- Investors gave Cempra Inc. the cold shoulder Tuesday morning after the company rescinded a European approval filing for its pneumonia medication.
- Questions posed by the European Medicines Agency (EMA) convinced Cempra that more data on solithromycin is needed before the drug can make it to market. Cempra submitted a marketing authorization application (MAA) for solithromycin as a community-acquired bacterial pneumonia (CABP) treatment last June.
- Cempra's decision is the latest setback for its lead candidate in that indication. On Dec. 29, the Food and Drug Administration rejected a New Drug Application (NDA) for solithromycin in CABP. The agency didn't ask for more data, but rather an altogether new study that better illuminated the drug's safety profile — particularly in regard to hepatoxicity.
That conclusion, which came in the form of a complete response letter (CRL), came on the heels of a seven to six vote from the Antimicrobial Drugs Advisory Committee favoring approval of the drug. While FDA generally toes the line when it comes to advisory committee votes, its departure was somewhat less surprising given that agency staff had just days before disclosed concerns about the drug's effects on liver health.
To better meet approval standards, the FDA held that solithromycin's safety database of 920 patients would have to grow about ten-fold to 9,000. The agency also flagged violations at two of the drug's manufacturing facilities.
And should Cempra adequately fix those safety and production concerns, the company would still be required to put in place an "enhanced pharmacovigilance program" that addressed post-marketing issues such as labeling, according to a Dec. 29 statement from Cempra.
"We believe the most efficient path to approval is to withdraw the MAA at this time and to resubmit it with the additional data requested by the FDA," Cempra's acting CEO David Zaccardelli said in a March 28 statement.
Getting hands on that data won't be easy, according to some industry followers. Jefferies analysts, for instance, said the cost of a study big enough to generate the 9,000-person safety database would be out of Cempra's reach. The company reported $231.5 million in cash and equivalents as of Dec. 31.
Cempra's stock has taken a beating with each successive setback. Shares fell about 63% after the FDA staff revealed their qualms and another 56% after the CRL. Shares sunk further, by about 5% to $4.14 per share, in Tuesday morning trading, well off the company's 52-week high of $26.95.