RJ Health Monthly Specialty FDA Actions Newsletter – August 2018 – Volume 1 – Number 8

RJ identified a total of seven new FDA drug approvals specific to the specialty market in August 2018, and six supplemental drug approvals. The FDA had assigned four new products for review. Review the August 2018 RJ Health monthly specialty FDA actions newsletter below for more information.

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 August 2018 FDA drug approvals

Drug Name Manufacturer Route of Administration Indication Approval Date
Mulpleta (lusutrombopag) Shionogi Oral Thrombocytopenia in adult patients with chronic liver disease (CLD) scheduled to undergo a medical procedure August 2, 2018
POTELIGEO® (mogamulizumab-kpkc) KYOWA KIRIN Intravenous Treatment of adult patients with relapsed or refractory mycosis fungoides or Sézary syndrome after at least one prior systemic therapy August 8, 2018
GALAFOLD™ (migalastat) AMICUS THERAPEUTICS Oral Treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data August 10, 2018
ONPATTRO (patisiran) Alnylam Pharma Intravenous Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults August 10, 2018
DIACOMIT (stiripentol) BIOCODEX Oral Treatment of seizures associated with Dravet syndrome in patients 2 years of age and older taking clobazam August 20, 2018
TAKHZYROTM (lanadelumab-flyo) Dyax Subcutaneous Prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients 12 years and older August 23, 2018

(Damoctocog alfa pegol)

Bayer Intravenous Routine prophylactic therapy in previously treated adults and adolescents aged 12 years and above with hemophilia A August 31, 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


Vertex Oral Expansion of the labeling to include patients 12 months and older with one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data August 15, 2018


EISAI Oral First-line treatment of patients with unresectable hepatocellular carcinoma (HCC) August 15, 2018


Bristol-Myers Squibb Intravenous Treatment of patients with metastatic small cell lung cancer (SCLC) with progression after platinum-based chemotherapy and at least one other line of therapy August 16, 2018


Regeneron Pharmaceuticals Intraocular Treatment of wet age-related macular degeneration (wet AMD) August 17, 2018
KEYTRUDA® (pembrolizumab) Merck Intravenous First-line treatment of patients with metastatic non-squamous, non-small cell lung cancer with no EGFR or ALK genomic tumor aberrations August 20, 2018
IMBRUVICA® (ibrutinib) AbbVie Oral Treatment of adult patients with Waldenström’s macroglobulinemia (WM) in combination with rituximab (RITUXAN). August 27, 2018


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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
KYPROLIS® (carfilzomib) Amgen sNDA In combination with dexamethasone for patients with relapsed or refractory multiple myeloma Intravenous Not Reported
Sprycel (dasatinib) Bristol-Myers Squibb sNDA In combination with chemotherapy for the treatment of pediatric patients with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) Oral December 29, 2018
prabotulinumtoxinA Evolus, Inc BLA Muscle Spasticity Intradermal February 2, 2019


Alexion Pharmaceuticals, Inc BLA Treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) Injectable* February 18, 2019

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

Looking Ahead – Anticipated FDA actions for specialty drugs in 2018

Drug Name Manufacturer Indication Route PDUFA Date
(moxetumomab pasudotox) MedImmune Hairy Cell Leukemia Intravenous September 3, 2018


F. Hoffmann-La Roche Non-Small Cell Lung Cancer Intravenous September 5, 2018


GlaxoSmithKline Chronic Obstructive Pulmonary Disease (COPD) Intravenous; Subcutaneous September 7, 2018
(incobotulinumtoxin A) Merz Pharmaceuticals Sialorrhoea Intramuscular September 14, 2018
(fremanezumab) Teva Pharmaceutical Industries Ltd Migraine Intravenous; Subcutaneous September 16, 2018
Fycompa (perampanel) Eisai Partial Seizure; Tonic-Clonic (Grand Mal) Seizure Oral September 28, 2018
(dacomitinib) Pfizer Non-Small Cell Lung Cancer Oral September 30, 2018
(duvelisib) Verastem Follicular Lymphoma; Refractory Chronic Lymphocytic Leukemia (CLL); Relapsed Chronic Lymphocytic Leukemia (CLL) Oral October 5, 2018


Acorda Therapeutics Parkinson’s Disease Inhalational October 5, 2018
(inotersen sodium) Ionis Pharmaceuticals Familial Amyloid Neuropathies Subcutaneous October 6, 2018
Dupixent (dupilumab) Regeneron Pharmaceuticals Asthma Intravenous; Subcutaneous October 20, 2018
(cemiplimab) Regeneron Pharmaceuticals Squamous Cell Carcinoma Intravenous October 28, 2018
Keytruda (pembrolizumab) Merck Squamous Non-Small Cell Lung Cancer Intravenous; Intravesical October 30, 2018
(pegfilgrastim-cbqv) Coherus BioSciences Febrile Neutropenia Subcutaneous November 3, 2018
Keytruda (pembrolizumab) Merck Hepatocellular Carcinoma Intravenous; Intravesical November 9, 2018
(lorlatinib) Pfizer Non-Small Cell Lung Cancer Oral November 10, 2018
adalimumab biosimilar Sandoz Ankylosing Spondylitis (Bekhterev’s Disease); Anterior Uveitis; Axial Spondyloarthritis; Crohn’s Disease (Regional Enteritis); Hidradenitis Suppurativa; Intermediate Uveitis; Plaque Psoriasis (Psoriasis Vulgaris); Polyarticular Juvenile Idiopathic Arthritis (PJIA); Posterior Uveitis; Psoriatic Arthritis; Rheumatoid Arthritis; Ulcerative Colitis Subcutaneous November 16, 2018
(emapalumab) NovImmune Hemophagocytic Lymphohistiocytosis Intravenous November 20, 2018
(larotrectinib) Loxo Oncology Inc Solid Tumor Oral November 26, 2018

(amifampridine phosphate)

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

(eltrombopag olamine)

Novartis Aplastic Anemia Oral November 30, 2018
(talazoparib) Pfizer Metastatic Breast Cancer Oral December 7, 2018

(calaspargase pegol)

Shire Acute Lymphocytic Leukemia Intravenous December 22, 2018
(glasdegib) Pfizer Acute Myelocytic Leukemia Oral December 27, 2018


Merck Merkel Cell Carcinoma Intravenous; Intravesical December 28, 2018


Bristol-Myers Squibb Acute Lymphocytic Leukemia Oral December 29, 2018

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/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ReportsBudgets/UCM591976.pdf

Pharma Intelligence Center Web Site. Accessed September 7, 2018. https://pharma.globaldata.com

U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed August 3, 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



If you enjoyed the August 2018 RJ Health Monthly Specialty FDA Actions Newsletter, you may also find the RJ Health Specialty Drug Pipeline Review collection helpful. View it here.