RJ Health Monthly Specialty FDA Actions Newsletter – September 2018 – Volume 1 – Number 9

In September there were a total of six new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were two supplemental drug approvals. The FDA had assigned six new products for review. Finally, there was one complete response letter with no current data on expected resubmission date.


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
LUMOXITI™

(moxetumomab pasudotox-tdfk)

Astra Zeneca Intravenous Treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) September 13, 2018
AJOVY

(fremanezumab-vfrm)

Teva Subcutaneous Preventive treatment of migraine in adults. September 14, 2018
COPIKTRA

(duvelisib)

VERASTEM Oral Treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior therapies and relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies September 24, 2018
VIZIMPRO®

(dacomitinib)

Pfizer Oral First-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletion or exon 21 L858R substitution mutations as detected by an FDA-approved test. September 27, 2018
Emgality

(galcanezumab-gnlm)

Eli Lilly Subcutaneous Preventive treatment of migraine in adults. September 27, 2018
LIBTAYO®

(cemiplimab-rwlc)

Regeneron Intravenous Treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation. September 28, 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
GamaSTAN 

 (immune globulin (human))

Grifols Intramuscular Hepatitis A virus (HAV) and measles post exposure prophylaxis. September 4, 2018
Actemra®

(tocilizumab)

Genentech Subcutaneous Treatment of active systemic juvenile idiopathic arthritis (SJIA) in patients two years of age and older. September 13, 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)²

Roche sBLA In combination with Avastin® (bevacizumab), carboplatin and paclitaxel for the initial treatment of people with metastatic non-squamous non-small cell lung cancer (NSCLC) Intravenous December 05, 2018
INBRIJA™

(levodopa inhalation powder)¹

Acorda Therapeutics, Inc. NDA Treatment of symptoms of OFF periods in people with Parkinson’s disease taking a carbidopa/levodopa regimen. Inhalation January 5, 2019
RUCONEST

[C1 Esterase Inhibitor (recombinant)]

Pharming sBLA (Complete Response Letter) Prophylaxis in patients with hereditary angioedema (HAE) Subcutaneous No Data Provided for Resubmission
Tenapanor Ardelyx, Inc NDA Treatment of patients with irritable bowel syndrome with constipation (IBS-C). Oral No Date Provided
DOPTELET

(avatrombopag)

Dova Pharmaceuticals, Inc. sNDA Treatment of adult patients with immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Oral No Date Provided
Erdafitini Janssen Pharmaceutical NDA Treatment of patients with locally advanced or metastatic urothelial cancer (UC) and certain fibroblast growth factor receptor (FGFR) genetic alterations whose tumors have progressed after prior chemotherapy. Oral No Date Provided
Lumateperone Intra-Cellular Therapies, Inc. NDA treatment of schizophrenia Oral No Date Provided

*Injectable designation applies to drugs that could be administered intravenously and subcutaneously.

  1. Extension of the Prescription Drug User Fee Act (PDUFA) goal date from October 5, 2018 to January 5, 2019.
  2. Extension of the Prescription Drug User Fee Act (PDUFA) goal date by three months.

Looking Ahead – Anticipated FDA actions for specialty drugs in 2018

Drug Name Manufacturer Indication Route PDUFA Date
(inotersen sodium) Ionis Pharmaceuticals Familial Amyloid Neuropathies Subcutaneous 10/6/2018
Dupixent 

(dupilumab)

Regeneron Pharmaceuticals Asthma Subcutaneous 10/20/2018
(cemiplimab) Regeneron Pharmaceuticals Squamous Cell Carcinoma Intravenous 10/28/2018
(pegfilgrastim-cbqv) Coherus BioSciences Febrile Neutropenia Subcutaneous 11/3/2018
(lorlatinib) Pfizer Non-Small Cell Lung Cancer Oral 11/10/2018
(emapalumab) NovImmune Hemophagocytic Lymphohistiocytosis Intravenous 11/20/2018
(larotrectinib) Loxo Oncology Solid Tumor Oral 11/26/2018
Firdapse 

(amifampridine phosphate)

Catalyst Pharmaceuticals Lambert–Eaton Myasthenic Syndrome (LEMS) Oral 11/28/2018
(gilteritinib fumarate) Astellas Pharma Refractory Acute Myeloid Leukemia; Relapsed Acute Myeloid Leukemia Oral 11/29/2018
Promacta 

(eltrombopag olamine)

Novartis Aplastic Anemia Oral 11/30/2018
Tecentriq 

(atezolizumab)

F. Hoffmann-La Roche Non-Small Cell Lung Cancer Intravenous 12/5/2018
 (talazoparib) Pfizer Metastatic Breast Cancer Oral 12/7/2018
Oncaspar-IV 

(calaspargase pegol)

Shire Acute Lymphocytic Leukemia (ALL, Acute Lymphoblastic Leukemia) Intravenous 12/22/2018
Imbruvica

(ibrutinib)

AbbVie Waldenstrom Macroglobulinemia (Lymphoplasmacytic Lymphoma) Oral 12/25/2018
(glasdegib) Pfizer Acute Myelocytic Leukemia (AML, Acute Myeloblastic Leukemia) Oral 12/27/2018
Keytruda

(pembrolizumab)

Merck & Co Merkel Cell Carcinoma Intravenous 12/28/2018
Sprycel 

(dasatinib)

Bristol-Myers Squibb Co Acute Lymphocytic Leukemia (ALL, Acute Lymphoblastic Leukemia) Oral 12/29/2018
Cabometyx 

(cabozantinib s-malate)

Exelixis Hepatocellular Carcinoma Oral 1/14/2019
(sacituzumab govitecan) Immunomedics Metastatic Breast Cancer Intravenous 1/18/2019
Apomorphine Sublingual Film

(apomorphine hydrochloride)

Sunovion Pharmaceuticals Parkinson’s Disease Sublingual 1/29/2019
(caplacizumab) Ablynx Thrombotic Thrombocytopenic Purpura (Moschcowitz Disease) Intravenous 2/6/2019
(ravulizumab) Alexion Pharmaceuticals Paroxysmal Nocturnal Hemoglobinuria Intravenous 2/18/2019
Opdivo

(nivolumab)

Bristol-Myers Squibb Non-Small Cell Lung Cancer Intravenous 2/20/2019
Opdivo + Yervoy

(ipilimumab + nivolumab)

Bristol-Myers Squibb Non-Small Cell Lung Cancer Intravenous 2/20/2019

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

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 October 1, 2018. https://pharma.globaldata.com

U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed October 1, 2018.  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