RJ Health Monthly Specialty FDA Actions Newsletter – October 2019 – Volume 2 – Number 10

In October there were 7 new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were 7 supplemental drug approvals. The FDA had acted on assignment of 3 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
BEOVU® (brolucizumab-dbll) Novartis Intravitreal Treatment of neovascular age-related macular degeneration. 10/07/2019
Scenesse® (afamelanotide) Clinuvel Subcutaneous To increase pain-free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria (EPP) 10/07/2019
Fluorodopa (FDOPA) The Feinstein Institute for Medical Research Intravenous A radioactive diagnostic agent indicated for use in positron emission tomography (PET) to visualize dopaminergic nerve terminals in the striatum for the evaluation of adult patients with suspected Parkinsonian syndromes (PS). 10/10/2019
VUMERITY™ (diroximel fumarate) Alkermes Oral Treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. 10/11/2019
Trikafta™ (elexacaftor, tezacaftor and ivacaftor) Vertex Oral treatment of cystic fibrosis in patients 12 years and older who have at least one F508del mutation in the CFTR gene. 10/21/2019
VUMERITY™ (diroximel fumarate) Alkermes Oral Treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. 10/29/2019
OXERVATE™ (cenegermin-bkbj) Dompé farmaceutici Ophthalmic Treatment of neurotrophic keratitis. 10/30/2019

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
Fasenra® (benralizumab) AstraZeneca Subcutaneous The addition of a single use auto-injector presentation for patient and/or caregiver administration in a non-clinical setting. 10/03/2019
WILATE (von Willebrand Factor/ Coagulation Factor VIII Complex (Human)) Octapharma Intravenous Treatment of adults and adolescents with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes and on demand treatment and control of bleeding episodes. 10/08/2019
Botox® (onabotulinumtoxinA) Allergan Intramuscular Treatment of lower limb spasticity in pediatric patients 2 to 17 years of age, excluding spasticity caused by cerebral palsy. 10/18/2019
Erelzi™ (etanercept-szzs) Sandoz Subcutaneous Addition of psoriatic arthritis and adult plaque psoriasis indications. 10/18/2019
STELARA® (ustekinumab) Janssen Subcutaneous Addition of the indication of treatment of adults with moderately to severely active ulcerative colitis 10/18/2019
ULTOMIRIS® (ravulizumab-cwvz) Alexion Intravenous Treatment of adults and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA). 10/18/2019
Zejula® (niraparib) TESARO Oral Treatment of adult patients with advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with three or more prior chemotherapy regimens and whose cancer is associated with homologous recombination deficiency (HRD) positive status defined by either: a deleterious or suspected deleterious BRCA mutation, or genomic instability and who have progressed more than six months after response to the last platinum-based chemotherapy.



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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
UX007 (triheptanoin) Ultragenyx Pharmaceutical NDA Treatment of long-chain fatty acid oxidation disorders (LC-FAOD) Oral 07/31/2020
Mirena® (levonorgestrel-releasing intrauterine system) Bayer sNDA Extend the indication to up to six years of pregnancy prevention based on results from a Phase 3 extension trial evaluating the efficacy and safety of Mirena. Intrauterine Not Reported
ROLONTIS (eflapegrastim) Spectrum BLA Treatment of neutropenia due to myelosuppressive chemotherapy Subcutaneous Not Reported

Looking Ahead – Anticipated FDA actions for specialty drugs in 2019 through Q3-2020

Drug Name Manufacturer Indication Route PDUFA Date
Ofev® (Nintedanib) Boehringer Ingelheim Interstitial Lung Diseases (Diffuse Parenchymal Lung Disease) Oral 11/20/2019
Exservan (riluzole) Aquestive Therapeutics Amyotrophic Lateral Sclerosis Oral 11/30/2019
RediTrex (methotrexate) Cumberland Pharmaceuticals Juvenile Rheumatoid Arthritis; Psoriasis; Psoriatic Arthritis; Rheumatoid Arthritis Intramuscular 12/1/2019
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
crizanlizumab Novartis Vaso-Occlusive Crisis Associated With Sickle Cell Disease Intravenous 1/15/2020
tazemetostat hydrobromide Epizyme Soft Tissue Sarcoma Oral 1/23/2020
givosiran Alnylam Pharmaceuticals 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
voxelotor Global Blood Therapeutics Sickle Cell Disease Oral 2/26/2020
zanubrutinib BeiGene Mantle Cell Lymphoma Oral 2/27/2020
teprotumumab Horizon Therapeutics Graves’ Ophthalmopathy Intravenous 3/8/2020
enfortumab vedotin Astellas Pharma Metastatic Transitional (Urothelial) Tract Cancer Intravenous 3/15/2020
Trikafta™ ((elexacaftor + ivacaftor + tezacaftor) + ivacaftor) Vertex Pharmaceuticals Cystic Fibrosis Oral 3/19/2020
ozanimod Celgene Relapsing Multiple Sclerosis (RMS) Oral 3/25/2020
trastuzumab deruxtecan AstraZeneca Metastatic Breast Cancer Intravenous 3/29/2020
neratinib Puma Biotechnology Metastatic Breast Cancer Oral 4/30/2020
isatuximab Sanofi Refractory Multiple Myeloma; Relapsed Multiple Myeloma Intravenous 4/30/2020
Keytruda® (pembrolizumab) Merck Squamous Cell Carcinoma Intravenous 6/29/2020
abicipar pegol Allergan Wet (Neovascular / Exudative) Macular Degeneration Intravitreal 7/9/2020
triheptanoin Ultragenyx 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 November 1, 2019. https://www.fda.gov/downloads/…/CDER/ReportsBudgets/UCM591976.pdf

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

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

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