tools Approved Drugs sideroblasts, refractory anemia with • On June 16, the FDA extended the excess blasts, and chronic indication of Mylotarg™ (gemtuzumab • On Aug. 5, the U.S. Food and Drug myelomonocytic leukemia) and (2) ozogamicin) (Pfizer Inc., Pfizer.com) for Administration (FDA) approved Blenrep intermediate-1, intermediate-2, and newly diagnosed CD33-positive acute (belantamab mafodotin-blmf) high-risk International Prognostic myeloid leukemia to include pediatric (GlaxoSmithKline, gsk.com) for adult Scoring System groups. patients one month and older. patients with relapsed or refractory multiple myeloma who have received at • On June 16, the FDA granted accelerated • On June 11, Pfizer Inc. (pfizer.com) ® least four prior therapies, including an approval to Keytruda (pembrolizumab) announced that the FDA has approved anti-CD38 monoclonal antibody, a (Merck, merck.com) for the treatment of Nyvepria™ (pegfilgrastim-apgf), a proteasome inhibitor, and an immuno- adult and pediatric patients with biosimilar to Neulasta® (pegfilgrastim). unresectable or metastatic tumor modulatory agent. ® mutational burden-high (≥10 mutations/ • On June 10, the FDA approved Opdivo ® • On May 29, the FDA approved Cyramza megabase [mut/Mb]) solid tumors, as (nivolumab) (Bristol Myers Squibb Co., (ramucirumab) (Eli Lilly and Company, determined by an FDA-approved test, bms.com) for patients with unresectable lilly.com) in combination with erlotinib that have progressed following prior advanced, recurrent, or metastatic for first-line treatment of metastatic treatment and who have no satisfactory esophageal squamous cell carcinoma non-small cell lung cancer (NSCLC) with alternative treatment options. On June after prior fluoropyrimidine- and epidermal growth factor receptor (EGFR) 24, the FDA approved Keytruda for platinum-based chemotherapy. exon 19 deletions or exon 21 (L858R) patients with recurrent or metastatic • On June 29, the FDA approved Phesgo™ mutations. cutaneous squamous cell carcinoma that (pertuzumab, trastuzumab, and • On June 12, Merck (merck.com) is not curable by surgery or radiation. On hyaluronidase-zzxf) (Genentech, Inc., announced that the FDA has approved an June 29, the FDA approved Keytruda for gene.com) for subcutaneous injection for expanded indication for Gardasil®9 the first-line treatment of patients with the following indications. The first is use (Human Papillomavirus 9-valent unresectable or metastatic microsatellite of the agent in combination with Vaccine, Recombinant) for the preven- instability-high or mismatch repair chemotherapy as: (1) neoadjuvant tion of oropharyngeal and other head deficient colorectal cancer. treatment of patients with human and neck cancers caused by HPV Types • On July 31, the FDA granted accelerated epidermal growth factor receptor 2 16, 18, 31, 33, 45, 52, and 58. approval to Monjuvi® (tafasitamab-cxix) (HER2)-positive, locally advanced, • On July 7, the FDA approved Inqovi® (MorphoSys US Inc., morphosys.com and inflammatory, or early stage breast (decitabine and cedazuridine) (Astex Incyte, incyte.com), a CD19-directed cancer (either greater than 2 cm in Pharmaceuticals, Inc., astx.com) for adult cytolytic antibody, indicated in combina- diameter or node positive) as part of a patients with myelodysplastic syn- tion with lenalidomide for adult patients complete treatment regimen for early dromes, including the following: (1) with relapsed or refractory diffuse large breast cancer and (2) adjuvant treatment previously treated and untreated, de novo B-cell lymphoma (DLBCL) not otherwise of patients with HER2-positive early and secondary myelodysplastic syn- specified, including DLBCL arising from breast cancer at high risk of recurrence. dromes with the following French- low grade lymphoma, and who are not The second is use of the agent in American-British subtypes (refractory eligible for autologous stem cell combination with docetaxel for anemia, refractory anemia with ringed transplant. treatment of patients with HER2-positive 10 accc-cancer.org | September–October 2020 | OI metastatic breast cancer who have not Drugs in the News • Y-mAbs Therapeutics, Inc. (ymabs.com) received prior anti-HER2 therapy or announced that the BLA for Danyelza™ chemotherapy for metastatic disease. • EMD Serono (emdserono.com/us-en) (naxitamab) for the treatment of announced that the FDA has approved patients with relapsed/refractory • On June 22, the FDA granted accelerated the supplemental biologics license ® high-risk neuroblastoma has been approval to Xpovio (selinexor) ® application (BLA) for Bavencio accepted for priority review by the FDA. (Karyopharm Therapeutics, karyopharm. (avelumab) for the maintenance Leap Therapeutics, Inc. (leaptx.com) com) for adult patients with relapsed or treatment of patients with locally • announced that the FDA has granted refractory diffuse large B-cell lymphoma advanced or metastatic urothelial orphan drug designation for DKN-01 for (DLBCL), not otherwise specified, carcinoma that has not progressed with the treatment of gastric and gastro- including DLBCL arising from follicular first-line platinum-containing esophageal junction cancer. lymphoma, after at least two lines of chemotherapy. systemic therapy. • AVEO Oncology (aveooncology.com) • Black Diamond Therapeutics, Inc. announced that the FDA accepted for • On June 18, the FDA granted accelerated (blackdiamondtherapeutics.com) ™ filing its NDA seeking approval for approval to Tazverik (tazemetostat) announced that the FDA granted fast Fotivda® (tivozanib), a vascular endothe- (Epizyme, Inc., epizyme.com), an track designation to BDTX-189 for the lial growth factor receptor tyrosine enhancer of zeste homolog 2 (EZH2) treatment of adult patients with solid kinase inhibitor as a treatment for inhibitor, for adult patients with relapsed tumors harboring an allosteric HER2 relapsed or refractory renal cell or refractory follicular lymphoma whose mutation or an EGFR or HER2 Exon 20 carcinoma. tumors are positive for an EZH2 insertion mutation who have progressed mutation as detected by an FDA- following prior treatment and who have • Hutchison China MediTech Limited approved test and who have received at no satisfactory treatment options. (chi-med.com) announced that the FDA least two prior systemic therapies and for has granted fast track designation for the adult patients with relapsed or refractory • CNS Pharmaceuticals, Inc. (cnspharma. development of fruquintinib for the follicular lymphoma who have no com) announced that the FDA has treatment of patients with metastatic satisfactory alternative treatment granted orphan drug designation for its colorectal cancer who have been options. lead product Berubicin for the treatment previously treated with fluoropyrimi- of malignant gliomas. dine-, oxaliplatin-, and irinotecan-based • On July 24, the FDA granted accelerated Checkmate Pharmaceuticals, Inc. chemotherapy; an anti-vascular approval to Tecartus™ (brexucabtagene • (checkmatepharma.com) announced endothelial growth factor biological autoleucel) (Kite Pharma, kitepharma. that the FDA granted fast track designa- therapy; and, if RAS wild type, an com), a CD19-directed genetically tion to its product candidate, CMP-001, anti-EGFR therapy. modified autologous T-cell immunother- in combination with a programmed cell apy, for the treatment of adult patients • Bristol Myers Squibb (bms.com) and death receptor 1 (PD-1) blocking with relapsed or refractory mantle cell bluebird bio, Inc. (bluebirdbio.com) antibody (nivolumab or pembrolizumab) lymphoma. announced the submission of their BLA for two development programs, to the FDA for idecabtagene vicleucel • On July 31, Roche (roche.com) announced including initial treatment of patients (ide-cel; bb2121), the companies’ ® that the FDA approved Tecentriq with unresectable Stage III or Stage IV investigational B-cell maturation ® (atezolizumab) plus Cotellic melanoma to prolong the time to disease antigen-directed chimeric antigen ® (cobimetinib) and Zelboraf progression and treatment of patients receptor T-cell immunotherapy, for the (vemurafenib) for the treatment of BRAF with unresectable or metastatic treatment of adult patients with relapsed V600 mutation-positive advanced melanoma refractory to prior anti-PD-1 and refractory multiple myeloma. melanoma patients. blockade to improve the overall tumor • Merck (merck.com) announced that the response rate. • On June 15, Jazz Pharmaceuticals plc FDA accepted and granted priority review (jazzpharma.com) and its partner • Celyad Oncology SA (celyad.com) for a new supplemental BLA for Key- PharmaMar (pharmamar.com) announced that the company’s investi- truda® (pembrolizumab) as monother- announced that the FDA approved gational new drug application (NDA) for apy for the treatment of adult patients Zepzelca™ (lurbinectedin) for the CYAD-211, a short hairpin RNA (shR- with relapsed or refractory classical treatment of adult patients with NA)-based allogeneic chimeric antigen Hodgkin lymphoma. The company also metastatic small cell lung cancer with receptor T candidate and second announced that the FDA has accepted disease progression on or after non-gene edited off-the-shelf program, is and granted priority review for a new platinum-based chemotherapy. in effect with the FDA. supplemental BLA seeking accelerated OI | September–October 2020 | accc-cancer.org 11 approval for Keytruda in combination submission of an NDA to the FDA for • Roche (roche.com) announced FDA with chemotherapy for the treatment of pralsetinib