RJ Health Monthly Specialty FDA Actions Newsletter – April 2018 – Volume 1 – Number 4

In April there were a total of five new drug approvals by the FDA we deemed specific to the specialty market. One of these approvals was a first-time generic approval for Zavesca®. In addition, there were six supplemental drug approvals. The FDA had assigned seven new products for review. Finally, there were six potential specialty products with PDUFA dates in May.


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
Rubraca® (Rucaparib Camsylate) Clovis Oncology Inc. Oral Adult patients with deleterious BRCA mutation(germline and/or somatic)-associated epithelial ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more chemotherapies April 6, 2018
Kevzara® (Sarilumab) Sanofi Subcutaneous Treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to one or more disease-modifying antirheumatic drugs (DMARDs) April 13, 2018
First Generic: Miglustat – (Bioequivalent to Zavesca® capsules) Amerigen Pharmaceuticals, Inc Oral Treatment of mild/moderate type 1 Gaucher disease April 17, 2018
TAVALISSE™ (fostamatinib disodium hexahydrate) Rigel Pharmaceuticals, Inc Oral Treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) April 17, 2018
Crysvita® (burosumab-twza) Ultragenyx Pharmaceutical, Inc. Subcutaneous Treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older April 17, 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
Yervoy® (Ipilimumab) Bristol Myers Squibb Intravenous Use of ipilimumab, in combination with nivolumab, for the treatment of patients with intermediate or poor risk, previously untreated advanced renal cell carcinoma (RCC) April 16, 2018
Opdivo® (Nivolumab) Bristol Myers Squibb Intravenous Use of nivolumab, in combination with ipilimumab, for the treatment of patients with intermediate or poor risk, previously untreated advanced renal cell carcinoma (RCC) April 16, 2018
Tagrisso® (osimertinib mesylate) AstraZeneca Oral 1st-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumours have epidermal growth factor receptor (EGFR) mutations, (exon 19 deletions or exon 21 L858R mutations), as detected by an FDA-approved test April 16, 2018
Vonvendi® [von Willebrand factor (recombinant)], Shire Intravenous Treatment for perioperative management of bleeding in adults (age 18 and older) with von Willebrand disease (VWD) April 17, 2018
Tafinlar® (dabrafenib) Novartis Oral  In combination with Mekinist (trametinib) for the adjuvant treatment of patients with melanoma with BRAF V600E or V600K mutations, as detected by an FDA-approved test, and involvement of lymph node(s), following complete resection April 30, 2018
Kymriah™ (tisagenlecleucel)1 Novartis Intravenous Treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL), high grade B-cell lymphoma and DLBCL arising from follicular lymphoma May 1, 2018

 


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

Drug Name Manufacturer Type of Application Indication Route PDUFA Date
Moxetumomab pasudotox AstraZeneca / Medimmune BLA Treatment of adult patients with hairy cell leukemia (HCL) who have received at least two prior lines of therapy Intravenous 3rd Quarter 2018
Risankizumab AbbVie / Boehringer Ingelheim BLA Treatment of patients with moderate to severe plaque psoriasis Subcutaneous No Date Published
Opsumit® (macitentan) Actelion sNDA Treatment of adults with inoperable chronic thromboembolic pulmonary hypertension (CTEPH, WHO Group 4) to improve exercise capacity and pulmonary vascular resistance (PVR) Oral No Date Published
Dacomitinib Pfizer NDA First-line treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR-activating mutations Oral September 2018
Keytruda® (pembrolizumab) Merck sBLA In combination with pemetrexed (ALIMTA) and platinum chemotherapy (carboplatin or cisplatin) as a first-line treatment for patients with metastatic nonsquamous non-small cell lung cancer (NSCLC) Intravenous September 23, 2018
Duvelisib Verastem, Inc. NDA Treatment of relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) Oral October 5, 2018
Cemiplimab Regeneron Pharmaceuticals / Sanofi BLA Treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or patients with locally advanced CSCC who are not candidates for surgery Intravenous October 28, 2018

Looking Ahead – Anticipated FDA actions for specialty drugs in the next month

Drug Name Manufacturer Indication Route PDUFA Date
Adcetris® (brentuximab vedotin) Seattle Genetics Inc Hodgkin Lymphoma Intravenous May 4, 2018
AndexXa® (andexanet alfa)2 Portola Pharmaceuticals Inc Bleeding And Clotting Disorders Intravenous May 4, 2018
Aimovig (erenumab) Amgen Migraine Subcutaneous May 17, 2018
Avatrombopag Dova Pharmaceuticals Inc Thrombocytopenia Oral May 21, 2018
Darzalex® (daratumumab) Johnson & Johnson Multiple Myeloma (Kahler Disease) Intravenous May 21, 2018
trastuzumab biosimilar Amgen Breast Cancer; Gastric Cancer Intravenous May 28, 2018
Mogamulizumab Kyowa Hakko Kirin Co Ltd Cutaneous T-Cell Lymphoma Intravenous June 4, 2018
Fremanezumab Teva Migraine Intravenous June 16, 2018
  1. Kymriah was approved for its second indication on May 1st ahead of its PDUFA date of July 17, 2018.
  2. AndexXa® was approved by the FDA on May 3, 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

References:

Pharma Intelligence Center Web Site. Accessed May 4, 2018. https://pharma.globaldata.com

U.S. Food and Drug Administration. Drugs@FDA: FDA Approved Drug Products. Accessed May 4, 2018.  https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm

U.S. Food and Drug Administration. Novel Drug Approvals for 2018. Accessed May 4, 2018.  https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm592464.htm