2017 will end soon. According to the data statistical analysis provided on FDA website, the U.S. FDA approved 43 original novel drugs in total in the first three quarters, including 27 new molecular entities (NME) and 16 biologics license applications (BLA) (see the attached table for the details). The total number approved in the first three quarters far exceeded that approved in 2016 (FDA approved 22 original novel drugs in total in 2016, including 15 NMEs and 7 BLAs).

Priority Review

Among the 43 original novel drugs approved, 19 received the priority review, accounting for 44% of the total approved. In the four special approval channels of FDA, the priority review is not only for serious diseases, but also applicable to common diseases. The key for entering priority review lies in whether the pharmaceutical products have the potential of being superior to existing treatment means. Pharmaceutical enterprises shall apply for the priority review themselves, and FDA will reply in 45 days. In addition, the priority review only targets at the review stage instead of accelerating clinical trials, unlike the Fast Track and Accelerated Approval.

Accelerated Approval

Currently, the queriable novel drugs that received accelerated approval in 2017 include: BAVENCIO (EMD SERONO INC) and ALUNBRIG (ARIAD).

Breakthrough Therapy

There have been 14 novel drugs that received the other special approval channel of FDA: the breakthrough therapy, which is a novel drug review method initiated by FDA in July 2012 and formally used from 2013, to accelerate drug approval, as a supplemental plan to promote and accelerate the development and review of products that treat serious diseases and solve unmet medical needs. However, the granting of such channel requires substantial evidence of better clinical improvements, and intensive communication with FDA regarding the non-clinical and clinical data, trial design, and review coordination of the development project during the entire clinical trial process. In general, drugs that receive breakthrough therapy designation can obtain closer guidance from FDA senior officials, etc., to guarantee provision of new treatment options to patients within the shortest time.

Orphan Drug Designation

 "Orphan drugs" are pharmaceutical product for preventing, treating, and diagnosing rare diseases. Orphan drugs have become warming up on the market in recent years with biotechnology development, and under guarantee of laws of the U.S. and EU, etc. on orphan drugs and with support of relevant policies. According to relevant data, FDA approved 9 orphan drugs in 2016, accounting for 41% of all the novel drugs approved. There were 16 orphan drugs approved in the first three quarters of this year.

Introduction of novel drugs that received over two of the above:

BAVENCIO (Avelumab): Approved by FDA on March 23, 2017, it is a PD-LI antibody injection developed by Pfizer and German Merck, and used to treat the rare skin cancer: Merkel cell carcinoma.

PD-1 antibody drug can inhibit the binding of PD-L1 (expressed on cancer cells) and PD-1 (expressed on T cells), thereby activating T cells and acquired immune system, and attacking cancer cells. Bavencio received FDA’s accelerated approval and breakthrough therapy designation, and is designated as orphan drug.

ZEJULA (Niraparib): Approved by FDA on March 27, 2017, it is a capsule preparation, with the approved specification of 100mg; Zejula is a PARP inhibitor developed by TESARO, Inc for the maintenance treatment of ovarian cancer.

Zejula received FDA’s priority review, breakthrough therapy designation, and orphan drug designation, being the third PARP inhibitor marketed, and the first PARP inhibitor used for the maintenance treatment of patients in the phase of response, with no BRCA mutation restriction, applicable to a broader population.

Rydapt (Midostaurin): Rydapt (Midostaurin) was approved by FDA on April 28, 2017 to be used in combination with chemotherapy for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who have a specific genetic mutation called FLT3.

Rydapt is approved to be used for use with a companion diagnostic, the LeukoStratCDx FLT3 Mutation Assay, which is used to detect the FLT3 mutation in patients with AML.

The drug received the priority review, fast track (for the mastocytosis), and breakthrough therapy designation (for the AML) of FDA, and is designated as an orphan drug.

Alunbrig (brigatinib): Alunbrig developed by ARIAD was approved by FDA on April 28, 2017, for patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib.

The drug is a potent inhibitor of ALK, which can inhibit ALK and ALK fusion proteins, thereby inhibiting tumor growth. Brigatinib showed good inhibition effects both in in vitro and in vivo tests.

Alunbrig received FDA’s priority review, breakthrough therapy, accelerated approval, and orphan drug designation.

Attached Table NDAs and BLAs Approved by FDA in the First Three Quarters of 2017

Review priority:

P – priority review

S – standard review

O – orphan drug designation

B – breakthrough therapy

A – accelerated approval

Reference data: FDA website