RJ Health Monthly Specialty FDA Actions Newsletter – November 2018 – Volume 1 – Number 11

In November there were a total of eight new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were five supplemental drug approvals. The FDA had assigned five 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 – New FDA drug approvals in the previous month

Drug Name

Manufacturer

Route of Administration

Indication

Approval Date

LORBRENA®

(lorlatinib)

Pfizer

Oral

Treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) whose disease has progressed on:

·      crizotinib and at least one other ALK inhibitor for metastatic disease; or

·      alectinib as the first ALK inhibitor therapy for metastatic disease; or

·      ceritinib as the first ALK inhibitor therapy for metastatic disease.

11/2/2018

UDENYCA

(pegfilgrastim-cbqv) – Biosimilar

Coherus BioSciences

Subcutaneous

Decrease the incidence of infection, as manifested by febrile neutropenia, in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia.

11/2/2018

Bafiertam

(monomethyl fumarate)

Banner Life Sciences

Oral

Treatment of adult patients with relapsing forms of multiple sclerosis

11/16/2018

GAMIFANT

(emapalumab-lzsg)

Novimmune

Intravenous

Treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy.

11/20/2018

DAURISMO™ (glasdegib)

Pfizer

Oral

In combination with low-dose cytarabine, for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients who are ≥75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.

11/21/2018

VITRAKVI® (larotrectinib)

LOXO ONCOLOGY

Oral

Treatment of adult and pediatric patients with solid tumors that:

• have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation,

• are metastatic or where surgical resection is likely to result in severe morbidity, and

• have no satisfactory alternative treatments or that have progressed following treatment.

11/26/2018

XOSPATA®

(gilteritinib)

Astellas

Oral

Treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test.

11/28/2018

TRUXIMA® (rituximab-abbs) – Biosimilar

Celltrion

Intravenous

Treatment of adult patients with:

•Non-Hodgkin’s Lymphoma (NHL)

  – Relapsed or refractory, low grade or follicular, CD20-positive B-cell NHL as a single agent.

  – Previously untreated follicular, CD20-positive, B-cell NHL in combination with first line chemotherapy and, in patients achieving a complete or partial response to a rituximab product in combination with chemotherapy, as single-agent maintenance therapy.

  – Non-progressing (including stable disease), low-grade, CD20-positive, B-cell NHL as a single agent after first-line cyclophosphamide, vincristine, and prednisone (CVP) chemotherapy.

11/28/2018


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
Empliciti®

(elotuzumab)

Bristol-Myers Squibb

Intravenous Empliciti® in combination with pomalidomide and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor. 11/06/2018
KEYTRUDA®

(pembrolizumab)

Merck

Intravenous

Treatment of patients with hepatocellular carcinoma who have been previously treated with sorafenib. 11/09/2018
ADCETRIS®

(brentuximab vedotin)

Seattle Genetics

Intravenous

treatment of adult patients with previously untreated systemic anaplastic large cell lymphoma or other CD30-expressing peripheral T-cell lymphomas (PTCL), including angioimmunoblastic T-cell lymphoma and PTCL not otherwise specified, in combination with cyclophosphamide, doxorubicin, and prednisone. 11/16/2018
VENCLEXTA® (venetoclax)

AbbVie

Oral

In combination with azacitidine or decitabine or low-dose cytarabine for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adults who are age 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy. 11/21/2018
Nucala

(mepolizumab)

GlaxoSmithKline

Subcutaneous

Add-on treatment for severe eosinophilic asthma in paediatric patients aged six to 11 years Not Reported

 


 

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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
Thiola

(tiopronin)

Retrophin

NDA Treatment of cystinuria Oral 06/30/2019

Lynparza

(olaparib)

AstraZeneca sNDA Maintenance treatment for ovarian cancer Oral Not Reported

Tecentriq

(atezolizumab)

Roche sBLA Combination therapy for breast cancer Intravenous Not Reported

DOPTELET

(avatrombopag)

Dova Pharmaceuticals sNDA treatment of chronic immune thrombocytopenia (ITP) in patients who have had an insufficient response to a previous treatment. ITP is an autoimmune bleeding disorder characterized by thrombocytopenia Oral 06/30/2019

ADCETRIS

 (brentuximab vedotin)

Seattle Genetics sBLA In combination with chemotherapy for the frontline treatment of patients with CD30-expressing peripheral T-cell lymphoma (PTCL). Intravenous

Not Reported


Looking Ahead – Anticipated FDA actions for specialty drugs in 2018 through mid-2019

Drug Name

Manufacturer

Indication Route PDUFA Date

Talzenna

(talazoparib)

Pfizer Metastatic Breast Cancer Oral 12/7/2018

Oncaspar-IV

(calaspargase pegol)

Shire Acute Lymphocytic Leukemia Intravenous 12/22/2018

Imbruvica

(ibrutinib)

AbbVie Waldenstrom Macroglobulinemia Oral 12/25/2018

Daurismo

(glasdegib)

Pfizer Acute Myelocytic Leukemia Oral 12/27/2018

Keytruda

(pembrolizumab)

Merck Merkel Cell Carcinoma Intravenous 12/28/2018

Sprycel

(dasatinib)

Bristol-Myers Squibb Acute Lymphocytic Leukemia Oral 12/29/2018

Venclexta

(venetoclax)

AbbVie Acute Myelocytic Leukemia Oral 1/1/2019

Cabometyx

(cabozantinib s-malate)

Exelixis Hepatocellular Carcinoma Oral 1/14/2019

sacituzumab govitecan

Immunomedics Metastatic Breast Cancer Intravenous 1/18/2019

caplacizumab

Ablynx Thrombotic Thrombocytopenic Purpura (Moschcowitz Disease) Intravenous 2/6/2019

ravulizumab

Alexion Pharmaceuticals Paroxysmal Nocturnal Hemoglobinuria Intravenous 2/18/2019

Opdivo + Yervoy

(ipilimumab + nivolumab)

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

Lonsurf

(tipiracil hydrochloride + trifluridine)

Taiho Oncology Adenocarcinoma Of The Gastroesophageal Junction Oral 2/24/2019

Tecentriq

(atezolizumab)

Genentech Metastatic Breast Cancer Intravenous 3/12/2019

Lynparza

(olaparib)

AstraZeneca Ovarian Cancer Oral 3/29/2019

trastuzumab biosimilar

Pfizer Metastatic Breast Cancer Intravenous 3/29/2019

RI-002

ADMA Biologics Primary Immune Deficiency (PID) Intravenous 4/2/2019

selinexor

Karyopharm Therapeutics Refractory Multiple Myeloma Oral 4/6/2019

Jakafi

(ruxolitinib phosphate)

Incyte Corp Graft Versus Host Disease (GVHD) Oral 4/24/2019

Eylea

(aflibercept)

Regeneron Pharmaceuticals Non-Proliferative Diabetic Retinopathy (NPDR) Intravitreal 5/13/2019

quizartinib dihydrochloride

Daiichi Sankyo Refractory Acute Myeloid Leukemia Oral

5/25/2019


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/…/ReportsBudgets/UCM591976.pdf

Pharma Intelligence Center Web Site. Accessed November 30, 2018. https://pharma.globaldata.com

U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed November 30, 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

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