RJ Health Monthly Specialty FDA Actions Newsletter – June 2019 – Volume 2 – Number 6

In June there were four new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were 12 supplemental drug approvals. The FDA had acted on assignment of six 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.


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

Drug Name Manufacturer Route of Administration Indication Approval Date
NUCALA (mepolizumab) GlaxoSmithKline Subcutaneous Add-on maintenance treatment of patients with severe asthma aged 12 years and older, and with an eosinophilic phenotype; 2)treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA) 06/06/2019
POLIVY™ (polatuzumab vedotin-piiq) Genentech Intravenous Indicated in combination with bendamustine and a rituximab product for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma, not otherwise specified, after at least two prior therapies. 06/10/2019
KANJINTI™ (trastuzumab-anns) – Biosimilar to Herceptin Amgen Intravenous –  Adjuvant treatment of HER2 overexpressing node positive or node negative (ER/PR negative or with one high risk feature) breast cancer.

– In combination with paclitaxel for first-line treatment of HER2-overexpressing metastatic breast cancer.

– In combination with cisplatin and capecitabine or 5-fluorouracil, for the treatment of patients with HER2 overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma who have not received prior treatment for metastatic disease.

Zirabev™ (bevacizumab-bvzr) Pfizer Intravenous Treatment of metastatic colorectal cancer, locally advanced, recurrent or metastatic non-squamous non-small cell lung cancer, recurrent glioblastoma in adults, metastatic renal cell carcinoma, and  persistent, recurrent, or metastatic cervical cancer. 06/27/2019

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
EMGALITY® (galcanezumab-gnlm) Eli Lilly Subcutaneous Treatment of episodic cluster headache in adults. 06/04/2019
KEYTRUDA® (pembrolizumab) Merck Intravenous Treatment of patients with recurrent or metastatic head and

neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum containing chemotherapy

KEYTRUDA® (pembrolizumab) Merck Intravenous The treatment of patients with metastatic small cell lung cancer with disease progression

on or after platinum-based chemotherapy and at least one other prior line of therapy.

INFLECTRA® (infliximab-dyyb) Celltrion Intravenous For reducing signs and symptoms and inducing and maintaining

clinical remission in pediatric patients 6 years of age and older with moderately to severely active ulcerative colitis who have had an inadequate response to conventional therapy.

Botox® (onabotulinumtoxinA) Allergan Intramuscular Treatment of upper limb spasticity in pediatric patients 2 to 17

years of age.

DOPTELET® (avatrombopag) Dova Pharmaceuticals Oral Treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia

who have had an insufficient response to a previous treatment.

Renflexis® (infliximab-abda) Samsung Bioepis Intravenous Reducing signs and symptoms and inducing and maintaining clinical remission in pediatric patients 6 years of age and older with moderately to severely active ulcerative colitis who have had an inadequate response to conventional therapy. 06/26/2019
Dupixent® (dupilumab) Regeneron Subcutaneous Use of dupilumab as add-on maintenance treatment in adult patients with inadequately

controlled chronic rhinosinusitis with nasal polyposis.

SOLIRIS® (eculizumab) Alexion Intravenous The treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive 06/27/2019
DARZALEX® (daratumumab) Janssen Intravenous Treatment of adult patients with multiple myeloma in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant. 06/27/2019

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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
EDSIVO™ (celiprolol) Acer NDA vascular Ehlers-Danlos syndrome (vEDS) Oral Complete Response Letter
ZEJULA® (niraparib) GlaxoSmithKline sNDA Treatment of advanced ovarian, fallopian tube, or primary peritoneal cancer Oral 10/24/2019
ULTOMIRIS® (ravulizumab-cwvz) Alexion sBLA Treatment of people with atypical hemolytic uremic syndrome (aHUS) in order to inhibit complement-mediated thrombotic microangiopathy (TMA). Intravenous 10/19/2019
Rituxan® (rituximab) Genentech sBLA In combination with glucocorticoids (GCC), for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children two years of age and older. Intravenous No Date Provided
RVT-802 Enzyvant BLA Treatment of pediatric congenital athymia Intravenous 12/2019
SCENESSE® (afamelanotide) Clinuvel NDA Prevention of phototoxicity and anaphylactoid reactions in adult patients with erythropoietic protoporphyria (EPP) Injectable 10/06/2019

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

Drug Name Manufacturer Indication Route PDUFA Date
Vyndaqel (tafamidis meglumine) Pfizer Familial Amyloid Cardiomyopathy Oral 7/1/2019
rituximab biosimilar Pfizer Follicular Lymphoma Intravenous 7/1/2019
Emflaza (deflazacort) PTC Therapeutics Duchenne Muscular Dystrophy Oral 7/4/2019
Xpovio (selinexor) Karyopharm Therapeutics Refractory Multiple Myeloma Oral 7/7/2019
Otezla (apremilast) Celgene Behcet Disease Oral 7/21/2019
riluzole Biohaven Amyotrophic Lateral Sclerosis Sublingual 7/21/2019
pexidartinib hydrochloride Plexxikon Tenosynovial Giant Cell Tumor Oral 8/3/2019
entrectinib 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
golodirsen Sarepta Therapeutics Duchenne Muscular Dystrophy Intravenous 8/19/2019
quizartinib Daiichi Sankyo Refractory Acute Myeloid Leukemia Oral 8/25/2019
fedratinib Celgene Myelofibrosis Oral 9/3/2019
Emgality (galcanezumab) Eli Lilly Cluster Headache Syndrome 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 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; Fallopian Tube Cancer; Peritoneal Cancer Oral 10/24/2019
darolutamide Orion Corp Hormone Refractory (Castration Resistant, Androgen-Independent) Prostate Cancer Oral 10/29/2019
Ofev (nintedanib) Boehringer Ingelheim Interstitial Lung Diseases Oral 11/20/2019
Exservan (riluzole) Aquestive Therapeutics Amyotrophic Lateral Sclerosis Oral 11/30/2019
luspatercept Celgene Thalassemia Subcutaneous 12/4/2019
RVT-802 Enzyvant Sciences DiGeorge Syndrome Intravenous 12/5/2019
Rituxan (rituximab) Genentech Granulomatosis with Polyangiitis (Wegener’s Granulomatosis) Intravenous 12/11/2019
Vumerity (diroximel fumarate) Alkerme Relapsing Remitting Multiple Sclerosis (RRMS) Oral 12/25/2019
eptinezumab Alder Biopharmaceuticals Migraine Subcutaneous 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 July 1, 2019. https://pharma.globaldata.com

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

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