Onyx Pharmaceuticals will have to delay its planned end of year filing with the FDA for its multiple myeloma drug, carfilzomib. The FDA today asked that the company to show it can match smaller amounts of the drug used in clinical trials with new runs made a greater scale. Perhaps this shows greater attention to scale up by the agency given some issues other companies have had with their manufacturing of biologic drugs this year - Genzyme, Dendreon, etc.
Onyx did encounter some problems with scaled up material that was believed to be related to temperature control problems with manufacturing equipment used in the runs. Onyx will now generate data to show the larger scale process is reproducible. There is no suggestion that the clinical profile of carfilzomib has changed in any way.
“This is a timing issue,” Onyx CEO Tony Coles said today on a conference call with analysts. “We believe the fundamental value proposition for carfilzomib is unchanged and the commercial opportunity is significant.”
The new drug is vitally important to Onyx’s growth prospects, as it seeks to diversify beyond its lone marketed product at the moment, sorafenib (Nexavar) for kidney and liver cancer. If the drug is approved, it would be a new treatment option for a disease that hits about 20,000 people each year in the U.S., and kills about half that many every year. Onyx expects to file with the FDA as early as mid-year 2011 after resolving the delay.
Posted by Bruce Lehr October 8th 2010.