RJ Health Monthly Specialty FDA Actions Newsletter – May 2018 – Volume 1 – Number 5

May 2018 Specialty Drug FDA Actions

In May there were a total of five new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were eight supplemental drug approvals. The FDA had assigned eight new products for review and assigned five potential specialty products with June 2018 PDUFA dates. Finally, the FDA shifted the PDUFA dates for two products to later this year.

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 – May 2018 FDA drug approvals


Drug Name Manufacturer Route of Administration Indication Approval Date

(andexanet alfa)

Portola Pharmaceuticals Inc Intravenous Antidote indicated for patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding. May 3, 2018

(epoetin alfa-epbx)

Biosimilar for Epogen/Procrit

Hospira Injection

Treatment of anemia due to: Chronic Kidney Disease (CKD) in patients on dialysis and not on dialysis, Zidovudine in HIV-infected patients, the effects of concomitant myelosuppressive chemotherapy, and upon initiation, there is a minimum of two additional months of planned chemotherapy, Reduction of allogeneic red blood cell (RBC) transfusions in patients undergoing elective, noncardiac, nonvascular surgery.

May 15, 2018


Amgen Subcutaneous Preventive treatment of migraine in adults. May 17, 2018
(abiraterone acetate)
Sun Pharma Oral

In combination with methylprednisolone for the treatment of patients with metastatic castration resistant prostate cancer (CRPC)

May 22, 2018
PALYNZIQ™ (pegvaliase-pqpz) BioMarin Pharmaceutical Subcutaneous

Treatment of adult patients with phenylketonuria who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.

May 24, 2018

May 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
KYMRIAH™ (tisagenlecleucel) Novartis Intravenous Treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma. May 1, 2018


GlaxoSmithKline Oral Administered in combination with Mekinist (trametinib) for the treatment of anaplastic thyroid cancer (ATC) that cannot be removed by surgery or has spread to other parts of the body (metastatic), and has a type of abnormal gene, BRAF V600E (BRAF V600E mutation-positive). May 4, 2018
Mekinist® (trametinib) Novartis Oral Administered in combination with Tafinlar (dabrafenib) for the treatment of anaplastic thyroid cancer (ATC) that cannot be removed by surgery or has spread to other parts of the body (metastatic), and has a type of abnormal gene, BRAF V600E (BRAF V600E mutation-positive). May 4, 2018
DARZALEX® (daratumumab) Genmab / Janssen Biotech Intravenous In combination with bortezomib, melphalan and prednisone (VMP) for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT). May 7, 2018


Novartis Oral Treatment of children and adolescents 10 to less than 18 years of age with relapsing forms of multiple sclerosis (RMS). May 11, 2018
Actemra® (tocilizumab) Genentech Subcutaneous Treatment of active polyarticular juvenile idiopathic arthritis (PJIA) in patients two years of age and older with the subcutaneous (SC) formulation of Actemra (tocilizumab). May 14, 2018


Amgen Subcutaneous Treatment of glucocorticoid-induced osteoporosis (GIOP) in men and women at high risk of fracture May 18, 2018
XELJANZ® (tofacitinib) Pfizer Oral Treatment of adult patients in the U.S. with moderately to severely active ulcerative colitis (UC). May 30, 2018


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


Drug Name Manufacturer Type of Application Indication Route PDUFA Date
TECENTRIQ® (atezolizumab) Genentech sBLA In combination with Avastin (bevacizumab), paclitaxel and carboplatin (chemotherapy), for the initial (first-line) treatment of people with metastatic non-squamous non-small cell lung cancer (NSCLC). Intravenous September 5, 2018
Emapalumab Novimmune BLA Treat patients with primary Hemophagocytic Lymphohistiocytosis (HLH), Injectable November 20, 2018
Larotrectinib Bayer NDA Treatment of adult and pediatric patients with locally advanced or metastatic solid tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion Oral November 26, 2018
Amifampridine phosphate Catalyst Pharmaceuticals, Inc. NDA Treatment of Lambert-Eaton myasthenic syndrome (LEMS) Oral November 28, 2018
Gilteritinib fumarate Astellas Pharma Inc. NDA Treatment of adult patients who have relapsed or refractory (resistant to treatment) Acute Myeloid Leukemia (AML) with a FLT3 mutation. Oral November 29, 2018
Rituximab biosimilar1 Celltrion BLA Follicular Lymphoma, Rheumatoid Arthritis, Non-Hodgkin Lymphoma,  Chronic Lymphocytic Leukemia (CLL),  Granulomatosis with Polyangiitis (Wegener Polyangiitis),  Microscopic Polyangiitis (MPA) Intravenous 4th Quarter 2018
Coagadex® (Coagulation Factor X, Human) Bio Products Laboratory Limited sBLA Treatment of Hereditary Factor X Deficiency Intravenous 4th Quarter 2018
CABOMETYX® (cabozantinib) Exelixis, Inc. NDA Treatment for patients with previously treated advanced hepatocellular carcinoma (HCC) Oral January 14, 2019
  1. Resubmission to FDA completed and is expected to have a determination within 6 months.

Looking Ahead – Anticipated specialty drug FDA actions for 2018 and other significant changes.


Drug Name Manufacturer Indication Route PDUFA Date
Pegfilgrastim biosimilar Biocon Ltd Chemotherapy Induced Neutropenia Subcutaneous June 4, 2018

(C1 esterase inhibitor [human])

Shire Supplemental Biologics License Application (sBLA) to expand the currently approved indication to include children aged 6 years and older with hereditary angioedema (HAE). Intravenous June 20, 2018


Merck Supplemental indication for cervical cancer Intravenous June 28, 2018

(tofacitinib citrate)

Pfizer Supplemental indication for

Ulcerative Colitis

Oral June 29, 2018

(binimetinib and encorafenib)

Array BioPharma Inc Treatment of patients with BRAF-mutant advanced, unresectable or metastatic melanoma. Oral June 30, 2018


Pfizer Supplemental indication for Hormone Refractory (Castration Resistant, Androgen-Independent) Prostate Cancer Oral July 2, 2018


Merck Supplemental indication for Primary Mediastinal B-Cell Lymphoma Intravenous July 3, 2018


Kyowa Hakko Kirin Co Ltd Cutaneous T-Cell Lymphoma Intravenous September 4, 2018
Fremanezumab2 Teva Migraine Intravenous September 16, 2018
  1. FDA extended review period from June 4, 2018 to September 4, 2018
  2. Review delayed by FDA from June 16, 2018 to September 16, 2018.

Review the April specialty drug FDA actions newsletter here.

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