RJ Health Monthly Specialty FDA Actions Newsletter – September 2019 – Volume 2 – Number 9

In September there was 1 new drug approval by the FDA we deemed specific to the specialty market. In addition, there were 9 supplemental drug approvals. The FDA had acted on assignment of 4 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
ASPARLAS™ (calaspargase pegol – mknl) Servier Pharmaceuticals Intravenous As part of a multi-agent chemotherapeutic regimen for the Treatment of acute lymphoblastic leukemia in pediatric and young adult patients age 1 month to 21 years. 09/05/2019

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
Ofev® (nintedanib) Boehringer Ingelheim Oral Slow the rate of decline in pulmonary function in patients with Systemic Sclerosis associated Interstitial Lung Disease. 09/06/2019
NUCALA (mepolizumab) GlaxoSmithKline Subcutaneous Add-on maintenance treatment of patients with severe asthma, and with an eosinophilic phenotype to include patients aged 6 to 11 years 09/12/2019
KEYTRUDA® (pembrolizumab) Merck Intravenous In combination with lenvatinib for the treatment of patients with advanced endometrial carcinoma that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation. 09/17/2019
Erleada® (apalutamide) Janssen Oral treatment of patients with metastatic castration-sensitive prostate cancer (mCSPC) 09/17/2019
LENVIMA® (lenvatinib) Eisai Oral In combination with pembrolizumab, is indicated for the treatment of patients with advanced endometrial carcinoma that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation 09/17/2019
Dysport® (abobotulinumtoxinA) Ipsen Biopharmaceuticals Intramuscular Treatment of upper limb spasticity in pediatric patients 2 years of age and older, excluding spasticity caused by cerebral palsy. 09/25/2019
DARZALEX® (daratumumab) Janssen Intravenous In combination with bortezomib, thalidomide, and dexamethasone for newly diagnosed multiple myeloma in patients who are eligible for autologous stem cell transplant (ASCT). 09/26/2019
Rituxan® (rituximab) Genentech Intravenous Expansion of the Granulomatosis with Polyangiitis (GPA) (Wegener’s Granulomatosis) and Microscopic Polyangiitis (MPA) indication to include pediatric patients 2 years of age and older. 09/27/2019
CRYSVITA® (burosumab-twza) Kyowa Kirin Subcutaneous Treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older 09/27/2019

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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
Voxelotor Global Blood Therapeutics NDA Treatment of sickle cell disease (SCD) Oral 02/26/2020
Teprotumumab Horizon Therapeutics BLA Treatment of active thyroid eye disease (TED) Intravenous 03/08/2020
Enfortumab vedotin Seattle Genetics BLA Treatment of patients with locally advanced or metastatic urothelial cancer who have received a PD-1/L1 inhibitor and who have received a platinum-containing chemotherapy in the neoadjuvant/adjuvant, locally advanced or metastatic setting. Intravenous 03/15/2020
TREMFYA® (guselkumab) Janssen sBLA Treatment of adult patients with active psoriatic arthritis Subcutaneous Not Reported

Looking Ahead – Anticipated FDA actions for specialty drugs in 2019 through Q3-2020

Drug Name Manufacturer Indication Route PDUFA Date
Scenesse® (afamelanotide acetate) Clinuvel Pharmaceuticals Porphyria (Erythropoietic Protoporphyri) Subcutaneous 10/6/2019
brolucizumab Novartis Wet (Neovascular / Exudative) Macular Degeneration Intravitreal 10/15/2019
Ultomiris™ (ravulizumab) Alexion Atypical Hemolytic Uremic Syndrome Intravenous 10/19/2019
Zejula® (niraparib) TESARO Epithelial Ovarian Cancer Oral 10/24/2019
Exservan (riluzole) Aquestive Therapeutics Amyotrophic Lateral Sclerosis Oral 11/30/2019
RediTrex™ (methotrexate) Cumberland Pharmaceuticals Juvenile Rheumatoid Arthritis; Psoriasis; Psoriatic Arthritis; Rheumatoid Arthritis Intramuscular 12/1/2019
luspatercept Celgene Thalassemia Subcutaneous 12/4/2019
RVT-802 Enzyvant Sciences DiGeorge Syndrome Injection 12/5/2019
infliximab biosimilar Amgen Rheumatoid Arthritis Intravenous 12/14/2019
Vumerity™ (diroximel fumarate) Alkermes Relapsing Remitting Multiple Sclerosis (RRMS) Oral 12/25/2019
Botox® (onabotulinumtoxinA) Allergan Lower Limb Muscle Spasticity Intramuscular 1/7/2020
crizanlizumab Novartis Vaso-Occlusive Crisis Associated With Sickle Cell Disease Intravenous 1/15/2020
tazemetostat hydrobromide Epizyme Soft Tissue Sarcoma Oral 1/23/2020
givosiran Alnylam Pharmaceuticals Acute Intermittent Porphyria; Hereditary Coproporphyria; Porphyria Variegata Subcutaneous 2/4/2020
avapritinib Blueprint Medicines Gastrointestinal Stromal Tumor (GIST) Oral 2/14/2020
KEYTRUDA® (pembrolizumab) Merck Hepatocellular Carcinoma Intravenous 2/18/2020
eptinezumab Alder Biopharmaceuticals Migraine Subcutaneous 2/21/2020
zanubrutinib BeiGene Mantle Cell Lymphoma Oral 2/27/2020
teprotumumab Horizon Therapeutics Graves’ Ophthalmopathy Intravenous 3/8/2020
enfortumab vedotin Astellas Metastatic Transitional (Urothelial) Tract Cancer Intravenous 3/15/2020
ozanimod Celgene Relapsing Multiple Sclerosis (RMS) Oral 3/25/2020
neratinib Puma Biotechnology Metastatic Breast Cancer Oral 4/30/2020
isatuximab Sanofi Refractory Multiple Myeloma; Relapsed Multiple Myeloma Intravenous 4/30/2020
abicipar pegol Allergan Wet (Neovascular / Exudative) Macular Degeneration Intravitreal 7/9/2020

View our full pipeline library here:


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 October 1, 2019. https://pharma.globaldata.com

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

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