Soligenix announced the Pediatric Committee (PDCO) of the European Medicines Agency (EMA) agreed to the Company's Pediatric Investigation Plan (PIP) for SGX942 (dusquetide). It was also agreed that the Company may defer conducting the PIP until successful completion of its ongoing pivotal Phase 3 clinical study evaluating SGX942 as a treatment for oral mucositis in patients with head and neck cancer (HNC).
As part of the regulatory process for the registration of new medicines in Europe, pharmaceutical companies are required to provide a PIP outlining their strategy for investigation of the new medicinal product in the pediatric population. An approved PIP is a prerequisite for filing a Marketing Authorization Application (MAA) for any new medicinal product in Europe. Soligenix will be able to file the adult indication MAA prior to completion of the PIP because the PDCO permitted the Company to defer conducting the pediatric program until the benefit/risk of treatment has been established in the adult population.
Following a potential approval of the SGX942 MAA for treatment of oral mucositis in adult patients with HNC, Soligenix will work with PDCO and EMA to include appropriate pediatric data in the approved labeling.
"PDCO's agreement that we may defer conducting our PIP, allowing us to move forward with a potential submission and approval of an MAA in adults while initiating the pediatric development program, is an important step forward in our efforts to bring SGX942 to market," said Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "Likewise, acceptance of our PIP is an important regulatory milestone for the product, which also allows us to potentially expand the label to include appropriate pediatric data. Oral mucositis remains an extremely debilitating side effect of cancer treatment and is particularly severe and prevalent in HNC patients. Soligenix looks forward to continuing to work with EMA and PDCO to bring this important therapy to the European market as quickly as possible."
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Soligenix is working with leading oncology centers to advance dusquetide development with the conduct of a Phase 3 clinical trial referred to as the "DOM–INNATE" study (Dusquetide treatment in Oral Mucositis – by modulating INNATE immunity). Patient recruitment is anticipated to be completed in 2019 with top-line results available in the first half of 2020, pending the outcome of the interim analysis in Q3 2019.
Mucositis is the clinical term for damage done to the mucosa by anticancer therapies. It can occur in any mucosal region, but is most commonly associated with the mouth, followed by the small intestine. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of mucositis, that mucositis affects approximately 500,000 people in the US per year and occurs in 40% of patients receiving chemotherapy. Mucositis can be severely debilitating and can lead to infection, sepsis, the need for parenteral nutrition and narcotic analgesia. The gastrointestinal damage causes severe diarrhea. These symptoms can limit the doses and duration of cancer treatment, leading to sub-optimal treatment outcomes.
The mechanisms of mucositis have been extensively studied and have been recently linked to the interaction of chemotherapy and/or radiation therapy with the innate defense system. Bacterial infection of the ulcerative lesions is now regarded as a secondary consequence of dysregulated local inflammation triggered by therapy-induced cell death, rather than as the primary cause of the lesions.
It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of oral mucositis, that oral mucositis in HNC is a subpopulation of approximately 90,000 patients in the US, with a comparable number in Europe. Oral mucositis almost always occurs in patients with HNC treated with CRT and is severe, causing inability to eat and/or drink, in >80% of patients. It is common (40-100% incidence) in patients undergoing high dose chemotherapy and hematopoietic cell transplantation, where the incidence and severity of oral mucositis depends greatly on the nature of the conditioning regimen used for myeloablation.
Oral mucositis in HNC remains an area of unmet medical need where there are currently no approved drug therapies.