETs are still diagnosed at an advanced stage and can rarely be cured in these cases.
The issues relating to 68Ga mean that many patients are not getting access to the latest diagnostic tools. 68Ga is normally eluted from expensive and inefficient generators on site which require highly trained personnel to prepare the products and maintain the generators at each treatment centre. The short half life and limited supply of 68Ga also present challenges in scheduling and scanning of patients leading to significant patient backlogs.
“64Cu-SARTATE™ utilises copper-64, which has a half life that allows finished product to be centrally manufactured in large volumes to meet fluctuating end-user demands without the logistical and financial challenges associated with 68Ga. 64Cu-SARTATE™ therefore has the potential to improve early-stage diagnosis to prolong survival. Given the superior affinity of our products to copper, 64Cu-SARTATE™ also has the benefit of measuring later time points compared to 68Ga-based products, which may have the added benefit of better diagnostic and therapeutic outcomes.
“The receipt of the ODD status from the US FDA would enable us to advance the development of this product more quickly and efficiently, getting Clarity closer to our ultimate goal of better treatment of children and adults with cancer.”
Cheung, Vincent T F, and Mohid S Khan. 2015. “A Guide to Midgut Neuroendocrine Tumours (NETs) and Carcinoid Syndrome.” Frontline Gastroenterology 6 (4): 264–69. https://doi.org/10.1136/flgastro-2014-100483.
Hallet, Julie, Calvin How Lim Law, Moises Cukier, Refik Saskin, Ning Liu, and Simron Singh. 2015. “Exploring the Rising Incidence of Neuroendocrine Tumors: A Population-Based Analysis of Epidemiology, Metastatic Presentation, and Outcomes.” Cancer 121 (4): 589–97. https://doi.org/10.1002/cncr.29099.
2018 Raphael, Michael J., David L. Chan, Calvin Law, and Simron Singh. 2017. “Principles of Diagnosis and Management of Neuroendocrine Tumours.” CMAJ : Canadian Medical Association Journal 189 (10): E398–404. https://doi.org/10.1503/cmaj.160771.
Dr Alan Taylor
Ph: +61 (0)413 871 165
Clarity is a personalised medicine company focused on the treatment of serious diseases. The Company is a leader in innovative radiopharmaceuticals, developing targeted therapies for the treatment of cancer and other serious diseases in adults and children.
Read the media release here.