RJ Health Monthly Specialty FDA Actions Newsletter – May 2019 – Volume 2 – Number 5

In May there were three new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were 14 supplemental drug approvals. The FDA had acted on assignment of three new product reviews, although no PDUFA dates were published.

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
Vyndaquel® (tafamidis) Pfizer Oral Treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. 05/03/2019
PIQRAY® (alpelisib) Novartis Oral Treatment of postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer. 05/24/2019
Zolgensma® Novartis Intravenous Treat children with spinal muscular atrophy (SMA) 05/24/2019

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
Tibsovo® (ivosidenib) Agios Pharmaceuticals Oral Treatment of adult patients with newly-diagnosed acute myeloid leukemia (AML) who are ≥ 75 years old or who have comorbidities that preclude use of intensive induction chemotherapy. 05/02/2019
Kadcyla® (ado-trastuzumab emtansine) Genentech Intravenous Adjuvant treatment of patients with HER2-positive early breast cancer who have residual invasive disease after neoadjuvant taxane and trastuzumab based treatment. 05/03/2019
CYRAMZA® (ramucirumab) Eli Lilly Intravenous Treatment of patients with hepatocellular carcinoma who have an alpha fetoprotein of ≥400 ng/mL and have been treated with sorafenib. 05/10/2019
EYLEA® (aflibercept) Regeneron Intravitreal Treatment of patients with Diabetic Retinopathy 05/13/2019
XEOMIN® (incobotulinumtoxinA) Merz Intramuscular First-line treatment of blepharospasm (involuntary blinking) in adult patients 05/13/2019
Bavencio® (avelumab) EMD Serono Intravenous In combination with Inlyta® (axitinib) for first-line treatment of patients with advanced renal cell carcinoma (RCC). 05/14/2019


CELLTRION Intravenous Treatment of HER2-overexpressing metastatic gastric cancer or gastroesophageal junction adenocarcinoma 05/16/2019
Venclexta® (venetoclax) Abbvie Oral Treatment of chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL). 05/15/2019
TRUXIMA® (rituximab-abbs) CELLTRION Intravenous Treatment of adult patients with previously untreated diffuse Large B-cell, CD20-positive non-Hodgkin’s lymphoma (NHL) in combination with cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP) or other anthracycline-based chemotherapy regimens.


TRUXIMA® (rituximab-abbs) CELLTRION Intravenous Treatment of adult patients with previously untreated and previously treated CD20-positive chronic lymphocytic leukemia (CLL) in combination with fludarabine and cyclophosphamide (FC). 05/23/2019
JAKAFI® (ruxolitinib) Incyte Oral Treatment of acute graft versus host disease. 05/24/2019
Revlimid® (lenalidomide) Celgene Oral In combination with rituximab for the treatment of patients with previously treated follicular lymphoma and marginal zone lymphoma. 05/28/2019
Revlimid® (lenalidomide) Celgene Oral In combination with rituximab for the treatment of patients with previously treated follicular lymphoma and marginal zone lymphoma. 05/28/2019
XOSPATA® (gilteritinib) Astellas Oral Improvement in Overall Survival in those treated with gilteritinib monotherapy versus salvage chemotherapy in adult patients with relapsed (disease that has returned) or refractory (resistant to treatment) Acute Myeloid Leukemia (AML) with an FMS-like tyrosine kinase 3 (FLT3) mutation. 05/29/2019

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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
Entyvio® (vedolizumab) Takeda BLA Maintenance therapy in adults with moderately to severely active ulcerative colitis (UC) Subcutaneous Not Reported
remestemcel-L Mesoblast Limited BLA Treatment of children with steroid-refractory acute graft versus host disease (aGVHD) Intravenous Not Reported
Tazemetostat Epizyme NDA Treatment of patients with metastatic or locally advanced epithelioid sarcoma not eligible for curative surgery. Oral Not Reported

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

Drug Name Manufacturer Indication Route PDUFA Date
Keytruda® (pembrolizumab) Merck Small-Cell Lung Cancer Intravenous 6/17/2019
EDSIVO™ (celiprolol) Acer Ehlers-Danlos Syndrome Vascular Type Oral 6/25/2019
Dupixent® (dupilumab) Regeneron Nasal Polyps Intravenous 6/26/2019
Soliris® (eculizumab) Alexion Neuromyelitis Optica Intravenous 6/28/2019
Doptelet® (avatrombopag maleate) Dova Idiopathic Thrombocytopenic Purpura Oral 6/30/2019
rituximab biosimilar Pfizer Follicular Lymphoma Intravenous 7/1/2019
selinexor Karyopharm Refractory Multiple Myeloma Oral 7/6/2019
Scenesse® (afamelanotide acetate) Clinuvel Porphyria Subcutaneous 7/8/2019
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; Solid Tumor Oral 8/18/2019
upadacitinib AbbVie Rheumatoid Arthritis Oral 8/19/2019
polatuzumab Genentech Diffuse Large B-Cell Lymphoma Intravenous 8/19/2019
golodirsen Sarepta 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
Darzalex® (daratumumab) Genmab Multiple Myeloma Intravenous 9/26/2019
brolucizumab Novartis Wet (Neovascular / Exudative) Macular Degeneration Intravitreal 10/15/2019
darolutamide Orion Hormone Refractory Prostate Cancer Oral 10/29/2019
Ofev® (nintedanib) Boehringer Ingelheim Interstitial Lung Diseases Oral 11/20/2019
Exservan (riluzole) Aquestive Amyotrophic Lateral Sclerosis Oral 11/30/2019
Vumerity™ (diroximel fumarate) Alkermes Relapsing Remitting Multiple Sclerosis (RRMS) Oral 12/25/2019
eptinezumab Alder Migraine Subcutaneous 2/21/2020

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 May 1, 2019. https://pharma.globaldata.com

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

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