New Treatment Options in Oncology: FDA and EMA Drug Approvals in Q2 2022

The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved 25 new oncology agents and new indications during the second quarter of 2022. One of the main highlights was the accelerated approval (FDA) of the tumor agnostic dabrafenib + trametinib (Tafinlar^® + Mekinist^®; Novartis) for treatment of unresectable/metastatic solid tumors with BRAF V600E mutation. This approval is expected to lead to increasingly routine use of BRAF tests in the diagnosis of solid tumors. Also of interest is the approval of trastuzumab deruxtecan (Enhertu^®; Daiichi Sankyo; Astra Zeneca) by both agencies for the treatment of unresectable/metastatic HER2-positive breast cancer. Trastuzumab deruxtecan was previously granted accelerated approval by the FDA in December 2019. Another new treatment for early breast cancer was approved by the FDA–olaparib (Lynparza^®; Astra Zeneca Pharmaceuticals) for patients with BRCA 1/2 mutations, HER2 negative, high-risk, and previously treated with chemotherapy. This is the first approved medicine targeting BRCA mutations in early breast cancer.

Additional highlights from the second quarter of 2022 include:

Three chimeric antigen receptor CAR T-cell therapies were approved for treatment of relapsed/refractory lymphoma: axicabtagene ciloleucel (Yescarta^®; Kite Pharma) by the EMA for follicular lymphoma and by the FDA for large B-cell lymphoma (LBCL); tisagenlecleucel (Kymriah^®; Novartis) FDA accelerated approval for follicular lymphoma; and lisocabtagene maraleucel (Breyanzi^®; Juno Therapeutics) by the FDA for large B-cell lymphoma LBCL
There were several new indications approved for existing therapies:
- Nivolumab + ipilimumab (Opdivo^® + Yervoy^®; Bristol-Myers Squibb) for first-line treatment esophageal squamous cell carcinoma (FDA)
- “Palliative” was removed from the label for leuprolide acetate (Lupron Depot^®; Abbvie Endocrine) (FDA)
- Pembrolizumab (Keytruda^®; Merck) received EMA approvals for patients with triple negative breast cancer and melanoma (adolescents aged 12 or older)
- Azacitadine (Vidaza^®; Celgene) in juvenile myelomonocytic leukemia (FDA)
- Ivosidenib tablets (Tibsovo^®; Servier) in AML
- Selinexor (Nexpovio^®; Karyopharm Europe) is now approved by the EMA for use in earlier lines of therapy (second line or later) for multiple myeloma (previously approval was only for patients who received at least 4 prior therapies)
Two new biologics for neutropenia received approval: pegfilgrastim-PBBK (Fylnetra^®; Kashiv Biosciences) approved by the FDA and lipegfilgrastim (Lonquex^®; Teva; pediatric indication) by the EMA
Two bevacizumab biosimilars were approved: Alymsys^® (Amneal Pharms) by the FDA and Vegzelma^® (Celltrion Healthcare) by the EMA
There was 1 product indication withdrawn in the US by the manufacturer–the ovarian cancer indication for rucaparib (Rubraca^®; Clovis Oncology) after a recent trial failed to show a survival benefit vs chemotherapy for this indication. The other indications were not affected by the withdrawal

The tables below summarize key Q2 oncology approvals:

FDA and EMA Approvals
Generic Name	Brand Name	Tumor Type^a	MOA	Manufacturer	Approval
axicabtagene ciloleucel	Yescarta	Relapsed/refractory LBCL (FDA) Relapsed/refractory follicular lymphoma (EMA)	CAR T-cell therapy	Kite Pharma	4/1/22
Fam-trastuzumab deruxtecan-nxki	Enhertu	Breast Cancer	HER2-directed antibody and topoisomerase inhibitor conjugate	Daiichi Sankyo; Astra Zeneca	5/4/22 (FDA) and 6/27/22 (EMA)

FDA Approvals
Generic Name	Brand Name	Tumor Type^a	MOA	Manufacturer	Approval
Azacitidine	Vidaza	Juvenile myelomonocytic leukemia (new indication)	Nucleoside metabolic inhibitor	Celgene	5/20/22
Bevacizumab-maly (biosimilar)	Alymsys	Colon, lung, glioblastoma, renal cell carcinoma, cervical, ovarian	VEGF inhibitor	Amneal Pharms	4/13/22
Dabrafenib + trametinib	Tafinlar + Mekinist	Solid tumors with BRAF V600E mutation	BRAF inhibitor	Novartis	6/22/22^b
ipilimumab + nivolumab	Yervoy + Opdivo	Esophageal squamous cell carcinoma	CTLA-4 blocking antibody + anti-PD-1	Bristol Myers Squibb	5/27/22
Ivosidenib tablets	Tibsovo	AML	IDH1 inhibitor	Servier	5/25/22
Leuprolide acetate	Lupron Depot	Prostate cancer (“palliative” was removed)	GnRH agonist	Abbvie Endocrine	3/23/22
Lisocabtagene maraleucel	Breyanzi	Large B-cell lymphoma	CAR T-cell therapy	Juno Therapeutics	6/24/22
Nivolumab + fluoropyrimidine- and platinum-based chemotherapy	Opdivo	Esophageal squamous cell carcinoma	PD-1 inhibition	Bristol Myers Squibb	5/27/22
Pegfilgrastim-pbbk (biologic)	Fylnetra	Neutropenia	Leukocyte growth factor	Kashiv Biosciences	5/26/22
Tisagenlecleucel	Kymriah	Relapsed/refractory follicular lymphoma	CAR T-cell therapy	Novartis	5/27/22^b

EMA Approvals
Generic Name	Brand Name	Indication	MOA	Manufacturer	Approval
Asciminib	Scemblix	Ph+ CML chronic phase	Kinase inhibitor		June 2022
Atezolizumab	Tecentriq	Early-stage NSCLC	PD-L1 inhibitor	Roche	April 2022
Bevacizumab (biosimilar)	Vegzelma	Colon, rectum, breast, NSCLC, RCC, epithelial ovarian, fallopian tube or primary peritoneal, cervix	Monoclonal antibody	Celltrion Healthcare Hungary Kft	June 2022
Capmatinib	Tabrecta	Advanced NSCLC	MET inhibitor	Novartis	April 2022
Dasatinib anhydrous	Daruph/Anafezyn or Dasatinib Zentiva	CML and ALL	TKI	Zentiva k.s.	May 22
Lipegfilgrastim	Lonquex	Neutropenia (pediatric)	G-CSF	Teva	June 2022
Melphalan flufenamide (melflufen)	Pepaxti	Multiple myeloma	Inhibits DNA and RNA synthesis	Oncopeptides AB	June 2022
Mosunetuzumab	Lunsumio	Relapsed/refractory follicular lymphoma	Bispecific monoclonal antibody	Roche	June 2022^c
Olaparib	Lynparza	Breast cancer	PARP inhibitor	Astra Zeneca	June 2022
Pembrolizumab	Keytruda	Triple negative breast cancer	PD-1 inhibitor	Merck	April 2022
Pembrolizumab	Keytruda	Melanoma (adolescents aged 12 and older)	PD-1 inhibitor	Merck	May 2022
Selinexor	Nexpovio	Multiple myeloma (second line or later)	Selective inhibitor of nuclear export (SINE)	Karyopharm Europe	May 2022
Selpercatinib	Retevmo	NSCLC (label update, now first -line)	RET inhibitor	Eli Lilly	April 2022
ALL, acute lymphocytic leukemia; AML, acute myeloid leukemia; BRAF, B-Raf proto-oncogene serine/threonine kinase; BRCA, breast cancer susceptibility gene; CAR T, chimeric antigen receptor T cells; CML, chronic myeloid leukemia; CTLA-4, cytotoxic T-lymphocyte antigen-4; DNA, deoxyribonucleic acid; EMA, European Medicines Agency; FDA, US Food and Drug Administration; G-CSF, granulocyte colony-stimulating factor; GnRH, gonadotropin-releasing hormone; HER2, human epidermal growth factor receptor; IDH1, isocitrate dehydrogenase-1; LBCL, large B-cell lymphoma; mAb, monoclonal antibody; MET, mesenchymal-epithelial transition; MOA, mechanism of action; NSCLC, non-small cell lung cancer; PARP(ADP-ribose) polymerase; PD-1, programmed cell death protein 1; PD-L1, PD-1 ligand 1; Ph, Philadelphia chromosome; RCC, renal cell carcinoma; RET, rearranged during transfection; RNA, ribonucleic acid; R/R, relapsed/refractory; TKI, tyrosine kinase inhibitor; VEGF, vascular endothelial growth factor. ^aPlease see each product’s Prescribing Information and Summary of Product Characteristics for full indications and labelling information. ^bAccelerated approval. ^cConditional marketing approval.

Sources:

https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=reportsSearch.process

https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications?t=308050

https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-19-22-april-2022

https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-16-19-may-2022

https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-20-23-june-2022

www.novartis.com/news/media-releases/novartis-tafinlar-mekinist-receives-fda-approval-first-tumor-agnostic-indication-braf-v600e-solid-tumors

www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/lynparza-recommended-in-eu-for-early-breast-cancer.html

www.astrazeneca.com/media-centre/press-releases/2022/enhertu-recommended-for-breast-cancer-eu-approval.html

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News & Resources

New Treatment Options in Oncology: FDA and EMA Drug Approvals in Q2 2022

FDA and EMA Approvals

Generic Name

Brand Name

Tumor Typea

MOA

Manufacturer

Approval

axicabtagene ciloleucel

Yescarta

Relapsed/refractory LBCL (FDA) Relapsed/refractory follicular lymphoma (EMA)

CAR T-cell therapy

Kite Pharma

4/1/22

Fam-trastuzumab deruxtecan-nxki

Enhertu

Breast Cancer

HER2-directed antibody and topoisomerase inhibitor conjugate

Daiichi Sankyo; Astra Zeneca

5/4/22 (FDA) and 6/27/22 (EMA)

FDA Approvals

Generic Name

Brand Name

Tumor Typea

MOA

Manufacturer

Approval

Azacitidine

Vidaza

Juvenile myelomonocytic leukemia (new indication)

Nucleoside metabolic inhibitor

Celgene

5/20/22

Bevacizumab-maly (biosimilar)

Alymsys

Colon, lung, glioblastoma, renal cell carcinoma, cervical, ovarian

VEGF inhibitor

Amneal Pharms

4/13/22

Dabrafenib + trametinib

Tafinlar + Mekinist

Solid tumors with BRAF V600E mutation

BRAF inhibitor

Novartis

6/22/22b

ipilimumab + nivolumab

Yervoy + Opdivo

Esophageal squamous cell carcinoma

CTLA-4 blocking antibody + anti-PD-1

Bristol Myers Squibb

5/27/22

Ivosidenib tablets

Tibsovo

AML

IDH1 inhibitor

Servier

5/25/22

Leuprolide acetate

Lupron Depot

Prostate cancer (“palliative” was removed)

GnRH agonist

Abbvie Endocrine

3/23/22

Lisocabtagene maraleucel

Breyanzi

Large B-cell lymphoma

CAR T-cell therapy

Juno Therapeutics

6/24/22

Nivolumab + fluoropyrimidine- and platinum-based chemotherapy

Opdivo

Esophageal squamous cell carcinoma

PD-1 inhibition

Bristol Myers Squibb

5/27/22

Pegfilgrastim-pbbk (biologic)

Fylnetra

Neutropenia

Leukocyte growth factor

Tumor Type^a

Relapsed/refractory LBCL (FDA)
Relapsed/refractory follicular lymphoma (EMA)

Daiichi Sankyo;
Astra Zeneca

Tumor Type^a

6/22/22^b

5/27/22^b

June 2022^c