Amgen Inc, the leading biotechnology company, announced submission of a Biologics License Application (BLA) with the US Food and Drug Administration (FDA) for palifermin, a first-in-class investigational compound in development by Amgen for oral mucositis (mouth sores).

The BLA was submitted under the FDA's Fast Track designation programme, which is designed to expedite FDA review of an investigational therapy for an unmet medical need. If approved, palifermin will be the first therapy indicated to reduce the incidence, duration and severity of oral mucositis in bone marrow transplant patients, the company said in the release.

"Palifermin is a first-in-class, innovative biologic that protects the epithelium of the mouth and gastrointestinal tract from damage caused by anti-cancer therapy," said Beth Seidenberg, chief medical officer and senior vice president of global development at Amgen. "The palifermin BLA filing is an important milestone for Amgen and our commitment to advance innovative molecules that address unmet need in grievous illnesses," he added.

The BLA filing contains data from the phase 3 pivotal study of palifermin which demonstrated that patients with hematologic malignancies undergoing high-dose chemotherapy, with or without irradiation, and bone marrow transplant support who received palifermin suffered less ulcerative oral mucositis (grades 2-4) compared to those receiving placebo (15.7 days vs. 8.4 days). In addition, palifermin helped protect patients from the most severe form of oral mucositis (grade 4) with 20 per cent of palifermin-treated patients experiencing this painful and debilitating side effect, compared to 62 per cent of placebo-treated patients.

In patients with oral mucositis, the sensitive cells (mucosa) lining the mouth and digestive tract are damaged by the drugs or radiation used in cancer treatment. Severe mucositis is extremely painful (often requiring opioid-like analgesics), interferes with alimentation (process of providing nutrition), increases the chance of serious infection and can force hospitalization. In addition, painful mouth sores can make everyday activities like eating, swallowing, talking and sleeping difficult or impossible.

Palifermin (a recombinant human keratinocyte growth factor) is a first-in-class investigational compound in development by Amgen for mucositis throughout the gastrointestinal tract. The potential therapeutic indication is to reduce the incidence, duration and severity of oral mucositis in patients with hematologic malignancies undergoing high-dose chemotherapy, with or without irradiation, followed by a bone marrow transplant.

Approximately 11,000 Americans with hematologic malignancies, including non-Hodgkins lymphoma, Hodgkin's disease, leukemia and multiple myeloma, undergo bone marrow transplants each year.