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.

Pembrolizumab FDA approved in hepatocellular carcinoma patients pretreated with sorafenib

On 9 November 2018, the FDA granted accelerated approval to pembrolizumab (Keytruda®, Merck) for the treatment of patients with hepatocellular carcinoma (HCC) previously treated with sorafenib.

Lorlatinib FDA approved for metastatic ALK-positive NSCLC

On November 2nd 2018, lorlatinib (LORBRENA®, Pfizer) received accelerated approval by the FDA for the second- and third-line treatment of patients with metastatic, anaplastic lymphoma kinase (ALK)-positive, non-small cell lung cancer (NSCLC). Third-line patients should have had progression on crizotinib and another ALK-inhibitor for metastatic disease, and second-line patients should have had progression on either alectinib or ceritinib in the first-line for metastatic disease.

Pembrolizumab plus chemotherapy approved for metastatic squamous NSCLC

On October 30th 2018, the FDA approved the use of programmed death 1 (PD-1) inhibitor pembrolizumab (Keytruda®, Merck) in combination with carboplatin plus paclitaxel or nab-paclitaxel for the first-line treatment of patients with metastatic squamous non-small cell lung cancer (NSCLC).

PARPi talazoparib FDA approved for deleterious gBRCAm HER2‑negative breast cancer

On October 16, 2018, the FDA approved PARP-inhibitor talazoparib (Talzenna®, Pfizer) for use in patients with deleterious or suspected deleterious germline BRCA-mutated locally advanced or metastatic human epidermal growth factor receptor 2 (HER2)-negative breast cancer. Patients should be selected for the PARP inhibitor (PARPi)-treatment using an FDA-approved companion diagnostic (CDx) for talazoparib.

PD-1 mAB cemiplimab FDA approved for locally advanced and metastatic cutaneous squamous cell carcinoma (CSCC)

On September 28, 2018, the FDA approved programmed death 1 (PD-1) immune-checkpoint inhibitor cemiplimab-rwlc (Libtayo®, Regeneron) for use in patients with metastatic cutaneous squamous cell carcinoma (CSCC) and patients with locally advanced CSCC unamenable for curative surgery or curative radiation.

Dacomitinib FDA approved for the first-line treatment of metastatic EGFR-mutated NSCLC

On September 27, 2018, the FDA approved dacomitinib (Vizimpro®, Pfizer) as first-line treatment for patients with metastatic non-small cell lung cancer (NSCLC) harbouring epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or exon 21 L858R mutations as confirmed by an FDA-approved test.

Duvelisib FDA approved for adult patients with R/R CLL or SLL

On September 24, 2018, the US FDA granted regular approval to duvelisib (Copiktra®, Verastem) for the treatment of adult patients with relapsed or refractory (R/R) chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL) after ≥2 prior therapies.

Duvelisib FDA approved for adult patients with R/R follicular lymphoma

On September 24, 2018, the US FDA granted accelerated approval to duvelisib (Copiktra®, Verastem) for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) after ≥2 prior therapies.

Moxetumomab pasudotox-tdfk FDA approved for hairy cell leukaemia

On September 13, 2018, the FDA approved moxetumomab pasudotox-tdfk (Lumoxiti®, AstraZeneca) for adult patients with relapsed or refractory (R/R) hairy cell leukaemia (HCL) who had received at minimum two prior systemic therapies, of which one a purine nucleoside analogue (PNA).

Venetoclax FDA label updated to include Minimal Residual Disease negativity data

On the 11th of September 2018, the FDA updated the label of venetoclax tablets (VENCLEXTA®, Abbvie) in combination with rituximab (MabThera®, Roche) to include information about pre-treated patients with chronic lymphocytic leukemia (CLL) in the Phase III MURANO trial who had achieved Minimal Residual Disease (MRD)-negativity.

Pembrolizumab and atezolizumab USPIs updated by FDA

On the 16th of August, 2018, the FDA updated the prescribing information for pembrolizumab (Keytruda®, Merck) and atezolizumab (Tecentriq®, Roche/Genentech) and added the requirement for the use of an FDA-approved companion diagnostic test (CDx) to determine PD-L1 levels in cisplatin-ineligible patients with locally advanced or metastatic urothelial carcinoma.

Nivolumab FDA approved for patients with metastatic small cell lung cancer (SCLC)

On the 16th of August 2018, the FDA granted accelerated approval to nivolumab (Opdivo®, Bristol-Myers Squibb) for use in patients with metastatic small cell lung cancer (SCLC) who progressed after two lines of therapy, including one line of platinum-based chemotherapy.

Pembrolizumab plus pemetrexed-platinum approved for use in the 1st-line treatment of metastatic non-squamous NSCLC

Pembrolizumab plus pemetrexed-platinum FDA approved for first-line treatment of metastatic non-squamous NSCLC

On 20th of August 2018, the FDA granted regular approval for pembrolizumab (Keytruda®, Merck) plus pemetrexed and a platinum salt in the first-line treatment of patients with metastatic, non-squamous non-small cell lung cancer (NSCLC), not harbouring any EGFR or ALK genomic tumour aberrations.

Pembrolizumab plus pemetrexed-platinum approved for use in the 1st-line treatment of metastatic non-squamous NSCLC

Lenvatinib FDA approved for 1st-line treatment of HCC

On August 16, 2018 the FDA approved lenvatinib (Lenvima®, Eisai) for the first-line treatment of hepatocellular carcinoma (HCC) patients not amenable for curative surgery.

FDA approved lusutrombopag (Mulpleta, Shionogi Inc.) for thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a medical or dental procedure. More Information.  July 31, 2018.

FDA approved mogamulizumab for adult patients with relapsed or refractory CTCL

On the 8th of August 2018, the FDA approved mogamulizumab-kpkc (Poteligeo®, Kyowa Kirin), a CC chemokine receptor type 4 (CCR4) directed monoclonal antibody, for use in adult patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL).

Iobenguane I-131 FDA approved for rare adrenal neuroendocrine tumours

On July 30, 2018, the FDA approved iobenguane I-131 (Azedra®, Progenics Pharmaceuticals), a radioactive therapeutic agent, for the treatment of adult and paediatric patients ≥12 years with iobenguane scan positive, locally advanced or metastatic pheochromocytoma or paraganglioma (PPGL) not amenable to surgery and indicated for systemic therapy.

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.

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

Filgrastim-aafi FDA approved as Filgrastim Biosimilar

On July 20, the FDA approved filgrastim-aafi (Nivestym®, Pfizer), as a biosimilar to filgrastim (Neupogen, Amgen). The label includes all indications of the reference product.

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

 

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 

 

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

 

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

 

FDA approved avatrombopag for thrombocytopenia in CLD patients scheduled for 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

 

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

 

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

 

Adjuvant dabrafenib plus trametinib FDA approved for BRAF V600E or V600K melanoma

The FDA granted regular approval to dabrafenib (Tafinlar®, Novartis) and trametinib (Mekinist®, Novartis) 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. 

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)

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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.

Frontline nivolumab plus ipilimumab approved for intermediate or poor risk advanced RCC

The FDA granted approvals to nivolumab and ipilimumab (Opdivo and Yervoy, Bristol-Myers Squibb) in combination for the treatment of intermediate or poor risk, previously untreated advanced renal cell carcinoma.

Everolimus suspension approved in TSC-associated partial-onset seizures

On April 10, the FDA approved everolimus tablets for oral suspension (Afinitor Disperz®, Novartis) for the adjunctive treatment of adult and pediatric patients ≥2 years with tuberous sclerosis complex (TSC)-associated partial-onset seizures.

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

 

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

 

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

 

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

 

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

 

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

 

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

 

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

 

 

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

 

 

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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.

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Pembrolizumab approved for advanced gastric cancer

On September 22, 2017, the FDA granted accelerated approval to pembrolizumab (KEYTRUDA, Merck) for use in patients with locally advanced or metastatic, gastric or gastroesophageal junction (GEJ) adenocarcinoma positive for PD-L1 as determined by an FDA-approved companion diagnostic (CDx) and progressing after ≥2 prior systemic therapies, including a fluoropyrimidine– and platinum-containing regimen, and, when appropriate, HER2targeted therapy.

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Nivolumab approved for sorafenib-refractory HCC

On September 22, 2017, the Food and Drug Administration granted accelerated approval to nivolumab (OPDIVO, Bristol-Myers Squibb Co.) for the treatment of hepatocellular carcinoma (HCC) in patients who have been previously treated with sorafenib. Approval was based on a 154-patient subgroup of CHECKMATE-040 (NCT 01658878), a multicenter, open-label trial conducted in patients with HCC and […]