NPS still expects to submit its biologics license application (BLA) by the end of the year but this is reliant on the CMO (contract manufacturing organisation) overcoming the production problem.
“It’s remarkably bad timing. It sounds like they’ve had a lot of consistency at the scale for some time,” Alan Carr, senior analyst at Needham & Company, told Outsourcing-Pharma.com. Susan Mesco, senior director of investor relations at NPS, confirmed the issue is unrelated to scale-up.
Mesco told this publication the CMO ran commercial-scale validation batches but is now unable to manufacture products that meet specifications. Early batches gave NPS CMC (chemistry, manufacturing and controls) data but it wants to replicate this output before submitting its BLA.
NPS told investors about the problem during its second quarter conference call. On the call Luke Beshar, chief financial officer at NPS, isolated the problem: “It’s a final fill/finish manufacturing process where the product is lyophilised and then put into a carpule with a rubber stopper and water.”
Nycomed has the same CMO problem. The Swiss pharma sells Natpara (recombinant human parathyroid hormone) in Europe where it is branded Preotact. On the call NPS said Nycomed is facing the same CMO problems and shortages are expected in some countries from this month.
Back on track
Both companies are hoping for a speedy resolution. NPS had targeted a BLA submission in the second half of 2012 but it has now refined this goal to filing by the end of the year. Executives on the call said they expect to meet this target but continued CMO problems could delay the filing.
“We expect to resolve this issue to support an end of 2012 BLA submission. However, if it does not resolve in a timely manner, the timeline may be delayed,” Francois Nader, CEO of NPS, told investors.
If NPS can meet its BLA filing goal Carr and Jim Molloy, senior research analyst at ThinkEquity, expect a US Food and Drug Administration (FDA) decision in the second half of 2013. Before then NPS expects a FDA decision on its short bowel syndrome drug Gattex (teduglutide).
NPS outsources the manufacture of Gattex too but it is free from the production problems faced by Natpara. “We use a different fill-finish for Gattex,” Mesco said. FDA pre-approval inspections of the Gattex CMOs went well, NPS said, and raised no issues likely to affect timelines.