RJ Health Monthly Specialty FDA Actions Newsletter – December 2018 – Volume 1 – Number 12

In December there were a total of four new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were six supplemental drug approvals. The FDA had assigned eight new products for review.

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.


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

Drug Name Manufacturer Route of Administration Indication Approval Date
Herzuma (trastuzumab-pkrb) – Biosimilar CELLTRION Intravenous Treatment of HER2-overexpressing breast cancer 12/14/2018
ASPARLAS™ (calaspargase pegol – mknl) Servier Intravenous

An asparagine specific enzyme indicated as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia in pediatric and young adult patients age 1 month to 21 years.

ULTOMIRIS™ (ravulizumab-cwvz) Alexion Intravenous Treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) 12/21/2018
ELZONRIS (tagraxofusp-erzs) STEMLINE THERAPEEUTICS Intravenous A CD123-directed cytotoxin for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients 2 years and older. 12/21/2018

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
Tecentriq (atezolizumab) Genentech Intravenous In combination with bevacizumab, paclitaxel, and carboplatin, for the first-line treatment of patients with metastatic non-squamous, non-small cell lung cancer (NSq NSCLC), with no EGFR or ALK genomic tumor aberrations. 12/06/2018
Nplate® (romiplostim) Amgen Subcutaneous

Treatment of pediatric patients 1 year of age and older with immune thrombocytopenia (ITP) for at least 6 months who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

KEYTRUDA® (pembrolizumab) Merck Intravenous

Treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma.

Lynparza (olaparib) AstraZeneca Oral First-line maintenance treatment of adult patients with deleterious or suspected deleterious germline or somatic BRCA-mutated (gBRCAm or sBRCAm) advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response to first-line platinum-based chemotherapy. 12/19/2018
SPRYCEL™ (dasatinib) Bristol-Myers Squibb Oral

Treatment of pediatric patients one year of age and older with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) in combination with chemotherapy.

RAVICTI (glycerol phenylbutyrate) Horizon Pharma Oral  Expansion of usage to include infants younger than two months of age living with a urea cycle disorder (UCD). 12/21/2018

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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
diroximel fumarate (BIIB098) Alkermes and Biogen NDA Treatment of relapsing forms of multiple sclerosis (MS) Oral Not Reported
ABP 710 (biosimilar candidate to REMICADE (infliximab)) Amgen BLA Treatment of moderate to severe rheumatoid arthritis, chronic severe plaque psoriasis, moderate to severe Crohn’s disease, moderate to severe ulcerative colitis, psoriatic arthritis and ankylosing spondylitis Intravenous Not Reported
Nplate (romiplostim) Amgen sBLA Treatment of adult patients with immune thrombocytopenia (ITP) who have had ITP for 12 months or less and an insufficient response to corticosteroids, immunoglobulins or splenectomy. Subcutaneous Not Reported
Cosyntropin depot (synthetic adrenocorticotropic hormone) Assertio Therapeutics NDA Diagnostic drug in the screening of patients presumed to have adrenocortical insufficiency. Subcutaneous Early 2020
STELARA® (ustekinumab) Janssen Pharmaceutical sBLA Treatment of adults with moderately to severely active ulcerative colitis. Subcutaneous Not Reported
AR101 Aimmunen Therapeutics BLA Treatment of peanut allergy in children and adolescents ages 4–17. Oral Not Reported
Upadacitinib AbbVie NDA Treat adults with moderate to severe rheumatoid arthritis. Oral Not Reported
ROLONTIS® (eflapegrastim) Spectrum Pharmaceuticals BLA Neutropenia due to myelosuppressive cytoxic chemotherapy Subcutaneous Not Reported


Looking Ahead – Anticipated FDA actions for specialty drugs in first half of 2019

Drug Name Manufacturer Indication Route PDUFA Date

Venclexta (venetoclax)

AbbVie Acute Myelocytic Leukemia (AML, Acute Myeloblastic Leukemia) Oral


Cabometyx (cabozantinib s-malate)

Exelixis Hepatocellular Carcinoma Oral 1/14/2019

sacituzumab govitecan

Immunomedics Metastatic Breast Cancer Intravenous 1/18/2019


Ablynx Thrombotic Thrombocytopenic Purpura (Moschcowitz Disease) Subcutaneous


Keytruda (pembrolizumab)

Merck Melanoma Intravenous


Ultomiris (ravulizumab) Alexion Pharmaceuticals Paroxysmal Nocturnal Hemoglobinuria Intravenous 2/18/2019

Opdivo + Yervoy (ipilimumab + nivolumab)

Bristol-Myers Squibb Non-Small Cell Lung Cancer Intravenous


Elzonris (tagraxofusp)

Stemline Therapeutics Natural Killer Cell Lymphomas Intravenous 2/21/2019

Lonsurf (tipiracil hydrochloride + trifluridine)

Taiho Oncology Gastric Cancer Oral



Novartis Secondary Progressive Multiple Sclerosis (SPMS) Oral 3/1/2019

Tecentriq (atezolizumab)

Genentech Small-Cell Lung Cancer Intravenous


Lynparza (olaparib)

AstraZeneca Epithelial Ovarian Cancer Oral 3/29/2019
trastuzumab biosimilar Pfizer Metastatic Breast Cancer Intravenous



ADMA Biologics Primary Immune Deficiency (PID) Intravenous 4/2/2019


Karyopharm Therapeutics Refractory Multiple Myeloma Oral


Jakafi (ruxolitinib phosphate)

Incyte Graft Versus Host Disease (GVHD) Oral 4/24/2019

Eylea (aflibercept)

Regeneron Pharmaceuticals Non-Proliferative Diabetic Retinopathy (NPDR) Intravitreal


quizartinib dihydrochloride

Daiichi Sankyo Refractory Acute Myeloid Leukemia Oral 5/25/2019

onasemnogene abeparvovec

AveXis Spinal Muscular Atrophy (SMA) Intravenous


EDSIVO (celiprolol) Acer Therapeutics Ehlers-Danlos Syndrome Vascular Type (Type IV) Oral 6/25/2019
Doptelet (avatrombopag maleate) Dova Pharmaceuticals Idiopathic Thrombocytopenic Purpura (Immune Thrombocytopenic Purpura) Oral 6/30/2019

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/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ReportsBudgets/UCM591976.pdf

Pharma Intelligence Center Web Site. Accessed December 31, 2018. https://pharma.globaldata.com

U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed December 31, 2018.  https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm

U.S. Food and Drug Administration. Novel Drug Approvals for 2017. Accessed September 7, 2018https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm