November 2011 | Volume 10 | Issue 11 | Feature | 1340 | Copyright © 2011
Pipeline Previews brings to you information on the newest drugs and medical products as they become available to the dermatologic community. This department may include additional information from the manufacturers, plus reports from physicians who wish to share their clinical experience with these new products. In addition, we will inform our readers about the latest drugs receiving Food and Drug Administration (FDA) approval.
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Genentech Submits New Drug Application to FDA
Genentech has announced that it has submitted a New Drug Application for vismodegib to the FDA for the treatment of people with advanced basal cell carcinoma (BCC) when surgery is no longer an option. Vismodegib is taken orally and designed to selectively inhibit signaling in the Hedgehog pathway, which is implicated in more than 90 percent of BCC cases. BCC is the most common type of skin cancer, and is generally considered curable by surgery. However, advanced BCC can cause disfiguring and debilitating effects and can ultimately be lifethreatening.
This submission to the FDA is based on results from the ERIVANCE BCC study that evaluated vismodegib in people with advanced BCC. ERIVANCE BCC is an international, singlearm, multicenter, two-cohort, open-label Phase 2 study that enrolled 104 patients with advanced BCC, including locally advanced BCC (laBCC) (71) and metastatic BCC (mBCC) (33). laBCC patients had lesions that were inappropriate for surgery (inoperable or for whom surgery would result in substantial deformity) and for which radiotherapy was unsuccessful or contraindicated. mBCC was defined as BCC that had spread to other parts of the body, including the lymph nodes, lungs, bones and/or internal organs. Study participants received 150 mg vismodegib orally, once daily until disease progression or intolerable toxicity.
The trial showed vismodegib substantially shrank tumors or healed visible lesions in 43 percent of patients with laBCC and 30 percent of patients with mBCC, as assessed by independent review, the primary endpoint of the study. The ORR as assessed by study investigators, a secondary endpoint, was 60 percent for laBCC and 46 percent for mBCC. The median duration of progression-free survival (PFS) by independent review for both metastatic and locally advanced BCC patients was 9.5 months
The most common drug-related adverse events were muscle spasms, hair loss, altered taste sensation, weight loss, fatigue, nausea, decreased appetite and diarrhea. Serious adverse events were observed in 25 percent of the study patients, but only four percent of patients had serious adverse events that were considered by the researchers to be related to vismodegib, including one case each of blocked bile flow from the liver (cholestasis), dehydration with loss of consciousness (syncope), pneumonia accompanied by an inability of the heart to pump enough blood (cardiac failure), and a sudden arterial blockage in the lung (pulmonary embolism).
FDA Gives Provisional Approval to MelaFind Device
Mela Sciences has announced that the FDA has deemed its noninvasive melanoma detection device, MelaFind, approvable. MelaFind is a multispectral computer vision system with a handheld imager that captures the image of a lesion; software uses algorithms to analyze the image, indicating within two minutes whether a biopsy should be done. Mela Sciences´ application relied on a trial of MelaFind that included 1,383 patients and showed a sensitivity of 98 percent. According to Mela Sciences, the device´s sensitivity rating for malignant melanoma was better than that of dermatologists, who showed a wide range of variability about which lesions would have been recommended for biopsy and which relegated to observation.
The FDA´s provisional approval follows a May 2011 Citizen Petition filed by Mela Sciences with the FDA seeking action on its application for approval. An FDA advisory panel, in November 2010, had voted eight to seven with one abstention on Mela Sciences´ initial approval.
Members of the FDA advisory panel had expressed concern that the MelaFind device might be used by non-dermatologists or that it would be used in place of clinical evaluation. As a result, Mela Sciences will have to work with the FDA on physician and patient labeling, a package insert and user´s guide, a training program for dermatologists, and the protocol for a post-marketing study.
The indications for use state that the MelaFind "is intended for use on clinically atypical cutaneous pigmented lesions with one or more clinical or historical characteristics of melanoma, excluding those with a clinical diagnosis of melanoma or likely melanoma." In addition, the product labeling will also state that the device is "only for use by physicians trained in the clinical diagnosis and management of skin cancer (i.e., dermatologists) who have also successfully completed a training program in the appropriate use of MelaFind."
Mela Sciences has stated that it expects MelaFind to be commercially available in the spring of 2012 to a select group of dermatologists in Connecticut, New Jersey, and New York as a trial run before making it available to a larger number of practices.