RJ Health Monthly Specialty FDA Actions Newsletter – August 2019 – Volume 2 – Number 8

In August there were 4 new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were 2 supplemental drug approvals. The FDA had acted on assignment of 5 new product reviews.

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
TURALIO™ (pexidartinib) Daiichi Oral Treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. 08/02/2019
Rozlytrek™ (entrectinib) Genentech Oral Treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion with a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy. 08/15/2019
Rinvoq™ (upadacitinib) AbbVie Oral treatment of adults with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to methotrexate. 08/16/2019
INREBIC® (fedratinib) Celgene Oral Treatment of adult patients with intermediate-2 or high-risk primary or secondary (postpolycythemia vera or post-essential thrombocythemia) myelofibrosis (MF). 08/16/2019

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
Myobloc® (rimabotulinumtoxinB) Solstice Neurosciences Intramuscular Treatment of sialorrhea in adults. 08/20/2019
Taltz® (Ixekizumab) Eli Lilly Subcutaneous Treatment of adults with ankylosing spondylitis 08/23/2019

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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
Givosiran Alnylam Pharmaceuticals NDA Treatment of acute hepatic porphyria (AHP) Subcutaneous 02/04/2020
Avapritinib Blueprint Medicines NDA Treatment of adult patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST. Oral 02/14/2020
VX-445 (elexacaftor) Vertex NDA In combination with  tezacaftor and ivacaftor for the treatment of cystic fibrosis. Oral 03/19/2020
XTANDI® (enzalutamide) Astellas sNDA Treatment of men with metastatic hormone-sensitive prostate cancer (mHSPC). Oral Q4 2019
Inebilizumab Viela Bio BLA Treatment of patients with neuromyelitis optica spectrum disorder (NMOSD) Intravenous Not Reported

Looking Ahead – Anticipated FDA actions for specialty drugs in 2019 through Q2-2020

Drug Name Manufacturer Indication Route PDUFA Date
Inrebic® (fedratinib) Impact Biomedicines Post-Polycythemia Vera Myelofibrosis (PPV-MF) Oral 9/3/2019
Emgality® (galcanezumab) Eli Lilly Cluster Headache Syndrome (Cluster Headache) Subcutaneous 9/5/2019
Botox® (onabotulinumtoxinA) Allergan Upper Limb Muscle Spasticity Intramuscular 9/8/2019
Darzalex® (daratumumab) Genmab Multiple Myeloma (Kahler Disease) Intravenous 9/26/2019
Brolucizumab Novartis Wet (Neovascular / Exudative) Macular Degeneration Intravitreal 10/15/2019
Ultomiris™ (ravulizumab) Alexion Pharmaceuticals Atypical Hemolytic Uremic Syndrome Intravenous 10/19/2019
Zejula® (niraparib) TESARO Epithelial Ovarian Cancer Oral 10/24/2019
Nubeqa™ (darolutamide) Bayer Hormone Refractory (Castration Resistant, Androgen-Independent) Prostate Cancer Oral 10/28/2019
Ofev® (Ofev) Boehringer Ingelheim Interstitial Lung Diseases (Diffuse Parenchymal Lung Disease) Oral 11/20/2019
Exservan (riluzole) Aquestive Therapeutics Amyotrophic Lateral Sclerosis Oral 11/30/2019
Luspatercept Celgene Thalassemia Subcutaneous 12/4/2019
RVT-802 Enzyvant Sciences DiGeorge Syndrome Intravenous 12/5/2019
Rituxan® (rituximab) Genentech Granulomatosis with Polyangiitis (Wegener’s Granulomatosis) Intravenous 12/11/2019
Infliximab biosimilar Amgen Rheumatoid Arthritis Intravenous 12/14/2019
Vumerity™ (diroximel fumarate) Alkermes Relapsing Remitting Multiple Sclerosis (RRMS) Oral 12/25/2019
Tazemetostat Epizyme Soft Tissue Sarcoma Oral 1/23/2020
Avapritinib Blueprint Medicines Gastrointestinal Stromal Tumor (GIST) Oral 2/14/2020
Keytruda® (pembrolizumab) Merck Hepatocellular Carcinoma Intravenous 2/18/2020
Eptinezumab Alder Biopharmaceuticals Migraine Intramuscular 2/21/2020
Zanubrutinib BeiGene Mantle Cell Lymphoma Oral 2/27/2020
Elexacaftor Vertex Cystic Fibrosis Oral 3/19/2020
Ozanimod Celgene Relapsing Multiple Sclerosis (RMS) Oral 3/25/2020
Isatuximab Sanofi Refractory Multiple Myeloma Intravenous 4/30/2020

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 June 29, 2018. https://www.fda.gov/downloads/…/CDER/ReportsBudgets/UCM591976.pdf

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

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

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