July Summary of FDA Regulatory Actions

During the first half of 2019 FDA approved thirteen new investigational drugs for market distribution. The FDA took action on fourteen investigational drugs in July. There were three new approvals in line with the approval pace of the first half. Two drugs received new Priority Designations from the FDA and the agency accepted four new NDAs and three new BLAs. On the other hand, there were setbacks for Intra-Cellular Therapies and Nektar.
​
July 2019 Approvals

1. The FDA approved Karyopharm Therapeutics’ selinexor (Xpovio), on 7/3/2019, in combination with dexamethasone for the treatment of adult patients with relapsed/refractory multiple myeloma who have received ≥4 prior therapies and whose disease is refractory to ≥2 proteasome inhibitors, ≥2 immunomodulatory agents, and a CD38-targeted monoclonal antibody. In February 2019, the FDA Oncologic Drugs Advisory Committee recommend a delay in approval of selinexor-dexamethasone until after the results of the Phase III BOSTON trial were available in the first half of 2020. In March 2019, the FDA delayed the PDUFA date for selinexor by three months.
2. The FDA approved Merck’s combination of imipenem, cilastatin, and relebactam (Recarbrio) on 7/17/2019 for the treatment of complicated urinary tract infections and complicated intra-abdominal infections.
3. The FDA approved ferric maltol (Feraccru/Accrufer, Shield Therapeutics) on 7/26/2019 for the treatment of iron deficiency in adults.
4. The FDA approved darolutamide (Nubeqa, Bayer) on 7/31/2019 for the treatment of nonmetastatic castration-resistant prostate cancer. Darolutamide was approved three months before its PDUFA date.

New Priority Designations

1. The FDA assigned a priority review for tazemetostat for the treatment of metastatic or locally advanced epithelioid sarcoma and assigned a PDUFA date of 1/23/2020.
2. The FDA designated La Jolla’s artesunate an orphan drug for the treatment of malaria. 

Accepted NDAs and BLAs

1. The FDA accepted the NDA for dasotraline in July 2019 for the treatment of patients with moderate-to-severe binge eating disorder and set a PDUFA date in May 2020.
2. The FDA accepted the NDA for opicapone on 7/10/2019, setting a PDUFA date of 4/26/2020. Neurocrine Biosciences has licensed opicapone from Bial for development and commercialization in the U.S. and Canada, as adjunct to levodopa therapy in patients with Parkinson's Disease experiencing end-of-dose motor fluctuations.
3. The FDA accepted Allergan’s AGN NDA looking for approval of bimatoprost sustained-release (bimatoprost SR) for treatment of open-angle glaucoma or ocular hypertension. Bimatoprost SR, an investigational implant, has been developed as the first sustained-release drop-free treatment option for lowering intraocular pressure in patients with open-angle glaucoma or ocular hypertension. The FDA is expected to give its decision in the first half of 2020.
4. The FDA accepted the NDA for Janssen and ViiV’s once monthly cabotegravir and rilpivirine injection and set a PDUFA date for the end of 2019.
5. The FDA accepted the NDA for RedHill Biopharma's RHB-105 (Talicia) for the treatment of H. pylori infection and assigned an 11/2/2019 PDUFA date. RHB-105 is a combination of rifabutin, amoxicillin and omeprazole
6. The FDA accepted the BLA for Sanofi’s isatuximab for multiple myeloma and set a PDUFA date for the end of April 2020. The drug has multiple mechanisms of action including direct induction of apoptosis, antibody-dependent cellular cytotoxicity, antibody-dependent cellular phagocytosis, and complement-dependent cytotoxicity.
7. The FDA accepted the BLA for Sanofi’s isatuximab for treatment of multiple myeloma and assigned a PDUFA date of 4/30/2020.
8. The FDA accepted the BLA and granted a priority review for Novartis’ crezanlizumab for the prevention of vaso-occlusive crises in sickle cell disease patients, suggesting a PDUFA date of 1/16/2020.

Set backs

1. The FDA canceled an advisory committee meeting to review Intra-Cellular Therapies’ lumateperone as a treatment for schizophrenia due to new information provided for the NDA. No change has been made for the September 2019 PDUFA date.
2. The FDA delayed a decision on NKTR-181 to allow additional time to review preclinical data from two additional studies. In July 2019, the FDA put review of NKTR-181 on hold and canceled an advisory committee review of the drug. Some analysts expect a 1-year delay in a review.