When the FDA told Alexion in a warning letter late last month that its quality system at a plant in Rhode Island was not "robust" enough, the agency suggested that Alexion hire experts to help it get on top of the problems there. That is what Alexion has done.
Late last month, Alexion Pharmaceuticals ($ALXN) assured investors it was on top of the issues with its pricey rare-disease drug Soliris. But the FDA report indicates that Alexion has had trouble figuring out the source of the contamination and so adequately attacking it.
Everyone who's anyone in Big Pharma wants to amp up in orphan drugs. Treatments for rare disorders, many of them deadly, aren't easily copied, and competitors are few, if not completely absent. Plus, payers are willing to pay top dollar for drugs aimed at very small groups of patients. Even if they are costly.
The FDA has issued a warning letter for the plant where Alexion Pharmaceuticals manufactures its pricey rare-disease drug Soliris. The letter has yet to make it to the FDA's website, but the company got ahead of the news by reporting to the SEC that the warning was delivered yesterday.
Analysts at GlobalData took a quarterly snapshot of the R&D expenses for a big batch of mid-cap biotechs and concluded that rising research costs were eroding corporate profitability. And they pinned the blame squarely on the pricey hunt for new cancer drugs.
It's a good time to be a biotech R&D boss. Emerging from the global financial downturn, the biotech industry has roared back with record market values and a steady stream of regulatory approvals. And top R&D executives are seeing that reflected in their paychecks.
The rare-disease therapy fights two life-threatening diseases--and it costs up to $400,000 per year, depending upon the patient. And if the company has its way, the drug may be targeted at another group of patients.
Alexion's ( $ALXN ) Soliris is arguably the most expensive drug in the world. The rare-disease therapy fights two life-threatening diseases--and it costs up to $400,000 per year, depending upon the patient. And if the company has its way, the drug may be targeted at another group of patients.
These days, if pharma executives aren't talking about targeted drugs and their companion diagnostics, they're touting the promise of rare-disease treatments. In what might be Exhibit A for that argument, Forbes profiles Alexion Pharmaceuticals, which made its name--and built its income statement--on Soliris, a drug now approved for a variety of rare-and-frightening disorders.
While R&D failures, patent problems and buyout fights are dominating Big Pharma news in recent weeks, the first half of the year has seen some sales results for some drugs that are nothing short of amazing. It illustrates how high returns can be when drugs make a competitive leap.