In September there were a total of six new drug approvals by the FDA we deemed specific to the specialty market. In addition, there were two supplemental drug approvals. The FDA had assigned six new products for review. Finally, there was one complete response letter with no current data on expected resubmission date.
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 |
LUMOXITI™
(moxetumomab pasudotox-tdfk) |
Astra Zeneca | Intravenous | Treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) | September 13, 2018 |
AJOVY™
(fremanezumab-vfrm) |
Teva | Subcutaneous | Preventive treatment of migraine in adults. | September 14, 2018 |
COPIKTRA
(duvelisib) |
VERASTEM | Oral | Treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior therapies and relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies | September 24, 2018 |
VIZIMPRO®
(dacomitinib) |
Pfizer | Oral | First-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletion or exon 21 L858R substitution mutations as detected by an FDA-approved test. | September 27, 2018 |
Emgality
(galcanezumab-gnlm) |
Eli Lilly | Subcutaneous | Preventive treatment of migraine in adults. | September 27, 2018 |
LIBTAYO®
(cemiplimab-rwlc) |
Regeneron | Intravenous | Treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation. | September 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 |
GamaSTAN
(immune globulin (human)) |
Grifols | Intramuscular | Hepatitis A virus (HAV) and measles post exposure prophylaxis. | September 4, 2018 |
Actemra®
(tocilizumab) |
Genentech | Subcutaneous | Treatment of active systemic juvenile idiopathic arthritis (SJIA) in patients two years of age and older. | September 13, 2018 |
Specialty Drug FDA Actions – New and supplemental FDA filings in the previous month
Drug Name | Manufacturer | Type of Application | Indication | Route | PDUFA Date |
TECENTRIQ®
(atezolizumab)² |
Roche | sBLA | In combination with Avastin® (bevacizumab), carboplatin and paclitaxel for the initial treatment of people with metastatic non-squamous non-small cell lung cancer (NSCLC) | Intravenous | December 05, 2018 |
INBRIJA™
(levodopa inhalation powder)¹ |
Acorda Therapeutics, Inc. | NDA | Treatment of symptoms of OFF periods in people with Parkinson’s disease taking a carbidopa/levodopa regimen. | Inhalation | January 5, 2019 |
RUCONEST
[C1 Esterase Inhibitor (recombinant)] |
Pharming | sBLA (Complete Response Letter) | Prophylaxis in patients with hereditary angioedema (HAE) | Subcutaneous | No Data Provided for Resubmission |
Tenapanor | Ardelyx, Inc | NDA | Treatment of patients with irritable bowel syndrome with constipation (IBS-C). | Oral | No Date Provided |
DOPTELET
(avatrombopag) |
Dova Pharmaceuticals, Inc. | sNDA | Treatment of adult patients with immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. | Oral | No Date Provided |
Erdafitini | Janssen Pharmaceutical | NDA | Treatment of patients with locally advanced or metastatic urothelial cancer (UC) and certain fibroblast growth factor receptor (FGFR) genetic alterations whose tumors have progressed after prior chemotherapy. | Oral | No Date Provided |
Lumateperone | Intra-Cellular Therapies, Inc. | NDA | treatment of schizophrenia | Oral | No Date Provided |
*Injectable designation applies to drugs that could be administered intravenously and subcutaneously.
- Extension of the Prescription Drug User Fee Act (PDUFA) goal date from October 5, 2018 to January 5, 2019.
- Extension of the Prescription Drug User Fee Act (PDUFA) goal date by three months.
Looking Ahead – Anticipated FDA actions for specialty drugs in 2018
Drug Name | Manufacturer | Indication | Route | PDUFA Date |
(inotersen sodium) | Ionis Pharmaceuticals | Familial Amyloid Neuropathies | Subcutaneous | 10/6/2018 |
Dupixent
(dupilumab) |
Regeneron Pharmaceuticals | Asthma | Subcutaneous | 10/20/2018 |
(cemiplimab) | Regeneron Pharmaceuticals | Squamous Cell Carcinoma | Intravenous | 10/28/2018 |
(pegfilgrastim-cbqv) | Coherus BioSciences | Febrile Neutropenia | Subcutaneous | 11/3/2018 |
(lorlatinib) | Pfizer | Non-Small Cell Lung Cancer | Oral | 11/10/2018 |
(emapalumab) | NovImmune | Hemophagocytic Lymphohistiocytosis | Intravenous | 11/20/2018 |
(larotrectinib) | Loxo Oncology | Solid Tumor | Oral | 11/26/2018 |
Firdapse
(amifampridine phosphate) |
Catalyst Pharmaceuticals | Lambert–Eaton Myasthenic Syndrome (LEMS) | Oral | 11/28/2018 |
(gilteritinib fumarate) | Astellas Pharma | Refractory Acute Myeloid Leukemia; Relapsed Acute Myeloid Leukemia | Oral | 11/29/2018 |
Promacta
(eltrombopag olamine) |
Novartis | Aplastic Anemia | Oral | 11/30/2018 |
Tecentriq
(atezolizumab) |
F. Hoffmann-La Roche | Non-Small Cell Lung Cancer | Intravenous | 12/5/2018 |
(talazoparib) | Pfizer | Metastatic Breast Cancer | Oral | 12/7/2018 |
Oncaspar-IV
(calaspargase pegol) |
Shire | Acute Lymphocytic Leukemia (ALL, Acute Lymphoblastic Leukemia) | Intravenous | 12/22/2018 |
Imbruvica
(ibrutinib) |
AbbVie | Waldenstrom Macroglobulinemia (Lymphoplasmacytic Lymphoma) | Oral | 12/25/2018 |
(glasdegib) | Pfizer | Acute Myelocytic Leukemia (AML, Acute Myeloblastic Leukemia) | Oral | 12/27/2018 |
Keytruda
(pembrolizumab) |
Merck & Co | Merkel Cell Carcinoma | Intravenous | 12/28/2018 |
Sprycel
(dasatinib) |
Bristol-Myers Squibb Co | Acute Lymphocytic Leukemia (ALL, Acute Lymphoblastic Leukemia) | Oral | 12/29/2018 |
Cabometyx
(cabozantinib s-malate) |
Exelixis | Hepatocellular Carcinoma | Oral | 1/14/2019 |
(sacituzumab govitecan) | Immunomedics | Metastatic Breast Cancer | Intravenous | 1/18/2019 |
Apomorphine Sublingual Film
(apomorphine hydrochloride) |
Sunovion Pharmaceuticals | Parkinson’s Disease | Sublingual | 1/29/2019 |
(caplacizumab) | Ablynx | Thrombotic Thrombocytopenic Purpura (Moschcowitz Disease) | Intravenous | 2/6/2019 |
(ravulizumab) | Alexion Pharmaceuticals | Paroxysmal Nocturnal Hemoglobinuria | Intravenous | 2/18/2019 |
Opdivo
(nivolumab) |
Bristol-Myers Squibb | Non-Small Cell Lung Cancer | Intravenous | 2/20/2019 |
Opdivo + Yervoy
(ipilimumab + nivolumab) |
Bristol-Myers Squibb | Non-Small Cell Lung Cancer | Intravenous | 2/20/2019 |
Terms & Abbreviations:
- aNDA: Abbreviated New Drug Application
- BLA: Biologics License Application
- FDA: Food and Drug Administration
- NDA: New Drug Application
- sBLA: supplemental Biologics License Application
- sNDA: supplemental New Drug Application
- 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
- 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
- sBLA: supplemental Biologics License Application
- 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/…/OfficeofMedicalProductsandTobacco/CDER/ReportsBudgets/UCM591976.pdf
Pharma Intelligence Center Web Site. Accessed October 1, 2018. https://pharma.globaldata.com
U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed October 1, 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