RJ Health Monthly Specialty FDA Actions Newsletter – January 2019 – Volume 2 – Number 1

In January there was one new drug approval by the FDA we deemed specific to the specialty market. In addition, there were three supplemental drug approvals. The FDA had acted on assignment of new PDUFA dates for three new products for review. Finally, there were two complete response letters issued and one manufacturer triggered withdrawal of an application.

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
ONTRUZANT   (trastuzumab-dttb) SAMSUNG BIOEPIS Intravenous The treatment of HER2-overexpressing breast cancer and  metastatic gastric or gastroesophageal junction adenocarcinoma 01/18/2019

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
CABOMETYX®   (cabozantinib) EXELIXIS Oral Treatment of hepatocellular carcinoma (HCC) who have been previously treated with sorafenib 01/15/2019
IMBRUVICA® (ibrutinib) AbbVie Oral Chronic Lymphocytic Leukemia/ Small Lymphocytic Lymphoma (CLL/SLL) in Previously Untreated Patients in combination with Obinutuzumab (GAZYVA®) 01/28/2019


Lilly Intravenous In combination of KEYTRUDA® (pembrolizumab) and Platinum Chemotherapy for the First-Line Treatment of Metastatic Non-squamous Non-Small Cell Lung Cancer 01/31/2019

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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
Sacituzumab Immunomedics BLA Treatment of patients with metastatic triple-negative breast cancer (mTNBC) who have received at least two prior therapies for metastatic disease. Intravenous 01/18/2019

Complete Response Letter Received On: 01/17/2019



Actelion sNDA treatment of adults with inoperable chronic thromboembolic pulmonary hypertension (CTEPH, WHO Group 4) to improve pulmonary vascular resistance (PVR) and exercise capacity. Oral Complete Response Letter Received On: 01/16/2019


Genentech sBLA In combination with Abraxane® (albumin-bound paclitaxel; nab-paclitaxel) and carboplatin for the initial (first-line) treatment of people with metastatic non-squamous non-small cell lung cancer (NSCLC) who do not have EGFR or ALK genomic tumor aberrations. Intravenous 09/02/2019

(tafamidis meglumine)

Pfizer NDA Treatment of  transthyretin amyloid cardiomyopathy (ATTR-CM) Oral 07/01/2019


CLINUVEL NDA Prevention of phototoxicity and anaphylactoid reactions in adult patients with erythropoietic protoporphyria (EPP) Parentral (implant) 07/08/2019
Opdivo (nivolumab) and Yervoy (ipilimumab) Bristol-Myers Squibb sBLA Non-small cell lung cancer (NSCLC) Intravenous Voluntary Application Withdrawal On:   01/25/2019


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

Drug Name

Manufacturer Indication Route


(caplacizumab) Ablynx Thrombotic Thrombocytopenic Purpura (Moschcowitz Disease) Subcutaneous 2/6/2019


Merck Melanoma Intravenous 2/16/2019


Alexion Pharmaceuticals Paroxysmal Nocturnal Hemoglobinuria Intravenous 2/18/2019


Stemline Therapeutics Natural Killer Cell Lymphomas Intravenous 2/21/2019

(tipiracil hydrochloride + trifluridine)

Taiho Oncology Gastric Cancer Oral 2/24/2019

(ruxolitinib phosphate)

Incyte Graft Versus Host Disease (GVHD) Oral 2/24/2019
(siponimod fumarate) Novartis Secondary Progressive Multiple Sclerosis (SPMS) Intravenous 3/1/2019


Genentech Small-Cell Lung Cancer Intravenous 3/18/2019


AstraZeneca Pharmaceuticals 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


Regeneron Pharmaceuticals 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) Intrathecal 5/31/2019


Acer Therapeutics Ehlers-Danlos Syndrome Vascular Type (Type IV) Oral 6/25/2019

(avatrombopag maleate)

Dova Pharmaceuticals Idiopathic Thrombocytopenic Purpura (Immune Thrombocytopenic Purpura) Oral 6/30/2019
(tafamidis meglumine) Pfizer Amyloid Cardiomyopathy Oral 7/1/2019

(afamelanotide acetate)

Clinuvel Pharmaceuticals Porphyria (Erythropoietic Protoporphyri) Subcutaneous 7/8/2019


Celgene Behcet Disease Oral 7/21/2019

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/…/ReportsBudgets/UCM591976.pdf

Pharma Intelligence Center Web Site. Accessed January 30, 2019. https://pharma.globaldata.com

U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed January 30, 2019.  https://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