RJ Health Monthly Specialty FDA Actions Newsletter – March 2019 – Volume 2 – Number 3

In March there were three new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were four supplemental drug approvals. The FDA had acted on the assignment of new PDUFA dates for ten new products for review. Finally, there was one product that had an extension of a PDUFA date to July.


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
Trazimera™

(trastuzumab-qyyp)

Pfizer Intravenous Adjuvant treatment of HER2-overexpressing node positive or node negative (ER/PR negative or with one high risk feature) breast cancer March 11, 2019
Mayzent® (siponimod) Novartis Oral Treat adults with relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease March 26, 2019
Mavenclad® (cladribine) EMD Serono Oral Treat relapsing forms of multiple sclerosis (MS) in adults, to include relapsing-remitting disease and active secondary progressive disease March 29, 2019

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
Tecentriq® (atezolizumab) Genentech Intravenous In combination with paclitaxel protein-bound for the treatment of adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) March 8, 2019
DUPIXENT®

(dupilumab)

Regeneron Subcutaneous Treatment of patients 12 to less than 18 years of age with moderate-to-severe atopic dermatitis March 11, 2019
Tecentriq® (atezolizumab) Genentech Intravenous In combination with carboplatin and etoposide March 18, 2019
Cimzia®

(certolizumab pegol)

UCB Subcutaneous Treatment of adults with active non-radiographic axial spondyloarthritis (nr-axSpA) with objective signs of inflammation March 28, 2019

 

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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
PRO 140

(Leronlimab)

CytoDyn Inc. BLA HIV Subcutaneous Not Reported
OFLUZA

(baloxavir marboxil)

Genentech sNDA Treatment for people at high risk of complications from the flu Oral November 4, 2019
Venclexta® (venetoclax) Roche sNDA In combination with Gazyva (obinutuzumab) in people with previously untreated chronic lymphocytic leukemia (CLL) and co-existing medical conditions Oral Not Reported
Gazyva® (obinutuzumab) Genetech sBLA In combination with Gazyva (obinutuzumab) in people with previously untreated chronic lymphocytic leukemia (CLL) and co-existing medical conditions Intravenous Not Reported
BOTOX® (onabotulinumtoxinA) Allergan sBLA Treatment of pediatric patients (2 years of age and older) with upper  limb spasticity. Injection Q2 2019
BOTOX® (onabotulinumtoxinA) Allergan sBLA Treatment of pediatric patients (2 years of age and older lower limb spasticity. Injection Q4 2019
Dupixent®

(dupilumab)

Regeneron sBLA Add-on maintenance treatment for adults with inadequately controlled severe chronic rhinosinusitis with nasal polyps (CRSwNP) Subcutaneous June 26, 2019
DARZALEX®

(daratumumab)

Janssen sBLA In combination with lenalidomide and dexamethasone (Rd) for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT) Intravenous Not Reported
Selinexor

(Extension of PDUFA date)

Karyopharm NDA Treatment of multiple myeloma Oral July 6, 2019
AR101 Aimmune Therapeutics BLA Treatment to reduce the risk of anaphylaxis following accidental exposure to peanut Oral Not Reported
Ozanimod Celgene NDA treatment of adults with relapsing forms of multiple sclerosis (RMS) Oral Not Reported

 

Looking Ahead – Anticipated FDA actions for specialty drugs in 2019 through Q4-2019

Drug Name Manufacturer Indication Route PDUFA Date
RI-002 ADMA Biologics Primary Immune Deficiency (PID) Intravenous 4/2/2019
Opdivo® + Yervoy®

(ipilimumab + nivolumab)

Bristol-Myers Squibb Non-Small Cell Lung Cancer Intravenous 5/1/2019
Eylea®

(aflibercept)

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

(ruxolitinib)

Incyte Graft Versus Host Disease (GVHD) Oral 5/24/2019
(quizartinib) Daiichi Sankyo Refractory Acute Myeloid Leukemia Oral 5/25/2019
(onasemnogene abeparvovec) AveXis Spinal Muscular Atrophy (SMA) Intrathecal 5/31/2019
Keytruda®

(pembrolizumab)

Merck Small-Cell Lung Cancer Intravenous 6/17/2019
Tibsovo®

(ivosidenib)

Agios Pharmaceuticals Acute Myelocytic Leukemia Oral 6/21/2019
EDSIVO™

(celiprolol)

Acer Therapeutics Ehlers-Danlos Syndrome Vascular Type (Type IV) Oral 6/25/2019
Revlimid®

(lenalidomide)

Celgene Follicular Lymphoma Oral 6/27/2019
Soliris®

(eculizumab)

Alexion Pharmaceuticals Neuromyelitis Optica (Devic’s Syndrome) Intravenous 6/28/2019
Bavencio®

(avelumab)

Merck Metastatic Renal Cell Carcinoma Intravenous 6/28/2019
Doptelet®

(avatrombopag)

Dova Pharmaceuticals Idiopathic Thrombocytopenic Purpura Oral 6/30/2019
(tafamidis meglumine) Pfizer Amyloid Cardiomyopathy Oral 7/1/2019
(selinexor) Karyopharm Therapeutics Refractory Multiple Myeloma Oral 7/6/2019
Scenesse®

(afamelanotide)

Clinuvel Pharmaceuticals Porphyria (Erythropoietic Protoporphyri) Subcutaneous 7/8/2019
Otezla®

(apremilast)

Celgene Behcet Disease Oral 7/21/2019
(riluzole) Biohaven Pharmaceutical Amyotrophic Lateral Sclerosis Sublingual 7/21/2019
(pexidartinib) Plexxikon Tenosynovial Giant Cell Tumor Oral 8/3/2019
(entrectinib) F. Hoffmann-La Roche Non-Small Cell Lung Cancer Oral 8/18/2019
(upadacitinib tartrate) AbbVie Rheumatoid Arthritis Oral 8/19/2019
(polatuzumab vedotin) Genentech Diffuse Large B-Cell Lymphoma Intravenous 8/19/2019
(golodirsen) Sarepta Therapeutics Duchenne Muscular Dystrophy Intravenous 8/19/2019
(fedratinib) Celgene Myelofibrosis Oral 9/3/2019
Emgality

(galcanezumab-gnlm)

Eli Lilly Cluster Headache Syndrome Subcutaneous 9/5/2019
Botox®

(onabotulinumtoxinA)

Allergan Upper Limb Muscle Spasticity Subcutaneous 9/8/2019
(teriparatide) Allergan Migraine Oral 12/31/2019

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/downloads/…/CDER/ReportsBudgets/UCM591976.pdf

Pharma Intelligence Center Web Site. Accessed April 1, 2019. https://pharma.globaldata.com

U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed April 1, 2019.  https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm

U.S. Food and Drug Administration. Novel Drug Approvals for 2019. Accessed April 1, 2019.  https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm629491.htm