RJ Health Monthly Specialty FDA Actions Newsletter – April 2019 – Volume 2 – Number 4

In April there were four new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were five supplemental drug approvals. The FDA had acted on assignment of new PDUFA dates for six 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

(Immune Globulin Intravenous, Human)

ADMA Biologics Intravenous Primary Humoral Immunodeficiency Disease in adults and adolescents (12 to 17 years of age). 04/01/2019


Amgen Subcutaneous Treatment of osteoporosis

in postmenopausal women at high risk for fracture.



AbbVie Subcutaneous Treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy 04/23/2019

(etanercept-ykro) – Biosimilar to Enbrel

Samsung Bioepis Subcutaneous Treatment of: Rheumatoid Arthritis, Polyarticular Juvenile Idiopathic Arthritis in patients aged 2 years or older, Psoriatic Arthritis (PsA), Ankylosing Spondylitis (AS), and Plaque Psoriasis (PsO) in patients 4 years or older 04/25/2019

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


Pfizer Oral Treatment of male patients with advanced or metastatic breast cancer. 04/04/2019
KEYTRUDA® (pembrolizumab) Merck Intravenous The first-line treatment of patients with stage III non-small cell lung cancer (NSCLC), who are not candidates for surgical resection or definitive chemoradiation, or metastatic NSCLC, and whose tumors express PD L1 [Tumor Proportion Score (TPS) ≥1%] as

determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations

KEYTRUDA® (pembrolizumab) Merck Intravenous First-line treatment of patients with advanced renal cell

Carcinoma in combination with axitinib.



Human Genome Sciences Intravenous Treatment of patients with active, antibody systemic lupus

erythematosus (SLE) to include children aged 5 to 17 years of age.



Sanofi Subcutaneous Reduce the risk of myocardial infarction, stroke and unstable angina requiring hospitalization in adults with established cardiovascular disease. 04/30/2019

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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
Pegfilgrastim Biosimilar Sandoz BLA Febrile neutropenia in cancer patients. Subcutaneous Not Reported
Luspatercept Celgene BLA Treat patients suffering from anemia Subcutaneous Not Reported


Aquestive Therapeutics NDA Amyotrophic lateral sclerosis Oral 11/30/2019
Darolutamide Orion & Bayer NDA non-metastatic castration-resistant prostate cancer (nmCRPC) Oral 10/29/2019
Brolucizumab Novartis BLA Wet age-related macular degeneration (wAMD) Intravitreal 10/15/2019
Eptinezumab Alder Biopharmaceuticals BLA Prevention of migraine Intravenous 02/21/2020

Looking Ahead – Anticipated FDA actions for specialty drugs in 2019 through Q4-2019

Drug Name Manufacturer Indication Route PDUFA Date
Opdivo® + Yervoy® (ipilimumab + nivolumab) Bristol-Myers Squibb Non-Small Cell Lung Cancer Intravenous 5/1/2019


Regeneron Pharmaceuticals Non-Proliferative Diabetic Retinopathy (NPDR) Intravitreal 5/13/2019

(ruxolitinib phosphate)

Incyte Graft Versus Host Disease (GVHD) Oral 5/24/2019
Onasemnogene abeparvovec AveXis Spinal Muscular Atrophy (SMA) Intrathecal; Intravenous 5/31/2019


Merck Small-Cell Lung Cancer Intravenous 6/17/2019


Agios Pharmaceuticals Acute Myelocytic Leukemia (AML, Acute Myeloblastic Leukemia) Oral 6/21/2019


Acer Therapeutics Ehlers-Danlos Syndrome Vascular Type (Type IV) Oral 6/25/2019


Celgene Follicular Lymphoma; Marginal Zone B-cell Lymphoma Oral 6/27/2019


Merck Metastatic Renal Cell Carcinoma Intravenous 6/28/2019


Alexion Pharmaceuticals Neuromyelitis Optica (Devic’s Syndrome) Intravenous 6/28/2019

(avatrombopag maleate)

Dova Pharmaceuticals Idiopathic Thrombocytopenic Purpura (Immune Thrombocytopenic Purpura) Oral 6/30/2019
Tafamidis meglumine Pfizer Amyloid Cardiomyopathy Oral 7/1/2019
Selinexor Karyopharm Therapeutics Refractory Multiple Myeloma Oral 7/6/2019
Riluzole Biohaven Pharmaceutical Amyotrophic Lateral Sclerosis Sublingual 7/21/2019
Pexidartinib Plexxikon Tenosynovial Giant Cell Tumor Oral 8/3/2019
Entrectinib F. Hoffmann-La Roche Non-Small Cell Lung Cancer; Solid Tumor Oral 8/18/2019
Upadacitinib tartrate AbbVie Rheumatoid Arthritis Oral 8/19/2019
Golodirsen Sarepta Therapeutics Duchenne Muscular Dystrophy Intravenous 8/19/2019
Polatuzumab vedotin Genentech Diffuse Large B-Cell Lymphoma Intravenous 8/19/2019
Quizartinib Daiichi Sankyo Co Refractory Acute Myeloid Leukemia; Relapsed Acute Myeloid Leukemia Oral 8/25/2019
Fedratinib Celgene Myelofibrosis; Post-Essential Thrombocythemia Myelofibrosis (Post-ET MF) Oral 9/3/2019


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


Allergan Upper Limb Muscle Spasticity Subcutaneous 9/8/2019
Teriparatide Pfenex Osteoporosis Subcutaneous 10/7/2019
Brolucizumab Novartis Wet (Neovascular / Exudative) Macular Degeneration Injection 10/15/2019
Darolutamide Orion Hormone Refractory (Castration Resistant, Androgen-Independent) Prostate Cancer Oral 10/29/2019


Aquestive Therapeutics Amyotrophic Lateral Sclerosis Oral 11/30/2019

(diroximel fumarate)

Alkermes Relapsing Remitting Multiple Sclerosis (RRMS) Oral 12/25/2019
Ubrogepant Allergan Migraine Oral 12/31/2019

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/…/CDER/ReportsBudgets/UCM591976.pdf

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

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

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