Awaiting FDA Word on New Iron-Replacement Drug

AMAG Pharma Awaiting FDA Word on New Iron-Replacement Drug
Ryan McBride 2/4/09

AMAG Pharmaceuticals could be months away from a long-awaited green light from the FDA to begin sales of ferumoxytol (Feraheme), an iron-replacement treatment for people with kidney disease, and a potential cash cow for the Cambridge, MA-based biotech firm (NASDAQ:AMAG).

The promise of ferumoxytol has boosted the prospects of AMAG (formerly Advanced Magnetics), transforming the firm from a small R&D shop to a growing company with manufacturing and marketing capabilities. The FDA—which told AMAG in October 2008 it would need to resolve manufacturing issues at its Cambridge plant to garner approval—is expected to tell the firm whether it can begin sales of ferumoxytol sometime this year.

Ferumoxytol is designed to improve the way iron levels are restored in patients with anemia related to chronic kidney disease, AMAG CEO Brian Pereira says. A key feature of the drug is its carbohydrate coating, he says. The coating enables the drug to be given in larger doses than existing iron-replacement therapies and limits toxic buildups of iron, which have been linked to drops in blood pressure and other side effects. Ferumoxytol is also supposed to be more convenient, because it is given in 17-second shots as opposed to the 15 to 30 minutes it takes to give intravenous infusions of existing therapies. That means patients could get the treatment at their doctor’s office rather than an infusion clinic.

Gearing up for commercialization, Pereira says, AMAG has built an 80-member sales force to market ferumoxytol to hematologists (blood disease specialists) and nephrologists (kidney doctors). Last year the company, which makes ferumoxytol at its facility in Cambridge, secured a deal with a contract manufacturer to ratchet up production of the drug after its potential approval.

“We’re ready for commercialization,” Pereira says. “We’re just waiting for approval.”

AMAG believes that the market for intravenous iron-replacement therapies could reach $1 billion by 2010. The company’s initial market includes 393,000 chronic kidney disease patients who are on dialysis. About 87 percent of patients in that market take intravenous iron treatments such as iron sucrose injection (Venofer), made by a U.S. subsidiary of Japanese drug firm Daiichi Sankyo Company, and Corona, CA-based Watson Pharmaceuticals’ sodium ferric gluconate (Ferrlecit) infusions. (The body needs iron to produce hemoglobin, a protein that carries oxygen through the bloodstream.) Kidney patients often suffer from iron deficiency, and sales of such drugs for that group of patients alone were expected to be $567 million last year, according to industry estimates cited by AMAG.

Pereira says that AMAG has taken action on all of the issues the FDA found at the firm’s Cambridge plant last year (he declined to get into specifics about the issues in order to protect trade secrets). But the CEO has no definite date on when the FDA will make a call on AMAG’s application to begin marketing the drug.

Still, analysts were optimistic about a timely FDA ruling on AMAG’s application after a meeting last week with Pereira. Leerkink Swann analysts Joseph Schwartz and Eric Varma issued a note to investors after the meeting to say that they expect the firm’s manufacturing issues with the agency to be resolved by mid-April, after which the company anticipates either an approval or an action date from the FDA to make a decision on whether to clear ferumoxytol for sale.

An approval of ferumoxytol would qualify the drug as a bit of an accidental success. Pereira tells me that the company—which for much of its 28-year history was dedicated to R&D of diagnostic imaging agents—originally intended to develop the drug as a contrast agent used to diagnose cardiovascular disease. The company still has plans to develop ferumoxytol as a contrast agent for use with magnetic resonance imaging (MRI) in patients with peripheral artery disease, in which leg arteries get clogged.

In fact, AMAG has big plans to expand its ferumoxytol business, including use of the drug to treat a larger pool of CKD patients, some 1.3 million people, with less severe forms of kidney disease who are not on dialysis. The firm is also planning to launch two late-stage clinical trials this year to test the drug as a iron treatment for anemia related to abnormal uterine bleeding and anemia caused by chemotherapy in cancer patients.

http://www.xconomy.com/boston/2009/02/04/amag-pharma-awaiting-fda-word-on-new-iron-replacement-drug/