- Europe's Committee for Medicinal Products for Human Use (CHMP) gave a negative opinion on Friday to Puma Biotechnology Inc.'s neratinib as an extended adjuvant treatment of early-stage HER2-positive breast cancer.
- The rejection is the first negative opinion for a new medicine that the CHMP has issued so far this year. The company revealed in late January that the committee had indicated a negative trend vote for the drug. Neratinib is already approved in the U.S. under the brand name Nerlynx.
- Puma has 15 days to submit a request for appeal of the decision, and the biotech said in a statement that it would submit a request for re-examination.
Despite an approval in the all-important U.S. market, neratinib has had a rather storied clinical journey. The European Medicines Agency, which typically follows the opinions of the CHMP, is expected to reject the drug. The EU regulatory body has indicated on multiple occasions that the tyrosine kinase inhibitor might not have a positive risk/benefit profile.
"The CHMP considered that a greater proportion of women given Nerlynx in the study lived for two years without their disease coming back than women given placebo (around 94% versus 92% respectively). However, it is uncertain that this difference in benefit would be seen in clinical practice," said the refusal statement.
Nerlynx also poses severe safety risks — the drug is known to cause significant bouts of diarrhea that can be particularly harmful to patients.
"Furthermore, Nerlynx causes side effects in the digestive system, particularly diarrhea, which affected most patients and might be difficult to manage. The Committee therefore concluded that the benefits were not enough to outweigh the risk of side effects and recommended that Nerlynx be refused marketing authorization," added the CHMP.
While the Food and Drug Administration and its advisory committee also noted this as a significant drawback, the U.S. regulatory agency ultimately decided it was up to physicians to determine if it was a worthwhile risk for patients.
When the company revealed the negative trend vote in January, its shares slid nearly 30% on the news and multiple analysts lowered their price targets for the stock. The biotech has been considered a takeout target and some analysts feared this would make the company less attractive to potential acquirers.