FDA Calendar Updates: Amgen, Amicus, Oncothyreon and Medarex

by: Mike Havrilla
Below is a summary of updates to the BioMedReports.com FDA Calendar, which includes a database of over 200 entries. I originally created the calendar to track companies with pending new drug, biological agent, or medical device new product decisions at the FDA.
With the launch of BioMedReports.com, the FDA Calendar has expanded to include the following categories: pending new submissions to the FDA (e.g. NDA, BLA, 510k, PMA, sNDA, sBLA filings), pending complete response letter (CRL) re-submissions to the FDA, and pending late-stage pivotal Phase 3 clinical trial results which are designed to support a filing for FDA approval.
Eurand (NASDAQ:EURX): On 6/22/09, EURX announced that the FDA advised the company that it has extended the PDUFA action date for its pending New Drug Application (NDA) for Zenpep (pancrelipase capsules) by three months for a new estimated decision deadline of 9/23/09. The FDA did not request that the company provide any further information, but attributed the reason for the delay to the need for additional time to review the current submission.
Zenpep is a proprietary pancreatic enzyme product developed for the treatment of exocrine pancreatic insufficiency, a condition associated with cystic fibrosis, chronic pancreatitis and other diseases. The product was developed in response to the 2004 FDA initial guidance on pancreatic enzyme products, which outlined the need to reduce the variability in enzyme levels and stability of currently marketed enzyme therapies and regulate them under NDAs. Zenpep is a highly stable formulation of a porcine pancreatic extract that is biologically similar to the endogenous human pancreatic secretions necessary for proper human digestion.
Amgen (NASDAQ:AMGN) has a pending BLA for denosumab with a PDUFA action date of 10/19/09 for a possible FDA decision. On 6/22/09, the FDA announced that Amgen’s experimental osteoporosis drug denosumab (proposed brand name Prolia) will be revied by an Advisory Panel at a meeting on 8/13/09.
The FDA Advisory Panel will discuss the company's proposed uses of
  1. treating and preventing osteoporosis in post-menopausal women and
  2. treating and preventing bone loss in patients undergoing hormone ablation therapy for prostate and breast cancer.
Amicus Therapeutics (NASDAQ:FOLD): On 6/22/09, FOLD announced it has commenced the U.S. registration Phase 3 trial with its investigational drug, Amigal (migalastat hydrochloride) for the treatment of Fabry disease. The company has reached agreement with the FDA on the key protocol design elements of the pivotal trial, including the use of the surrogate primary endpoint of the change in the amount of kidney interstitial capillary GL-3, the substrate that accumulates in the cells of Fabry patients. In addition, the FDA is in agreement that the company is eligible to seek Accelerated Approval for Amigal according to Subpart H regulations. The company has begun submitting the Phase 3 protocol to investigational sites worldwide and expects to begin the dosing of subjects in the second half of 2009 (2H09). The trial will consist of a six-month double-blind, randomized, placebo-controlled treatment stage and will enroll approximately 60 subjects (at about 30 clinical sites worldwide) who are naive to enzyme replacement therapy (ERT) or who have not received ERT for at least six months prior to the start of treatment with Amigal.
Oncothyreon (NASDAQ:ONTY): On 6/22/09, ONTY announced that Merck KGaA (OTCPK:MKGAY) has initiated a global Phase 3 trial of Stimuvax (BLP25 liposome vaccine, L-BLP25) in patients with hormone receptor-positive, locally advanced, recurrent or metastatic breast cancer. Stimuvax is an investigational therapeutic cancer vaccine being developed by Merck KGaA under a license agreement with ONTY. The Phase 3 trial, named STRIDE (STimulating immune Response In aDvanced brEast cancer), is anticipated to enroll more than 900 patients at approximately 180 sites in over 30 countries - including North America, Europe, Asia and Australia. Patients with estrogen receptor-positive and/or progesterone receptor-positive, non-resectable locally advanced, recurrent or metastatic breast cancer receiving hormonal therapy will be randomized to receive either Stimuvax or a placebo in a 2:1 ratio. The primary endpoint of STRIDE is progression-free survival. Overall survival, quality of life, tumor response and safety will also be assessed in this study. Stimuvax is also being evaluated in a 1300-patient Phase 3 START trial in patients with non-small cell lung cancer with data anticipated in 2011.
Medarex (NASDAQ:MEDX) and Bristol-Myers (NYSE:BMY) expect to report top-line Phase 3 clinical trial results for ipilimumab in the treatment of metastatic melanoma during 1Q10 after reaching the targeted number of events during 4Q09, based on guidance provided at a presentation on 6/17/09. On 6/22/09, shares of MEDX gained 12.5% following a Mayo Clinic press release at its Discovery’s Edge online research magazine that reported on a prostate cancer study with ipilimumab, which is a fully human antibody that binds to CTLA-4 (cytotoxic T lymphocyte-associated antigen 4 is a molecule on T-cells that plays a critical role in regulating natural immune responses).
Two Mayo Clinic patients whose prostate cancer had been considered inoperable have no evidence of disease thanks in part to an experimental drug therapy (ipilimumab) that was used in combination with standard hormone treatment (androgen ablation) and radiation therapy. In these two cases, physicians say the approach initiated the death of a majority of cancer cells and caused extensive tumor shrinkage, allowing surgery. Both men experienced consistent drops in their prostate specific antigen (PSA) counts over the following weeks until both were deemed eligible for surgery, which was successful and both men have resumed their normal lives.
Disclosure: No positions.