New FDA Approval Report
The FDA recently released its report titled “Novel New Drugs 2013 Summary,” in which they discuss approvals that occurred in calendar year 2013. In 2013, the FDA approved 27 novel new medicines, called new molecular entities or NMEs. The number of approvals in 2013 was down from 2012 with 39 approvals and down from 2011 with 31 approvals. However when looking at the number of approvals over time, 2013 was consistent with the average over time. For instance, between the years 2004-2012 the average number of NME approvals was 26 per year.
Other highlights from the report include:
- 9 of the 27 NMEs were considered “first in class” which means that they utilize a novel mechanism of action for treating a disease.
- 9 of the 27 NMEs were approved to treat orphan diseases. Orphan diseases are considered rare diseases that affect 200,000 or fewer Americans.
- 13 of the 27 NMEs were designated in one or more expedited approval categories – Fast Track, Breakthrough, Priority Review, and Accelerated Approval
- Fast Track designation is identified by FDA as drugs with the potential to address unmet medical needs. “Fast Track speeds new drug development and review, for instance, by increasing the level of communication FDA allocates to developers and by enabling developers to use a “rolling review” process such that CDER can review portions of an application ahead of the submission of the full application.” (10 NMEs had this designation)
- Breakthrough designation is identified by FDA as drugs with preliminary clinical evidence that shows the potential of substantial improvement over at least one clinically significant endpoint compared with current therapy. “A breakthrough therapy designation conveys all of the fast track program features as well as more intensive FDA guidance on an efficient drug development program.” (3 NMEs had this designation)
- Priority Review is determined by FDA that the drug has the potential to provide a significant advance on medical care. With priority review, FDA sets a target to review the drug within six months instead of the standard that is 10 months. (10 NMEs had this designation)
- Accelerated Approval allows early approval of a drug for a serious or life-threatening illness that offers benefits over current treatments. “This approval is based on a “surrogate endpoint” (e.g., a laboratory measure) or other clinical measure that FDA considers reasonable likely to predict clinical benefit. After this approval, the drug must undergo additional testing to confirm that benefit; this speeds the availability of the drug.” (2 NMEs had this designation)
- 24 of the 27 were approved on the first cycle, which means that they were approved without the need for additional information that would delay approval.
- 20 of the 27 were approved first in the United States before any other country.
Highlights – Biologics
Gazyva sponsored by Roche is a humanized anti-CD20 monoclonal antibody manufactured in CHO cells to treat chronic lymphocytic leukemia. It had received orphan designation, breakthrough designation, and priority review. It was approved on the first cycle and was first approved in the United States.
Kadcyla sponsored by Roche is a HER-2 targeted antibody (manufactured in CHO cells) and microtubule inhibitor conjugate for treatment of HER-2 positive metastatic breast cancer. Kadcyla is a first in class NME that had received both fast track and priority review designation. It was approved on the first cycle and was first approved in the United States.
Biologics approved by CBER
Three biologics approved in 2013 but not included in the report because they were approved by CBER are Rixubis, NovoEight and Flublok.
Rixubis sponsored by Baxter is recombinant Factor IX manufactured in CHO cells to treat Hemophilia B.
NovoEight sponsored by Novo Nordisk is a recombinant anti-hemophilic Factor VIII manufactured in CHO cells to treat Hemophilia A.
Flublok sponsored by Protein Sciences is an influenza vaccine manufactured in the spodoptera frugiperda insect cell line to prevent influenza. This vaccine is considered first in class.