Out of the box FDA says Perjeta faces shortage challenges

Shortages of life-sustaining cancer drugs have become an industry concern but to essentially announce a shortage even as a potential blockbuster drug is being approved for release takes the industry into new territory.

Yet that is what happened Monday when the FDA gave the nod to Roche's ($RHHBY) pertuzumab, developed by its Genentech division, for certain patients with late-stage breast cancer. The drug will be sold as Perjeta.

"Given the need for additional treatments for metastatic breast cancer, we made the decision to approve this drug today and not to delay its availability to patients pending resolution of the production issues relating to future supply," Dr. Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, said in a release. "Genentech is currently developing a plan to mitigate the effect on patients of any potential shortage of Perjeta."

Genentech says the drug will be available to patients in the U.S. within two weeks. A Genentech spokeswoman tells The Wall Street Journal that the company has enough supply to meet anticipated demand, but acknowledges that there are production problems. Patrick Yang, who heads Genentech's pharma global technical operations, says in a release that the problem relates to a "cell growth tissue." Genentech has agreed to special steps going forward, to assure the FDA that it is getting the problems resolved, including reviews of data from the next several productions of the medicine. "We expect to meet demand for Perjeta following today's FDA approval," Yang says.

The shortages of drugs in general and crucial cancer drugs in particular have set off a rising chorus of complaints. American Society of Clinical Oncology doctors at the ASCO meeting this month suggested that Congress enact fines for drugmakers that don't give the FDA an early heads up if they face anything that might lead to a shortage. Shortages have declined this year as drugmakers have been trying to do just that. Still, a potential shortage from the get-go for a drug that was given the agency's expedited 6-month review to get it to market faster, is a new twist.

Perjeta will be used in tandem with Genentech's Herceptin with each aimed at different regions of the HER2 receptor, giving the cancer a one-two punch. The two drugs would be used along with Sanofi's ($SNY) Taxotere, also available as a generic. The drug has some impressive progression-free survival data and analyst estimates have pegged sales as ranging from $600 million to $1 billion-plus. With a couple of additional indications now under study, Perjeta could climb to peak sales of $8.5 billion, Jeffries analysts have said.

- read FDA release
- see the Genentech release
- here's the Wall Street Journal story

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