RJ Health Monthly Specialty FDA Actions Newsletter – November 2019 – Volume 2 – Number 11

In November there were 9 new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were 2 supplemental drug approvals. The FDA had acted on assignment of 7 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
Ibrance® (palbociclib) Pfizer Oral Treatment of advanced breast cancer. 11/01/2019
ZIEXTENZO™ (pegfilgrastim-bmez) – Biosimilar Sandoz Subcutaneously To decrease the incidence of infection, as manifested by febrile neutropenia, in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia. 11/04/2019
REBlOZYl® (luspatercept – aamt) Celgene Subcutaneously Treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions. 11/08/2019
Brukinsa™ (zanubrutinib) BeiGene Oral Treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. 11/14/2019
ABRILADA™ (adalimumab-afzb) – Biosimilar Pfizer Subcutaneously 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). 11/15/2019
ADAKVEO® (crizanlizumab-tmca) Novartis Intravenous Reduce the frequency of vasoocclusive crises in adults and pediatric patients aged 16 years and older with sickle cell disease. 11/15/2019
GIVLAARI™ (givosiran) Alnylam Pharmaceuticals Intravenous Treatment of adults with acute hepatic porphyria (AHP). 11/20/2019
EXSERVAN™ (riluzole) Aquestive Therapeutics Oral Treatment of amyotrophic lateral sclerosis (ALS). 11/22/2019
OXBRYTA™ (voxelotor) GLOBAL BLOOD THERAPEUTICS Oral Treatment of sickle cell disease in adults and pediatric patients 12 years of age and older. 11/25/2019

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
CALQUENCE® (acalabrutinib) AstraZeneca Oral Treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). 11/21/2019
ANTHIM® (obiltoxaximab) ELUSYS THERAPEUTICS Intravenous Treatment of inhalational anthrax due to B. anthracis in combination with appropriate antibacterial drugs when alternative therapies are not available or are not appropriate. 11/27/2019

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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
Satralizumab Roche BLA Neuromyelitis optica spectrum disorder (NMOSD) Injection Not Reported
Opdivo® + Yervoy® (ipilimumab + nivolumab) Bristol-Myers Squibb sBLA Treatment of patients with advanced hepatocellular carcinoma (HCC) previously treated with sorafenib. Intravenous 03/10/2020
Biosimilar to AVASTIN® (bevacizumab) Samsung Bioepis / Merck BLA Same as reference product. Intravenous Not reported
Collagenase Clostridium Histolyticum (CCH) Endo BLA Treatment of cellulite in the buttocks. Subcutaneous 07/06/2020
FINTEPLA® (ZX008, fenfluramine oral solution) Zogenix NDA Treatment of seizures associated with Dravet syndrome. Oral 03/25/2020
Obeticholic Acid (OCA) Intercept Pharmaceuticals NDA Treatment of fibrosis due to nonalcoholic steatohepatitis (NASH). Oral 03/26/2020
DaxibotulinumtoxinA Revance Therapeutics BLA Treatment of moderate to severe glabellar (frown) lines. Subcutaneous Q3/Q4 2020

Looking Ahead – Anticipated FDA actions for specialty drugs in 2019 through Q4-2020

Drug Name Manufacturer Indication Route PDUFA Date
Reblozyl® (luspatercept) Celgene Thalassemia Subcutaneous 12/4/2019
RVT-802 Enzyvant Sciences DiGeorge Syndrome Injection 12/5/2019
Rituxan® (rituximab) Genentech Microscopic Polyangiitis (MPA) Intravenous 12/11/2019
infliximab biosimilar Amgen Rheumatoid Arthritis Intravenous 12/14/2019
AV-001 Avadel Pharmaceuticals Unspecified Parenteral 12/15/2019
Vumerity™ (diroximel fumarate DR) Biogen Relapsing Remitting Multiple Sclerosis (RRMS) Oral 12/25/2019
Xtandi® (enzalutamide) Astellas Hormone-Sensitive Prostate Cancer Oral 12/31/2019
Botox® (onabotulinumtoxinA) Allergan Lower Limb Muscle Spasticity Intramuscular 1/7/2020
Adakveo® (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 Non Muscle Invasive Bladder Cancer (NMIBC) (Superficial Bladder Cancer) Intravenous 1/31/2020
Givlaari™ (givosiran) Alnylam Pharmaceuticals Inc Acute Intermittent Porphyria Subcutaneous 2/4/2020
avapritinib Blueprint Medicines Gastrointestinal Stromal Tumor (GIST) Oral 2/14/2020
eptinezumab Alder Biopharmaceuticals Migraine Subcutaneous 2/21/2020
Oxbryta™ (voxelotor) Global Blood Therapeutics Sickle Cell Disease Oral 2/26/2020
Brukinsa™ (zanubrutinib) Beigene Mantle Cell Lymphoma Oral 2/27/2020
teprotumumab Horizon Therapeutics Graves’ Ophthalmopathy Intravenous 3/8/2020
Opdivo® + Yervoy® (ipilimumab + nivolumab) Bristol-Myers Squibb Hepatocellular Carcinoma Intravenous 3/10/2020
enfortumab vedotin Astellas Metastatic Transitional (Urothelial) Tract Cancer Intravenous 3/15/2020
ozanimod Celgene Relapsing Multiple Sclerosis (RMS) Oral 3/25/2020
trastuzumab deruxtecan AstraZeneca Metastatic Breast Cancer Intravenous 3/29/2020
Imfinzi (durvalumab) AstraZeneca Small-Cell Lung Cancer Intravenous 3/31/2020
neratinib Puma Biotechnology Metastatic Breast Cancer Oral 4/30/2020
isatuximab Sanofi Multiple Myeloma Intravenous 4/30/2020
selumetinib sulfate AstraZeneca Neurofibromatoses Type I (Von Recklinghausen’s Disease) Oral 5/14/2020
risdiplam F. Hoffmann-La Roche Spinal Muscular Atrophy (SMA) Oral 5/24/2020
Instiladrin® (nadofaragene firadenovec) FKD Therapies Non Muscle Invasive Bladder Cancer (NMIBC) (Superficial Bladder Cancer) Intrapleural 5/25/2020
pemigatinib Incyte Metastatic Bile Duct Cancer Oral 5/30/2020
inebilizumab Viela Bio Neuromyelitis Optica (Devic’s Syndrome) Intravenous 6/11/2020
Xiaflex® (collagenase clostridium histolyticum) Endo Pharmaceuticals Cellulite Subcutaneous 7/6/2020
abicipar pegol Allergan Wet (Neovascular / Exudative) Macular Degeneration Intravitreal 7/9/2020
triheptanoin Ultragenyx Pharmaceutical Fatty Acid Biosynthesis Disorders Oral 7/31/2020
satralizumab F. Hoffmann-La Roche Neuromyelitis Optica (Devic’s Syndrome) Subcutaneous 8/28/2020

View our full pipeline library here:


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 December 1, 2019. https://www.fda.gov/downloads/…/CDER/ReportsBudgets/UCM591976.pdf

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

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

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