The United States Food and Drug Agency (US FDA) is the world’s leading organisation for drug approvals. MediPaper offers you insight in the US FDA approvals for oncology and haematology drugs, as reported by the US FDA – in real time.

Ivosidenib FDA approved for relapsed or refractory acute myeloid leukemia

On July 20, 2018, the US FDA approved ivosidenib (Tibsovo©, Agios Pharmaceuticals) for use in adult patients with relapsed or refractory acute myeloid leukaemia (AML) harbouring a susceptible IDH1 mutation as detected by an FDA-approved companion diagnostic (CDx) test, the Abbott RealTime IDH1 Assay.

Ribociclib FDA label updated to include pre- and perimenopausal women

On July 18, 2018, the US FDA expanded the ribociclib (Kysqali©, Novartis)  approval. Ribociclib plus an aromatase inhibitor as initial endocrine-based therapy is no longer limited to postmenopausal HR-positive, HER2-negative advanced or metastatic breast cancer patients alone and now includes pre- and perimenopausal women. Furthermore, the FDA has added the combination ribociclib plus fulvestrant as initial endocrine-based therapy for postmenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer.

FDA approved enzalutamide (XTANDI, Astellas Pharma US, Inc.), for patients with castration-resistant prostate cancer (CRPC). More Information.  July 13, 2018

Enzalutamide FDA approved for castration-resistant prostate cancer (CRPC)

On July 13, the FDA approved enzalutamide (Xtandi©, Astellas), for use in patients with castration-resistant prostate cancer (CRPC).

Ipilimumab plus nivolumab FDA approved for MSI-H and dMMR pre-treated mCRC

On July 10th, the FDA granted accelerated approval to ipilimumab (Yervoy©, Bristol-Myers Squibb) for use in combination with nivolumab (Opdivo©) for the treatment of adult and paediatric patients ≥12 years of age with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan.

Combination encorafenib plus binimetinib for unresectable/metastatic BRAF V600E or V600K-positive melanoma FDA approved

On June 27th, the FDA approved Array Biomarpharma's combination encorafenib (BRAFTOVI®) plus binimetinib (and MEKTOVI®) for patients with unresectable or metastatic melanoma harbouring a BRAF V600E or V600K mutation detected by an FDA-approved test.

FDA  has limited the use of Tecentriq and Keytruda for patients with locally advanced or metastatic urothelial cancer who are not eligible for cisplatin-containing therapy.  More Information.  June 19, 2018

FDA limits use of atezolizumab and pembrolizumab in urothelial carcinoma

On June 19, the FDA limited the use of atezolizumab and pembrolizumab for the treatment of locally advanced or metastatic urothelial cancer patients ineligible for cisplatin-containing therapy.

Bevacizumab in combination with carboplatin and paclitaxel FDA approved for several types of gynaecological cancers

 

  • On June 13, 2018, the FDA approved bevacizumab (AvastinⓇ, Genentech, Inc.) in combination with carboplatin and paclitaxel, followed by single-agent bevacizumab for use after surgical resection in patients with stage III or IV epithelial ovarian, fallopian tube, or primary peritoneal cancer.

FDA approved pembrolizumab for 3rd-line PMBCL in adult and pediatric patients

 

  • On June 13, 2018, the US FDA has granted accelerated approval to pembrolizumab (KeytrudaⓇ, Merck & Co. Inc.) for the treatment of adult and pediatric patients with primary mediastinal large B-cell lymphoma (PMBCL), relapsed after two or more prior lines of prior therapy.

FDA approved pembrolizumab for chemotherapy-refractory r/m cervical cancer with PD-L1 CPS ≥1 

 

  • On June 12, 2018 the FDA approved pembrolizumab (KeytrudaⓇ, Merck & Co. Inc.) for patients with chemotherapy-refractory recurrent or metastatic (r/m) cervical cancer and with a combined positive score (CPS)≥1 PD-L1 level expression.

FDA approves venetoclax in 2nd-line for patients with CLL or SLL

 

  • FDA granted regular approval to venetoclax (VENCLEXTA,  AbbVie Inc. and Genentech Inc.) for patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), with or without 17p deletion, who have received at least one prior therapy.

FDA approved biosimilar pegfilgrastim to decrease the chance of febrile neutropenia in patients with non-myeloid cancers on myelosuppressive chemotherapy

 

  • FDA approved Fulphila (pegfilgrastim-jmdb, Mylan GmbH), a biosimilar of Neulasta (pegfilgrastim, Amgen, Inc.) to decrease the chance of infection due to febrile neutropenia in patients receiving myelosuppressive chemotherapy with clinically significant incidence of febrile neutropenia and with non-myeloid cancers.

FDA approved avatrombopag for thrombocytopenia in CLD patient scheduled for a procedure

 

  • On May 21, the FDA approved avatrombopag (DopteletⓇ, AkaRx Inc.) for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) scheduled to undergo a procedure.

FDA safety alert: use of immunotherapy in metastatic urothelial cancer with low PD-L1 expression

On May 18, the United States (US) Food and Drug Administration (FDA) issued an alert about the decreased survival in studies with pembrolizumab (Keytruda®) and atezolizumab (Tecentriq®) as single agent first-line therapy for patients with metastatic urothelial cancer (mUCC) and low expression of the programmed death ligand 1 (PD-L1) protein.

Biosimilar epoetin alfa-epbx approved by FDA for the treatment of anemia due to chronic kidney disease, use of zidovudine for HIV, and concomitant myelosuppressive chemotherapy

 

  • On May 14, 2018 the FDA announced approval of biosimilar epoetin alfa-epbx (Retacrit®, Hospira, a subsidiary of Pfizer) for the treatment of anemia due to chronic kidney disease (CKD) in patients on dialysis and not on dialysis, use of zidovudine in patients with HIV infection, and the effects of concomitant myelosuppressive chemotherapy. Epoetin alfa-epbx is also approved for the reduction of allogeneic red blood cell transfusions in patients undergoing elective, noncardiac, nonvascular surgery. Retacrit® is a biosimilar of Epogen/Procrit (epoetin alfa, Amgen).

FDA approves daratumumab plus VMP for transplant ineligible myeloma patients

 

On 7 May 2018 the US FDA approved daratumumab (DARZALEX®) in combination with the VMP regimen (bortezomib (VELCADE®), melphalan, and prednisone) for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT).

FDA approves dabrafenib plus trametinib for anaplastic thyroid cancer with BRAF V600E mutation

 

  • FDA approves dabrafenib plus trametinib for anaplastic thyroid cancer with BRAF V600E mutation. More Information. May 4, 2018.

Tisagenlecleucel CAR-T receives additional FDA Approval for DLBCL

Tisagenlecleucel CAR-T receives additional FDA Approval for DLBCL

  • FDA approved tisagenlecleucel (KYMRIAH, Novartis Pharmaceuticals Corp.) a CD19-directed genetically modified autologous T-cell immunotherapy, for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma. More Information.

See all CAR-T therapies and indications

 

FDA granted regular approval to dabrafenib and trametinib in combination for the adjuvant treatment of patients with BRAF V600E or V600K melanoma

 

  • FDA granted regular approval to dabrafenib (TAFINLAR, Novartis Pharmaceuticals Corp.) and trametinib (MEKINIST, Novartis Pharmaceuticals Corp.) in combination 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.  More Information.

FDA approved osimertinib for the 1st-line treatment of patients with EGFR exon 19 deletions or exon 21 L858R mutations positive metastatic non-small cell lung cancer (NSCLC)

 

  • FDA approved osimertinib (Tagrisso, AstraZeneca Pharmaceuticals LP) for the 1st-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test.  More Information.

FDA approved fostamatinib disodium hexahydrate tablets for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP)

 

  • FDA approved fostamatinib disodium hexahydrate tablets (TAVALISSE, Rigel Pharmaceuticals, Inc.) for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. More Information.

FDA granted approvals to nivolumab plus ipilimumab for the treatment of intermediate or poor risk, previously untreated advanced RCC

 

FDA approved everolimus tablets for oral suspension for adjunctive treatment of adult and pediatric TSC-associated partial-onset seizures

  • FDA  granted approvals to nivolumab and ipilimumab (Opdivo and Yervoy, Bristol-Myers Squibb Co.) in combination for the treatment of intermediate or poor risk, previously untreated advanced renal cell carcinoma. More Information.  April 16, 2018
  • FDA approved rucaparib for maintenance treatment of recurrent ovarian, fallopian tube, or primary peritoneal cancer

     

    • FDA approved rucaparib for maintenance treatment of recurrent ovarian, fallopian tube, or primary peritoneal cancer. More Information.

    FDA granted accelerated approval to blinatumomab for the treatment of adult and paediatric patients with B-cell precursor ALL in first or second complete remission with MRD ≥0.1%

     

    FDA approved nilotinib for paediatric patients with newly diagnosed Ph+ CML-CP or Ph+ CML-CP resistant or intolerant to prior TKI

     

    • FDA approved nilotinib (TASIGNA, Novartis Pharmaceuticals Corporation) for pediatric patients 1 year of age or older with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) or Ph+ CML-CP resistant or intolerant to prior tyrosine-kinase inhibitor (TKI) therapy. More Information.  March 22, 2018

    FDA approved brentuximab vedotin plus chemotherapy for untreated stage III or IV classical Hodgkin lymphoma (cHL)

     

    • FDA  approved brentuximab vedotin (Adcetris, Seattle Genetics, Inc.) to treat adult patients with previously untreated stage III or IV classical Hodgkin lymphoma (cHL) in combination with chemotherapy. More Information.

    FDA approved abemaciclib plus aromatase inhibitor for untreated postmenopausal women with HR-positive HER2-negative advanced or metastatic breast cancer

     

    • FDA approved abemaciclib (VERZENIO, Eli Lilly and Company) in combination with an aromatase inhibitor as initial endocrine-based therapy for postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer. More Information.

    FDA approved durvalumab as maintenace for unresectable stage III NSCLC responding to platinum-based chemoradiation therapy

     

    • FDA approved durvalumab (Imfinzi, AstraZeneca Inc.) for patients with unresectable stage III non-small cell lung cancer (NSCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy. More Information.

    FDA approves apalutamide for non-metastatic castration-resistant prostate cancer

     

    • FDA approves apalutamide for non-metastatic castration-resistant prostate cancer. More Information.

    FDA approved abiraterone acetate tablets plus prednisone for metastatic high-risk CSPC

     

    • FDA approved abiraterone acetate (Zytiga, Janssen Biotech Inc.) tablets in combination with prednisone for metastatic high-risk castration-sensitive prostate cancer (CSPC). More Information.

    FDA approved lutetium Lu 177 dotatate for the treatment of somatostatin receptor-positive GEP-NETs

     

    • FDA approved lutetium Lu 177 dotatate (LUTATHERA, Advanced Accelerator Applications USA, Inc.) a radiolabeled somatostatin analogue, for the treatment of somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumours (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumours in adults. More Information.

    FDA granted regular approval to olaparib in the treatment of (suspected) deleterious germline BRCA-mutated, HER2-negative MBC

     

    • FDA granted regular approval to olaparib tablets (Lynparza, AstraZeneca Pharmaceuticals LP), a poly (ADP-ribose) polymerase (PARP) inhibitor, for the treatment of patients with deleterious or suspected deleterious germline BRCA-mutated (gBRCAm), HER2-negative metastatic breast cancer who have been treated with chemotherapy either in the neoadjuvant, adjuvant, or metastatic setting. More Information

     

    FDA approved a broadened indication of afatinib in the 1st-line treatment of metastatic NSCLC with non-resistant EGFR mutations

     

    • FDA granted approval to afatinib (Gilotrif, Boehringer Ingelheim Pharmaceutical, Inc.) for a broadened indication in 1st-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumours have non-resistant epidermal growth factor receptor (EGFR) mutations as detected by an FDA-approved test.  More Information.

     

    Discontinuation, post-discontinuation monitoring criteria, and guidance for treatment re-initiation in patients taking nilotinib for Ph+ CML with a sustained molecular response

    On 22 December, the FDA updated the product label for nilotinib (Tasigna®, Novartis) to include discontinuation, post-discontinuation monitoring criteria, and guidance for treatment re-initiation in patients taking nilotinib for Philadelphia chromosome positive (Ph+) chronic myeloid leukemia (CML) who have achieved a sustained molecular response (MR 4.5). More Information

    Hydroxyurea FDA approved for the reduction in frequency of painful crises and blood transfusions in paediatric sickle cell anemia patients

    On 21 December the FDA granted regular approval to hydroxyurea (Siklos®, Addmedica) for the reduction in frequency of painful crises and the need for blood transfusions in paediatric sickle cell anaemia patients ≥2 years of age with recurrent moderate to severe painful crises.

    Nivolumab approved for melanoma with lymph node involvement or metastatic disease after resection

    On 20 December 2017, the FDA granted regular approval to the anti-PD1 monoclonal antibody, nivolumab (Opdivo®, Bristol-Myers Squibb) for the adjuvant treatment of patients with melanoma with lymph node involvement or in patients with metastatic disease after complete resection. Nivolumab received prior FDA approval for the treatment of patients with unresectable or metastatic melanoma.

    Pertuzumab plus trastuzumab and chemotherapy FDA approved as adjuvant treatment of HER2-positive breast cancer with high recurrence score

    On 20 December 2017, the FDA granted regular approval to pertuzumab (Perjeta®, Roche/Genentech) for use with trastuzumab and chemotherapy as adjuvant treatment of patients with HER2-positive early breast cancer and a high recurrence risk.

    Cabozantinib FDA approved for treatment of patients with advanced renal cell carcinoma

    On 19 December 2017, the FDA granted regular approval to cabozantinib (Cabometyx®, Exelixis) for treatment of patients with advanced renal cell carcinoma (RCC).

    Bosutinib FDA approved for the treatment of patients with newly-diagnosed chronic phase Ph+ CML

    On 19 December 2017, the FDA granted accelerated approval to bosutinib (Bosulif®, Pfizer) for treatment of patients with newly-diagnosed chronic phase (CP) Philadelphia chromosome positive (Ph+) chronic myelogenous leukaemia (CML).

    Mylan biosimilar trastuzumab FDA approved for HER2+ breast or metastatic gastric cancer

    On 1 December 2017, the FDA approved Ogivri® (trastuzumab-dkst, Mylan) as a biosimilar to Herceptin® (trastuzumab, Genentech) for the treatment of patients with HER2-overexpressing breast or metastatic gastric or gastroesophageal junction adenocarcinoma.

    FoundationOne companion diagnostic FDA approved

    On 30 November 2017, the FDA granted marketing approval for the FoundationOne companion diagnostic (F1CDx®, Foundation Medicine), a next generation sequencing (NGS) based in vitro diagnostic (IVD) to detect genetic mutations in 324 genes and two genomic signatures in any solid tumour.

    Adjuvant sunitinib FDA approved for renal cell carcinoma patients at high risk of recurrence following nephrectomy.

    On 16 November 2017, the FDA approved sunitinib malate (Sutent®, Pfizer) for the adjuvant treatment of adult renal cell carcinoma patients at high risk of recurrence following nephrectomy.

    Obinutuzumab plus chemotherapy FDA approved for follicular lymphoma

    On 16 November 2017, the FDA granted regular approval to obinutuzumab (Gazyva®, Genentech) in combination with chemotherapy, followed by obinutuzumab monotherapy in adult patients with previously untreated stage II bulky, III, or IV follicular lymphoma (FL) patients who achieved at least a partial remission.

    Dasatinib FDA approved in paediatric patients with chronic phase Ph+ CML

    On 9 November 2017, the FDA granted regular approval to dasatinib (Sprycel®, Bristol-Myers Squibb) for the treatment of paediatric patients with chronic phase (CP) Philadelphia chromosome-positive (Ph+) chronic myeloid leukaemia (CML).

    Brentuximab vedotin FDA approved for pre-treated primary cutaneous anaplastic large cell lymphoma or CD30-expressing mycosis fungoides

    On 9 November 2017, the FDA granted regular approval to brentuximab vedotin (Adcetris®, Seattle Genetics) for the treatment of adult patients with primary cutaneous anaplastic large cell lymphoma (pcALCL) or CD30-expressing mycosis fungoides (MF) who underwent prior systemic therapy.

    Alectinib FDA approved for ALK-positive metastatic NSCLC

    On 6 November 2017, the FDA granted regular approval to alectinib (Alecensa®, Roche/Genentech) for the treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC), as detected by an FDA-approved test.

    Vemurafenib FDA approved for treatment of Erdheim-Chester Disease with BRAF V600 mutation

    On 6 November 2017, the FDA granted regular approval to vemurafenib (Zelboraf®, Roche) for the treatment of patients with Erdheim-Chester Disease (ECD) harbouring a BRAF V600 mutation.

    Acalabrutinib FDA approved for treatment of adult patients with pre-treated mantle cell lymphoma

    On 31 October 2017, the FDA granted accelerated approval to acalabrutinib (Calquence®, AstraZeneca under license of Acerta Pharma BV) for treatment of adult patients with mantle cell lymphoma (MCL) and pre-treated with one or more prior therapy.

    CAR-T Axicabtagene ciloleucel FDA approved for relapsed or refractory large B-cell lymphoma

    On 18 October 2017, the FDA granted regular approval to CAR-T axicabtagene ciloleucel (Yescarta®, Kite Pharma) for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of prior systemic therapy. Large B-cell lymphoma includes diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.

    Abemaciclib in combination with fulvestrant FDA approved for HR+, HER2- advanced or metastatic breast cancer progressing following endocrine therapy

    On 28 September 2017, the FDA approved abemaciclib (Verzenio®, Eli Lilly and Company) in combination with fulvestrant for women with HR-positive, HER2-negative advanced or metastatic breast cancer with disease progression following endocrine therapy.