Three years after the FDA handed out its first rejection of A.P. Pharma's ($APPA) antinausea therapy APF530, regulators did it again, still unsatisfied by the biotech's marketing application. But this time the company insisted right off the bat that it can regroup, get back to the FDA and launch this therapy after about a 6-month delay in its schedule.
The FDA has a number of problems with the application for the treatment, a long-acting formulation of the antiemetic granisetron. There's the CMC side: "The FDA has requested the refinement of one product quality analytical test method, and that certain deficiencies identified during facility pre-approval inspections be addressed." Regulators are also demanding a "human factors validation study evaluating the usability of the APF530 syringe system [the company had originally proposed a two-syringe approach which was later reduced to one] together with its proposed product labeling and instructions for use be conducted with product assembled using a validated, commercial process." And finally there's a "re-analysis of the existing Phase III clinical data that reclassifies patients into those receiving moderately emetogenic chemotherapy and highly emetogenic chemotherapy according to the recently modified ASCO 2011 Guideline."
A.P. Pharma has been traveling a long, hard road in search of an approval to sell its drug, designed to prevent vomiting in chemotherapy patients. Back in 2010 the biotech was ordered back to the drawing board with instructions to mount new studies on bioavailability and metabolism while refusing a request to waive rules on a QT study. The company has been through several cost-cutting programs as it doggedly concentrated on APF530.
This morning, investors fled the company, driving its battered shares down 37% to a mere 22 cents..
Nevertheless, the company's CEO insists that it can respond to the FDA and be on the market with only a relatively brief interruption of its planned launch schedule. If they are right this time, the drug has the potential to earn hundreds of millions of dollars. Jefferies analysts have estimated peak sales potential at $525 million by 2020.
"We appreciate the FDA's thorough review of the APF530 NDA," said CEO John B. Whelan. "While disappointed in today's notification, we believe that the issues raised in the CRL are addressable, and we remain firmly committed to the successful development of APF530, which we believe will fulfill an important unmet need and improve the lives of patients suffering from CINV. In order to allow us time to carefully address the issues raised in the CRL, we are now projecting product launch for the first half of 2014, versus our prior guidance of the second half of 2013."
- here's the press release