RJ Health Monthly Specialty FDA Actions Newsletter – July 2019 – Volume 2 – Number 7

In July there were six new drug approvals by the FDA we deemed specific to the specialty market, three of the approvals were for biosimilar products. In addition, there were 2 supplemental drug approvals. The FDA had acted on assignment of eight new product reviews.

Drugs included in this review are generally regarded by the industry as specialty drugs.  Qualifiers may include anticipated administration by a professional, distribution by a specialty pharmacy or submission and reimbursement under the medical claim or Medicare Part B.

View our up-to-date Specialty Drug Pipeline Library

New Specialty Drug Approvals – New FDA drug approvals in the previous month

Drug Name Manufacturer Route of Administration Indication Approval Date
Xembify (immune globulin subcutaneous, human- klhw) Grifols Intravenous Treatment of patients over two years old with primary immunodeficiencies. 07/05/2019
Xpovio (selinexor) Karyopharm Oral Treatment of relapsed or refractory multiple myeloma (RRMM) in adults. 07/03/2019
Zirabev (bevacizumab-bvzr)

Biosimilar to Avastin®

Pfizer Intravenous Treatment of five different types of cancer, including metastatic colorectal cancer and unresectable, locally advanced, recurrent or metastatic non-squamous non-small cell lung cancer (NSCLC). 07/01/2019
HADLIMA (adalimumab-bwwd)

Biosimilar to Humira®

Samsung Bioepis Subcutaneous Treatment of Rheumatoid Arthritis (RA), Juvenile Idiopathic Arthritis (JIA), Psoriatic Arthritis (PsA), Ankylosing Spondylitis (AS), Adult Crohn’s Disease (CD), Ulcerative Colitis (UC), and Plaque Psoriasis (Ps). 07/23/2019
RUXIENCETM (rituximab-pvvr)

Biosimilar to Rituxan®

Pfizer Intravenous Treatment of Non-Hodgkin’s Lymphoma (NHL) and Chronic Lymphocytic Leukemia (CLL), 07/23/2019
NUBEQA (darolutamide) Bayer Oral Treatment of patients with non-metastatic castration resistant prostate cancer (nmCRPC). 07/30/2019

New Specialty Supplemental Indication Updates – Approved updated supplemental indications on currently marketed drugs in the previous month

Drug Name Manufacturer Route of Administration Indication Approval Date
OTEZLA® (apremilast)


Celgene Oral Treatment of adult patients with oral ulcers associated with Behçet’s disease. 07/19/2019
KEYTRUDA® (pembrolizumab)


Merck Intravenous Treatment of patients with recurrent locally advanced or metastatic squamous cell

carcinoma of the esophagus whose tumors express PD-L1 [Combined Positive Score

(CPS) ≥10] as determined by an FDA-approved test, with disease progression after one

or more prior lines of systemic therapy.


Specialty Drug FDA Actions – New and supplemental FDA filings in the previous month

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
Crizanlizumab (SEG101) Novartis BLA Prevention of vaso-occlusive crises (VOCs) in patients with sickle cell disease (SCD) Intravenous Not reported

(Priority Review – 6 months)

DARZALEX (daratumumab) Janssen sBLA Subcutaneous (SC) formulation Subcutaneous Not reported
Isatuximab Sanofi BLA Treatment of relapsed/refractory multiple myeloma (RRMM) patients. Intravenous Not reported
Teprotumumab Horizon Therapeutics BLA Treatment of active thyroid eye disease (TED) Intravenous Not reported
KEYTRUDA (pembrolizumab)


Merck SBLA Six supplemental Biologics License Applications (sBLAs) to update the dosing frequency to include an every-six-weeks (Q6W) dosing schedule option. Intravenous 02/18/2020
NERLYNX (Neratinib) Puma Biotechnology sNDA In combination with capecitabine for the treatment of patients with HER2-positive metastatic breast cancer who have failed two or more prior lines of HER2-directed treatments (third-line disease). Oral Not reported
Tazemetostat Epizyme NDA Treatment of metastatic or locally advanced zpithelioid sarcoma not eligible for curative surgery. Oral 01/23/2020
VX-445 (elexacaftor), tezacaftor and ivacaftor Vertex NDA Triple combination therapy for cystic fibrosis (CF) Oral Not reported

Looking Ahead – Anticipated FDA actions for specialty drugs in 2019 through Q1-2020

Drug Name Manufacturer Indication Route PDUFA Date
pexidartinib hydrochloride


Plexxikon Tenosynovial Giant Cell Tumor Oral 8/3/2019


F. Hoffmann-La Roche Non-Small Cell Lung Cancer Oral 8/18/2019
upadacitinib tartrate AbbVie Rheumatoid Arthritis Oral 8/19/2019
Polivy (polatuzumab vedotin)


Genentech Diffuse Large B-Cell Lymphoma Intravenous 8/19/2019


Sarepta Therapeutics Duchenne Muscular Dystrophy Intravenous 8/19/2019
quizartinib dihydrochloride


Daiichi Sankyo Refractory Acute Myeloid Leukemia Oral 8/25/2019
fedratinib Celgene Post-Essential Thrombocythemia Myelofibrosis Oral 9/3/2019
Emgality (galcanezumab)


Eli Lilly Cluster Headache Syndrome (Cluster Headache) Subcutaneous 9/5/2019
Botox (onabotulinumtoxinA)


Allergan Upper Limb Muscle Spasticity Intramuscular 9/8/2019
AV-001 Avadel Pharmaceuticals Unspecified Parenteral 9/15/2019
Darzalex (daratumumab) Genmab Multiple Myeloma (Kahler Disease) Intravenous 9/26/2019
Scenesse (afamelanotide acetate)


Clinuvel Pharmaceuticals Porphyria (Erythropoietic Protoporphyri) Subcutaneous 10/6/2019
teriparatide Pfenex Inc Osteoporosis Subcutaneous 10/7/2019
brolucizumab Novartis Wet (Neovascular / Exudative) Macular Degeneration Intravitreal 10/15/2019
Ultomiris (ravulizumab) Alexion Atypical Hemolytic Uremic Syndrome Intravenous 10/19/2019
Zejula (niraparib) TESARO Epithelial Ovarian Cancer Oral 10/24/2019
Nubeqa (darolutamide) Bayer Hormone Refractory Prostate Cancer Oral 10/28/2019
Ofev (nintedanib) Boehringer Ingelheim Interstitial Lung Diseases Oral 11/20/2019
luspatercept Celgene Thalassemia Subcutaneous 12/4/2019
RVT-802 Enzyvant Sciences DiGeorge Syndrome Injection 12/5/2019
Rituxan (rituximab)


Genentech Granulomatosis with Polyangiitis (Wegener’s Granulomatosis) Intravenous 12/11/2019
Vumerity (diroximel fumarate) Alkermes Relapsing Remitting Multiple Sclerosis (RRMS) Oral 12/25/2019
crizanlizumab Novartis Vaso-Occlusive Crisis Associated With Sickle Cell Disease Intravenous 1/15/2020
tazemetostat hydrobromide Epizyme Soft Tissue Sarcoma Oral 1/23/2020
Keytruda (pembrolizumab)


Merck Hepatocellular Carcinoma Intravenous 2/18/2020
eptinezumab Alder Biopharmaceuticals Migraine Intramuscular 2/21/2020
ozanimod Celgene Relapsing Multiple Sclerosis (RMS) Oral 3/25/2020

Terms & Abbreviations:

  1. aNDA: Abbreviated New Drug Application
  2. BLA: Biologics License Application
  3. FDA: Food and Drug Administration
  4. NDA: New Drug Application
  5. sBLA: supplemental Biologics License Application
  6. sNDA: supplemental New Drug Application
  7. PDUFA: Prescription Drug User Fee Act: a law passed in 1992 which allowed the Food and Drug Administration (FDA) to collect fees from drug manufacturers to fund the new drug approval process. By collecting the fee, the FDA is required to meet benchmarks related to the speed of the NDA/BLA review process
  8. PDUFA Date: deadlines for the FDA to approve new drugs, typically 10 months from NDA/BLA completions, drugs selected for priority review may be allotted 6 months
  9. sBLA: supplemental Biologics License Application
  10. sNDA: supplemental New Drug Application


U.S. Food and Drug Administration. Center for Drug Evaluation and Research Advancing Health Through Innovation 2017 New Drug Therapy Approvals. Downloaded June 29, 2018. https://www.fda.gov/downloads/…/CDER/ReportsBudgets/UCM591976.pdf

Pharma Intelligence Center Web Site. Accessed August 1, 2019. https://pharma.globaldata.com

U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed August 1, 2019. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm

U.S. Food and Drug Administration. Novel Drug Approvals for 2019. Accessed August 1, 2019. https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm629491.htm


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