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

In February there were two new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were two supplemental drug approvals. The FDA had acted on assignment of new PDUFA dates for three new products for review. Finally, there was one complete response letter issued.

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
JEUVEAU (prabotulinumtoxina-xvfs) EVOLUS Intramuscular The temporary improvement in the appearance of moderate to severe glabellar lines associated with corrugator and/or procerus muscle activity in adult patients 02/01/2019


ABLYNX NV Intravenous or subcutaneous The treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy 02/06/2019
HERCEPTIN HYLECTA (trastuzumab and hyaluronidase-oysk) GENENTECH Subcutaneous The treatment of HER2-overexpressing breast cancer. (1.1, 1.2) Select patients for therapy based on an FDA-approved companion diagnostic for trastuzumab 02/27/2019

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
KEYTRUDA® (pembrolizumab) Merck Sharp & Dohme Intravenous The adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection 02/15/2019

(trifluridine and tipiracil)

TAIHO ONCOLOGY Oral The treatment of adult patients with metastatic gastric or gastroesophageal junction adenocarcinoma previously treated with at least two prior lines of chemotherapy that included a fluoropyrimidine, a platinum, either a taxane or irinotecan, and if appropriate, HER2/neu-targeted therapy. 02/22/2019

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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
VUMERITY (diroximel fumarate) Alkermes plc and Biogen NDA Treatment of relapsing forms of multiple sclerosis (MS) Oral Q4 2019
Eptinezumab Alder BioPharmaceuticals BLA Migraine prevention Intravenous Not Provided
TIBSOVO® (ivosidenib) Agios Pharmaceuticals sNDA Treatment of patients with newly diagnosed acute myeloid leukemia (AML) with an isocitrate dehydrogenase 1 (IDH1) mutation who are not eligible for standard therapy Oral 06/21/2019

Sarepta Therapeutics NDA Duchenne muscular dystrophy (DMD) Intravenous 08/19/2019
Sacituzumab govitecan Immunomedics NDA Breast cancer Intravenous Complete Response Letter: 01/2019
Bavencio (avelumab) and Inlyta (axitinib) EMD Serono and Pfizer sBLA Advanced renal cell carcinoma (RCC) Intravenous Not Provided
Kadcyla (trastuzumab emtansine) Roche sBLA Adjuvant (after surgery) treatment of HER2-positive early breast cancer (eBC) patients having residual disease even after neoadjuvant (before surgery) therapy Intravenous Not Provided

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

Drug Name Manufacturer Indication Route PDUFA Date
(siponimod fumarate) Novartis Secondary Progressive Multiple Sclerosis (SPMS) Intravenous 3/1/2019
Tecentriq (atezolizumab) Genentech Small-Cell Lung Cancer Intravenous 3/18/2019
Lynparza (olaparib) AstraZeneca  Epithelial Ovarian Cancer Oral 3/29/2019
trastuzumab biosimilar Pfizer Metastatic Breast Cancer Intravenous 3/29/2019
RI-002 ADMA Biologics Primary Immune Deficiency (PID) Intravenous 4/2/2019
(selinexor) Karyopharm Therapeutics Refractory Multiple Myeloma Oral 4/6/2019
Opdivo + Yervoy (ipilimumab + nivolumab) Bristol-Myers Squibb Non-Small Cell Lung Cancer Intravenous 5/1/2019
Eylea (aflibercept) Regeneron Non-Proliferative Diabetic Retinopathy (NPDR) Intravitreal 5/13/2019
(quizartinib dihydrochloride) Daiichi Sankyo Refractory Acute Myeloid Leukemia; Relapsed Acute Myeloid Leukemia Oral 5/25/2019
(onasemnogene abeparvovec) AveXis Spinal Muscular Atrophy (SMA) Intravenous 5/31/2019
Keytruda (pembrolizumab) Merck Small-Cell Lung Cancer Intravenous 6/17/2019
Tibsovo (ivosidenib) Agios Pharmaceuticals Acute Myelocytic Leukemia (AML, Acute Myeloblastic Leukemia) Oral 6/21/2019
EDSIVO (celiprolol [INN]) Acer Therapeutics Ehlers-Danlos Syndrome Vascular Type (Type IV) Oral 6/25/2019
Soliris (eculizumab) Alexion  Neuromyelitis Optica (Devic’s Syndrome) Intravenous 6/28/2019
Bavencio (avelumab) Merck Metastatic Renal Cell Carcinoma Intravenous 6/28/2019
Doptelet (avatrombopag maleate) Dova Idiopathic Thrombocytopenic Purpura (Immune Thrombocytopenic Purpura) Oral 6/30/2019
(tafamidis meglumine) Pfizer Amyloid Cardiomyopathy Oral 7/1/2019
Scenesse (afamelanotide acetate) Clinuvel Porphyria (Erythropoietic Protoporphyri) Subcutaneous 7/8/2019
Otezla (apremilast) Celgene Behcet Disease Oral 7/21/2019
(pexidartinib hydrochloride) Plexxikon Tenosynovial Giant Cell Tumor Oral 8/3/2019
(entrectinib) Hoffmann-La Roche Non-Small Cell Lung Cancer; Solid Tumor Oral 8/18/2019
(upadacitinib tartrate) AbbVie Rheumatoid Arthritis Oral 8/19/2019
(polatuzumab vedotin) Genentech Diffuse Large B-Cell Lymphoma Intravenous 8/19/2019
(golodirsen) Sarepta Therapeutics Duchenne Muscular Dystrophy Intravenous 8/19/2019
(teriparatide) Pfenex Osteoporosis Subcutaneous 10/7/2019


  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/…/ReportsBudgets/UCM591976.pdf

Pharma Intelligence Center Web Site. Accessed February 28, 2019. https://pharma.globaldata.com

U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed February 28, 2019.  https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm

U.S. Food and Drug Administration. Novel Drug Approvals for 2019. Accessed February 28, 2019.  https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm629491.htm