RJ Health Systems Monthly Specialty FDA Actions (PDUFA Dates) Newsletter – January/February 2018 – Volume 1 – Number 1/2

2017 was a busy year for the Food and Drug Administration (FDA) with a 100 percent score in meeting the assigned (Prescription Drug User Fee Act) PDUFA dates for 46 products.

Of these products 33% were first in class, 39% had an orphan designation, 85% were approved on the first review, and 78% were approved first in the United States.

2018 looks to be just as busy for the FDA with many drugs slated for review or to be assigned for review.  Keeping track of all this movement can be challenging and let’s face it, very time-consuming.

In our maiden edition of the RJ Health FDA Actions Newsletter, our goal was to provide a summary of current month actions taken by the FDA and to provide a view into potential approvals in the coming month that are associated with specialty drugs.  We take pride in our monitoring process and wanted to share our expert insight with our colleagues in the industry.  We also hope that this information is helpful to you in tracking current and future drugs of relevance in the specialty market.

In January and February, there were a total of nine drug approvals by the FDA we deemed specific to the specialty market.  Two of these approvals were for novel therapeutic products, with the remaining being supplemental indications for products that are already marketed.  In addition to these approvals, the FDA had assigned seven new PDUFA dates and accepted filing for one biosimilar for approval review. Finally, there are four potential specialty products with PDUFA dates in March.

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

(lutetium Lu 177 dotatate)

AAA USA Intravenous somatostatin receptor positive GEP-NETs including foregut, midgut, and hindgut neuroendocrine tumors in adults 26-Jan-18

(bictegravir, emtricitabine, and tenofovir alafenamide)



Oral  HIV-1 infection in adults 7-Feb-18

(tezacaftor/ivacaftor and ivacaftor)

Vertex Pharmaceuticals Oral


Cystic Fibrosis in People Ages 12 and Older with Certain Mutations in the CFTR Gene 12-Feb-18




Janssen Oral Hormone Refractory (Castration Resistant, Androgen-Independent) Prostate Cancer 14-Feb-18



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




Subcutaneous Prevention Of Skeletal-Related Events In Patients With Multiple Myeloma 4-Jan-18


AstraZeneca and Merck Oral Germline BRCA-mutated (gBRCAm), human epidermal growth factor receptor 2 (HER2)-negative metastatic breast cancer who have been previously treated with chemotherapy in the neoadjuvant, adjuvant or metastatic setting 12-Jan-18




Boehringer Ingelheim


Oral Metastatic non-small cell lung cancer (NSCLC) whose tumors have non-resistant epidermal growth factor receptor (EGFR) mutations as detected by an FDA-approved test 12-Jan-18



(arsenic trioxide)

Teva Intravenous


Acute promyelocytic leukemia (APL) 12-Jan-18




Intravenous Relapsed or refractory multiple myeloma








Subcutaneous moderate-to-severe scalp psoriasis 23-Jan-18








Removal of psoriatic arthritis and plaque psoriasis indication from approvable indications due to Request from Sandoz 26-Jan-18

(glatiramer acetate)

Momenta Pharmaceuticals, Inc. Subcutaneous Multiple Sclerosis





(hydroxyprogesterone caproate )

Antares Pharma, Inc.


Intramuscular Premature Labor (Tocolysis)

(Note: New dosage form)





AstraZeneca Intravenous


Non-small cell lung cancer (NSCLC)




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


Drug Name Manufacturer Type of Application Indication Route PDUFA Date
adalimumab biosimilar Sandoz/Novartis BLA Same as Humira reference Subcutaneous None Reported

(Brentuximab Vedotin)

Seattle Genetics Inc, sBLA Advanced Classical Hodgkin lymphoma Intravenous 1-May-18



(C1 esterase inhibitor)

Shire sBLA Expand usage for approved indication to include children aged 6 years and older with hereditary angioedema (HAE) Intravenous








sBLA Pemphigus Vulgaris








Novartis sBLA Diffuse Large B-Cell Lymphoma (DLBCL) Intravenous


patisiran Alnylam Pharmaceuticals Inc, NSA Hereditary ATTR (hATTR) amyloidosis Intravenous




vosidenib Agios Pharmaceuticals, Inc. NDA Relapsed or refractory  acute myeloid leukemia (AML) with an isocitrate dehydrogenase 1 (IDH1) mutation Oral 21-Aug-18

(C1 esterase inhibitor)

Pharming Group sBLA Routine Prophylaxis of Hereditary Angioedema Intravenous 21-Sep-18


Looking Ahead – Anticipated FDA actions for specialty drugs in the next month


Drug Name Manufacturer Indication Route PDUFA Date
Yonsa (abiraterone acetate) Churchill Pharmaceutical LLC Metastatic Hormone Refractory (Castration Resistant, Androgen-Independent) Prostate Cancer Oral 19-Mar-2018
tildrakizumab Sun Pharma/Merck Plaque Psoriasis subcutaneous 26-Mar-2018
Blincyto (blinatumomab) Amgen Inc


Acute Lymphocytic Leukemia (ALL) Intravenous 29-Mar-2018


Genzyme Corp


Radiation Induced Myelosuppression subcutaneous or intravenous 29-Mar-2018


Below are links to more detailed publications regarding drugs included in the review above:

LUTATHERA ® (lutetium Lu 177 dotatate): Drug Alert – FDA Approval – lutetium Lu 177 dotatate injection, for intravenous use ( LUTATHERA ®)

Rituxan ® (Rituximab) Biosimilars: Rituxan (Rituximab) Biosimilars – 2018 Drug Pipeline Review

Terms & Abbreviations:

aNDA: Abbreviated New Drug Application
BLA: Biologics License Application
FDA: Food and Drug Administration
NDA: New Drug Application
sBLA: supplemental Biologics License Application
sNDA: supplemental New Drug Application
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
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
sBLA: supplemental Biologics License Application
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 February 16,2018. https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ReportsBudgets/UCM591976.pdf?utm_campaign=Advancing%20Health%20through%20Innovation%3A%20New%20Drug%20Approvals%20and%20Other%20Drug%20Therapy%20Advances%20of%202017&utm_medium=email&utm_source=Eloqua

Pharma Intelligence Center Web Site. Accessed February 28, 2018. https://pharma.globaldata.com

U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed February 28, 2018.  https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm

U.S. Food and Drug Administration. Novel Drug Approvals for 2017. Accessed February 28, 2018.  https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm