RJ Health Monthly Specialty FDA Actions Newsletter – July 2018 – Volume 1 – Number 7

In July there were a total of four new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were six supplemental drug approvals. The FDA had assigned seven new products for review.


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 – July 2018 new FDA drug approvals

Drug Name Manufacturer Route of Administration Indication Approval Date
TPOXX

(tecovirimat)

Siga Technologies Oral Treatment of patients with human smallpox disease caused by variola virus July 13, 2018
Tibsovo® (ivosidenib) Agios Pharmaceuticals Oral Treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test. July 20, 2018
NIVESTYM (filgrastim-aafi) Pfizer Subcutaneous or intravenous A biosimilar to Neupogen1 (filgrastim), for all eligible indications of the reference product. July 20, 2018
AZEDRA® (iobenguane I 131) Progenics Pharma Intravenous Treatment of adult and pediatric patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy. July 30, 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
Xeomin® (incobotulinumtoxinA) Merz Intramuscular Chronic sialorrhea in adults July 3, 2018
YERVOY®

(ipilimumab)

Bristol Myers Squibb Intravenous Treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI H) or DNA mismatch repair deficient (dMMR), metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan, in combination with nivolumab. July 10, 2018
OPDIVO

(nivolumab)

Bristol Myers Squibb Intravenous Treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI H) or DNA mismatch repair deficient (dMMR), metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan, in combination with ipilimumab. July 10, 2018
XTANDI® (enzalutamide) Astellas Oral Treatment of patients with both non-metastatic and metastatic castration-resistant prostate cancer. July 13, 2018
KISQALI®

(ribociclib)

Novartis Oral Treatment of women with hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative advanced or metastatic breast cancer. July 18, 2018
Zomacton (somatropin) Ferring Subcutaneous Idiopathic Short Stature (ISS),  Short Stature Homeobox-Containing Gene (SHOX), Turner Syndrome (TS) ,Small for Gestational Age (SGA) July 19, 2018

 


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

 

Drug Name

Manufacturer Type of Application Indication Route PDUFA Date
Herceptin (trastuzumab) co-formulation recombinant human hyaluronidase enzyme (ENHANZE® technology)

Halozyme

BLA Breast cancer Intravenous

Not Reported

EVENITY (romosozumab)

Amgen & UCB

BLA

(resubmission)

Treatment of osteoporosis in postmenopausal women at high risk for fracture Subcutaneous

Not Reported

VENCLEXTA™ (venetoclax)

Abbvie

sNDA Treat acute myeloid leukaemia (AML) Oral

Not Reported

BIVIGAM (Intravenous Immune Globulin [Human], 10%) – Relaunch application

ADMA Biologics

BLA Primary humoral immunodeficiency Intravenous

October 25, 2018

KEYTRUDA (pembrolizumab)

Merck

sBLA In combination with carboplatin-paclitaxel or nab-paclitaxel as a first-line treatment for metastatic squamous non-small cell lung cancer (NSCLC), regardless of PD-L1 expression. Intravenous

October 30, 2018

KEYTRUDA (pembrolizumab)

Merck

sBLA

Treatment for previously treated patients with advanced hepatocellular carcinoma (HCC) Intravenous

November 9, 2018

Sacituzumab govitecan

Immunomedics

BLA

Treatment of patients with metastatic triple-negative breast cancer (mTNBC) who previously received at least two prior therapies for metastatic disease.

Intravenous

January 18, 2019



Looking Ahead
– Anticipated FDA actions for specialty drugs in 2018

Drug Name Manufacturer Indication Route PDUFA Date
patisiran Alnylam Familial Amyloid Neuropathies Intravenous 11-Aug-2018
migalastat hydrochloride Amicus Therapeutics Fabry Disease Oral 13-Aug-2018
OPDIVO®

(nivolumab)

Bristol Myers Squibb Small-Cell Lung Cancer Intravenous 16-Aug-2018
LENVIMA® (lenvatinib) Eisai Hepatocellular Carcinoma Oral 24-Aug-2018
lanadelumab Shire Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) Subcutaneous 26-Aug-2018
volanesorsen Akcea Therapeutics Familial Chylomicronemia (Type I Hyperlipoproteinemia) Intravenous 30-Aug-2018
moxetumomab pasudotox MedImmune Hairy Cell Leukemia Intravenous 03-Sep-2018
Poteligeo

(mogamulizumab)

Kyowa Hakko Kirin Cutaneous T-Cell Lymphoma Intravenous 04-Sep-2018
Tecentriq®

(atezolizumab)

F. Hoffmann-La Roche Non-Small Cell Lung Cancer Intravenous 05-Sep-2018
Nucala®

(mepolizumab)

GlaxoSmithKline Chronic Obstructive Pulmonary Disease (COPD) Subcutaneous 07-Sep-2018
fremanezumab Teva Migraine Intravenous 16-Sep-2018
C1 esterase inhibitor (recombinant) Pharming Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) Intravenous 21-Sep-2018
dacomitinib Pfizer Non-Small Cell Lung Cancer Oral 30-Sep-2018
duvelisib Verastem Follicular Lymphoma; Refractory Chronic Lymphocytic Leukemia (CLL); Relapsed Chronic Lymphocytic Leukemia (CLL) Oral 05-Oct-2018
Inotersen Ionis Pharmaceuticals Familial Amyloid Neuropathies Subcutaneous 06-Oct-2018
DUPIXENT® (dupilumab) Regeneron Pharmaceuticals Asthma Intravenous 20-Oct-2018
Cemiplimab Regeneron Pharmaceuticals Squamous Cell Carcinoma Intravenous 28-Oct-2018
KEYTRUDA

(pembrolizumab)

Merck Squamous Non-Small Cell Lung Cancer Intravenous 30-Oct-2018
pegfilgrastim Coherus BioSciences Febrile Neutropenia Subcutaneous 03-Nov-2018
KEYTRUDA

(pembrolizumab)

Merck Hepatocellular Carcinoma Intravenous 09-Nov-2018
Lorlatinib Pfizer Non-Small Cell Lung Cancer Oral 10-Nov-2018
adalimumab biosimilar Sandoz Ankylosing Spondylitis (Bekhterev’s Disease); Anterior Uveitis; Axial Spondyloarthritis; Crohn’s Disease (Regional Enteritis); Hidradenitis Suppurativa; Intermediate Uveitis; Plaque Psoriasis (Psoriasis Vulgaris); Polyarticular Juvenile Idiopathic Arthritis (PJIA); Posterior Uveitis; Psoriatic Arthritis; Rheumatoid Arthritis; Ulcerative Colitis Subcutaneous 16-Nov-2018
emapalumab NovImmune Hemophagocytic Lymphohistiocytosis Intravenous 20-Nov-2018
larotrectinib Loxo Oncology Solid Tumor Oral 26-Nov-2018
Firdapse

(amifampridine)

Catalyst Pharmaceuticals Lambert–Eaton Myasthenic Syndrome (LEMS) Oral 28-Nov-2018
gilteritinib Astellas Refractory Acute Myeloid Leukemia; Relapsed Acute Myeloid Leukemia Oral 29-Nov-2018
Promacta (eltrombopag) Novartis AG Aplastic Anemia Oral 30-Nov-2018
talazoparib Pfizer Metastatic Breast Cancer Oral 07-Dec-2018
Oncaspar-IV

(calaspargase pegol)

Shire Acute Lymphocytic Leukemia (ALL, Acute Lymphoblastic Leukemia) Intravenous 22-Dec-2018
glasdegib Pfizer Acute Myelocytic Leukemia (AML, Acute Myeloblastic Leukemia) Oral 27-Dec-2018


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

References:

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/…/OfficeofMedicalProductsandTobacco/CDER/ReportsBudgets/UCM591976.pdf

Pharma Intelligence Center Web Site. Accessed August 3, 2018. https://pharma.globaldata.com

U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed August 3, 2018.  https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm

U.S. Food and Drug Administration. Novel Drug Approvals for 2017. Accessed August 3, 2018.  https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm