RJ Health Monthly Specialty FDA Actions Newsletter – October 2018 – Volume 1 – Number 10

In October there were a total of seven new drug approvals by the FDA we deemed specific to the specialty market. One of these approvals was for a biosimilar of Humira, which due to patents, is not slated to be launched until 2023. In addition, there were five supplemental drug approvals, two of which were for Hemlibra. The FDA had assigned four 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


Ionis subcutaneous Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults October 5, 2018


Leadiant Biosciences intramuscular Treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients October 5, 2018


Pfizer Oral Treatment of adult patients with deleterious or suspected deleterious germline breast cancer susceptibility gene (BRCA)-mutated (gBRCAm) human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer. October 16, 2018

(baloxavir marboxil)

Shionogi Oral Treatment of acute uncomplicated influenza in patients 12 years of age and older who have been symptomatic for no more than 48 hours October 24, 2018


Spectrum Pharmaceuticals, Intravenous Rescue after high-dose methotrexate therapy in patients with osteosarcoma. Diminishing the toxicity associated with overdosage of folic acid antagonists or impaired methotrexate elimination. Treatment of patients with metastatic colorectal cancer in combination with fluorouracil. October 19, 2018

(baloxavir marboxil)

Shionogi Oral Treatment of acute uncomplicated influenza in patients 12 years of age and older who have been symptomatic for no more than 48 hours. October 24, 2018

(adalimumab-adaz) – Biosimilar to Humira

Sandoz Subcutaneous Approved to reference product for the treatment of: Rheumatoid Arthritis, Juvenile Idiopathic Arthritis (JIA),  Psoriatic Arthritis (PsA), Ankylosing Spondylitis (AS), Adult Crohn’s Disease (CD), Ulcerative Colitis (UC), and Plaque Psoriasis (Ps) October 30, 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


Chugai Pharmaceutical Subcutaneous Treatment for hemophilia A for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors, administered once weekly, every two weeks, or every four weeks. October 4, 2018


Chugai Pharmaceutical Subcutaneous Treatment for hemophilia A dosing options of every two weeks or every four weeks in adults and children with hemophilia A with factor VIII inhibitors October 4, 2018
HUMIRA® (adalimumab) AbbVie Subcutaneous Expand the dosing recommendations to include adolescents with hidradenitis suppurativa October 16. 2018


Sanofi Subcutaneous Add-on maintenance treatment for people suffering from moderate-to-severe asthma in patients aged 12 years and above with eosinophilic phenotype or oral corticosteroid-dependent asthma. October 19, 2018


Merck Intravenous First-line treatment of patients with metastatic squamous non-small cell lung cancer, in combination with carboplatin and either paclitaxel or nab-paclitaxel. October 30, 2018

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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
LONSURF® (trifluridine/tipiracil) Taiho Oncology sNDA Treatment for patients with previously treated, advanced or metastatic gastric adenocarcinoma, including cancer of the gastroesophageal junction. Oral February 24, 2019


Novartis NDA Secondary progressive multiple sclerosis (SPMS) in adults Oral March 2019

(immune globulin)

ADMA Biologics, Inc. BLA Treatment of Primary Immune Deficiency Disease (“PIDD”) and the prevention and treatment of certain infectious diseases Intravenous April 2, 2019


Acer Therapeutics Inc NDA Treatment of vascular Ehlers-Danlos syndrome (vEDS). Oral Q2 2019

Looking Ahead – Anticipated FDA actions for specialty drugs in 2018 through mid-2019

Drug Name Manufacturer Indication Route PDUFA Date
(pegfilgrastim-cbqv) Coherus BioSciences Febrile Neutropenia Subcutaneous 11/3/2018
(lorlatinib) Pfizer Non-Small Cell Lung Cancer Oral 11/10/2018


Hemophagocytic Lymphohistiocytosis Intravenous 11/20/2018
(larotrectinib [INN]) Loxo Oncology Solid Tumor Oral 11/26/2018

(amifampridine phosphate)

Catalyst Pharmaceuticals Lambert–Eaton Myasthenic Syndrome (LEMS) Oral 11/28/2018
(gilteritinib fumarate) Astellas Refractory Acute Myeloid Leukemia; Relapsed Acute Myeloid Leukemia Oral 11/29/2018

(eltrombopag olamine)

Novartis Aplastic Anemia Oral 11/30/2018


F. Hoffmann-La Roche Non-Small Cell Lung Cancer Intravenous 12/5/2018


Pfizer Metastatic Breast Cancer Oral 12/7/2018

(calaspargase pegol)

Shire Acute Lymphocytic Leukemia Intravenous 12/22/2018


AbbVie Waldenstrom Macroglobulinemia Oral 12/25/2018
(glasdegib) Pfizer Acute Myelocytic Leukemia Oral 12/27/2018


Merck & Co Merkel Cell Carcinoma Intravenous 12/28/2018


Bristol-Myers Squibb Acute Lymphocytic Leukemia (ALL, Acute Lymphoblastic Leukemia) Oral 12/29/2018

(cabozantinib s-malate)

Exelixis Hepatocellular Carcinoma Oral 1/14/2019
(sacituzumab govitecan) Immunomedics Metastatic Breast Cancer Intravenous 1/18/2019
(caplacizumab) Ablynx Thrombotic Thrombocytopenic Purpura (Moschcowitz Disease) Intravenous 2/6/2019
(ravulizumab) Alexion Pharmaceuticals Paroxysmal Nocturnal Hemoglobinuria Intravenous; Subcutaneous 2/18/2019


Bristol-Myers Squibb Non-Small Cell Lung Cancer Intravenous 2/20/2019
Opdivo + Yervoy

(ipilimumab + nivolumab)

Bristol-Myers Squibb Non-Small Cell Lung Cancer Intravenous 2/20/2019
LONSURF® (trifluridine/tipiracil) Taiho Oncology Adenocarcinoma Oral 2/24/2019


Novartis Secondary progressive multiple sclerosis Oral  March 2019

(immune globulin)

ADMA Biologics Primary Immune Deficiency (PID) Intravenous 4/2/2019
(selinexor) Karyopharm Therapeutics Refractory Multiple Myeloma Oral 4/6/2019

(ruxolitinib phosphate)

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

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 November 1, 2018. https://pharma.globaldata.com

U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed November 1, 2018https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm

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