RJ Health Monthly Specialty FDA Actions Newsletter – March 2018 – Volume 1 – Number 3

In March there were a total of seven drug approvals by the FDA we deemed specific to the specialty market.  In addition to these approvals, the FDA had assigned five new PDUFA dates. Finally, there were fifteen potential specialty products with PDUFA dates in April, although two of those products received a complete response letter on April 5th.  These two products represented biosimilars for Herceptin and Rituxan to be manufactured by Celltrion.


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
Trogarzo™(ibalizumab-uiyk) Taimed Biologics Intravenous HIV-1 infection 3/6/2018
Ilumya™ (tildrakizumab-asmn) Sun Pharmaceutical Subcutaneous Moderate-to-severe plaque psoriasis 3/21/2018

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
Hizentra® (Immune Globulin Subcutaneous [Human] 20% Liquid) CSL Behring Intravenous Chronic inflammatory demyelinating polyneuropathy (CIDP) 3/16/2018
Adcetris®(Brentuximab Vedotin) Seattle Genetics Inc, Intravenous Advanced Classical Hodgkin lymphoma 3/20/2018
Tasigna® (Nilotinib) Novartis Oral Pediatric patients 1 year of age or older with newly diagnosed Philadelphia chromosome positive chronic myeloid 3/22/2018
Blincyto® (Blinatumomab) Amgen Intravenous B-cell precursor acute lymphoblastic leukemia (ALL) 3/29/2018
Leukine® (Sargramostim) Genzyme Subcutaneous Radiation Induced Myelosuppression 3/29/2018


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


Drug Name Manufacturer Type of Application Indication Route PDUFA Date
Cabometyx®(cabozantinib s-malate) Exelixis sNDA Hepatocellular carcinoma (HCC) Oral Not Reported
Dupixent® (dupilumab) Regeneron Pharmaceutical sBLA Add-on maintenance treatment in certain adults and adolescents (12 years of age and older) with moderate-to-severe asthma Subcutaneous 10/20/2018
Opdivo® + Yervoy® (Ipilimumab + Nivolumab) Bristol Myers Squibb sBLA Microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) Intravenous 7/10/2018
Xeomin® ( incobotulinumtoxin A) Merz sBLA Chronic sialorrhea (excessive drooling) due to Parkinson’s Disease Intramuscular 12/1/2018
Xtandi® (Enzalutamide) Pfizer sNDA Non-metastatic Castration-resistant Prostate Cancer Oral 7/2/2018

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


Drug Name Manufacturer Indication Route PDUFA Date
Pembrolizumab Merck & Co Inc Primary Mediastinal B-Cell Lymphoma Intravenous 03-Apr-2018
Rucaparib camsylate Clovis Oncology Inc Ovarian Cancer Oral 06-Apr-2018
Abemaciclib Eli Lilly and Co Metastatic Breast Cancer Oral 12-Apr-2018
Plasminogen (human) ProMetic Life Sciences Inc Plasminogen replacement Intravenous 14-Apr-2018
Nivolumab Bristol-Myers Squibb Co Metastatic Melanoma Intravenous 16-Apr-2018
Ipilimumab + nivolumab Bristol-Myers Squibb Co Metastatic Renal Cell Carcinoma Intravenous 16-Apr-2018
Durvalumab MedImmune LLC Non-Small Cell Lung Cancer Intravenous 17-Apr-2018
Burosumab Ultragenyx Pharmaceutical Inc X-Linked Hypophosphatemic Rickets Intravenous 17-Apr-2018
Fostamatinib disodium Rigel Pharmaceuticals Inc Idiopathic Thrombocytopenic Purpura Oral 17-Apr-2018
Olaparib AstraZeneca Pharmaceuticals LP Metastatic Breast Cancer Oral 18-Apr-2018
Apalutamide Johnson & Johnson Prostate Cancer Oral 30-Apr-2018
Rituximab biosimilar1 Celltrion Inc Chronic Lymphocytic Leukemia (CLL)

Follicular Lymphoma

Wegener Polyangiitis

Microscopic Polyangiitis (MPA)

Non-Hodgkin Lymphoma

Rheumatoid Arthritis

Intravenous 30-Apr-2018
Trastuzumab biosimilar1 Celltrion Inc Breast Cancer & Gastric Cancer Intravenous 30-Apr-2018
Trastuzumab biosimilar Pfizer Inc Metastatic Breast Cancer Intravenous 30-Apr-2018

1.April 5, 2018 – Celltrion announced the FDA issued a complete response letter (CRL) for their rituximab and trastuzumab biosimilar products requesting additional information. While these actions may delay the FDA approval date, the manufacturer still anticipates approval in 2018. (Reference: The Center for Biosimilars)

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

Ibalizumab-uiyk Biologic (TrogarzoTM) – 2018 Pipeline Review

Tildrakizumab positioning in plaque psoriasis treatment – 2018 Pipeline Review

Rituxan (Rituximab) Biosimilars – 2018 Drug Pipeline Review

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 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