Broad Scope of Unique Targets and Malignancies for New FDA and EMA Drug Approvals as 2023 Concluded

In the fourth quarter of 2023, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved more than 20 new oncology agents and 16 new indications for previously approved agents. The approvals were for approximately 24 solid tumor types and 8 hematologic malignancies. There were 2 diagnostic agents, 5 biologics, 4 programmed cell death protein 1 inhibitors, and drugs with several other targets approved. One treatment was approved by both the FDA and the EMA: pembrolizumab (Keytruda^®; Merck & Co) for treatment of gastric or gastroesophageal junction (GEJ) adenocarcinoma.

Additional highlights from the fourth quarter of 2023

First FDA approval of a therapy to treat progressing desmoid tumors: nirogacestat (Ogsiveo™; SpringWorks Therapeutics)
First FDA approval of a therapy intended to reduce the risk of relapse in pediatric patients with high-risk neuroblastoma: eflornithine (Iwilfin™; USWM)
First FDA approval that includes patients with ROS proto-oncogene 1 (ROS1)-positive non-small cell lung cancer (NSCLC) who have previously received a ROS1 tyrosine kinase inhibitor (TKI), in addition to patients who are TKI naive
Pembrolizumab (Keytruda; Merck & Co) had 7 new approvals; 5 by the FDA, 1 by the EMA, and 1 by both agencies
- There was also an update to the gastric or GEJ adenocarcinoma indication for pembrolizumab (Keytruda; Merck & Co) by the FDA that restricts use to patients whose tumors express programmed cell death ligand 1 (PD-L1) (combined positive score ³1) as determined by an FDA-approved test
A combination of 2 products that previously received accelerated approval gained full approval by the FDA: enfortumab vedotin (Padcev^®; Astellas) and pembrolizumab (Keytruda; Merck & Co), for urothelial carcinoma
1 new supportive care biologic agent was approved by the FDA: efbemalenograstim alfa-vuxw (Ryzneuta^®; Evive Biotech), for prevention of chemotherapy-induced neutropenia
4 companion diagnostics were approved by the FDA
- FoundationOne^® companion diagnostic for patients with NSCLC who may be eligible for combination treatment with binimetinib (Mektovi^®; Array Biopharma) and encorafenib (Braftovi^®; Array Biopharma)
- FoundationOne companion diagnostic for patients with hormone receptor-positive, human epidermal growth factor receptor 2-negative breast cancer who may be eligible for treatment with capivasertib (Truqap™; AstraZeneca)
- Abbott RealTime IDH1 assay to select patients appropriate for ivosidenib (Tibsovo^®; Servier)
- Agilent PD-L1 IHC 22C3 pharmDx to select patients with gastric or GEJ adenocarcinoma appropriate for treatment with pembrolizumab (Keytruda; Merck & Co)
2 formulations of gadopiclenol, an agent for contrast-enhanced magnetic resonance imaging, received positive opinion by the EMA: Elucirem™(Guerbet) and Vueway™(Bracco Imaging), to detect lesions in the central nervous system and other body regions (eg, breast, liver, prostate)
Under its Project Orbis initiative, the FDA collaborated with other health agencies to grant 7 approvals. Since its launch in 2019, there has been continued growth in the number of applications submitted to Project Orbis; EMA announced plans to join the multicountry review consortium as an observer
6 FDA approvals used the Real-Time Oncology Review (RTOR) pilot program, which streamlined data submission prior to the filing of the entire clinical application: nivolumab (Opdivo^®; Bristol Myers Squibb), eflornithine (Iwilfin
Re-examination of adagrasib (Krazati^®; Mirati Therapeutics) by the EMA resulted in a conditional marketing authorization
Non-renewal of the conditional marketing authorization for belantamab mafodotin (Blenrep; GSK Ireland) in multiple myeloma was confirmed by the EMA

The tables below summarize key Q4 2023 oncology approvals.

FDA and EMA Approvals
Generic Name	Brand Name	Tumor Type^a	MOA	Manufacturer	Approval
Pembrolizumab^b	Keytruda^®	Gastric or GEJ adenocarcinoma	PD-1 inhibitor	Merck & Co	811/16/23 (FDA) and October 2023 (EMA)^e

FDA Approvals
Generic Name	Brand Name	Tumor Type^a	MOA	Manufacturer	Approval
Avelumab^b	Bavencio	Merkel cell carcinoma	PD-L1 inhibitor	EMD Serono	9/6/23
Bosutinib monohydrate	Bosulif	Pediatric Ph+ CML	Tyrosine kinase inhibitor	PF Prism CV	9/26/23
Dabrafenib	Tafinlar	Tumor agnostic	B-Raf inhibitor	Novartis	8/31/23
Dostarlimab-gxly^b	Jemperli	dMMR endometrial cancer	PD-1 inhibitor	GlaxoSmithKline	7/31/23
Elranatamab-bcmm^b	Elrexfio	Multiple myeloma	Bispecific antibody	Pfizer	8/14/23^c
Fosaprepitant	Focinvez	Chemotherapy-induced nausea and vomiting	Antiemetic	Spes Pharmaceuticals	8/22/23
Luspatercept	Reblozyl	Anemia in patients with MDS	Recombinant fusion protein	Bristol Myers Squibb	8/28/23
Melphalan	Hepzato Kit	Uveal melanoma with unresectable hepatic metastases	Alkylating agent	Delcath Systems	8/14/23
Momelotinib	Ojjaara	Myelofibrosis and anemia	JAK1/JAK2 and activin A receptor type 1 inhibitor	GlaxoSmithKline	9/15/23
Motixafortide	Aphexda™	Stem cell mobilization in patients with multiple myeloma	CXCR4 antagonist	BioLineRx	9/8/23
Niraparib-abiraterone acetate	Akeega	Prostate cancer	PARP inhibitor plus abiraterone acetate	Janssen Biotech	8/11/23
Pralsetinib	Gavreto^®	NSCLC	RET inhibitor	Genentech	8/20/23
Temozolomide	Temodar	Anaplastic astrocytoma	Alkylating agent	Merck	9/14/23
Therascreen PDGFRA RGQ PCR Kit	Therascreen PDGFRA Kit	GI tumors; companion diagnostic for avapritinib	PDGFRA assay	Qiagen	8/7/23
Trametinib	Mekinist	Tumor agnostic, age 1 and older	MEK inhibitor	Novartis	8/31/23
N/A	FoundationOne CDx	Solid tumors	Tissue-based diagnostic	Foundation Medicine	8/14/23

EMA Approvals
Generic Name	Brand Name	Tumor Type^a	MOA	Manufacturer	Approval
Brentuximab vedotin	Adcetris	Stage III Hodgkin lymphoma	Antibody-drug conjugate	Takeda Pharmaceutical	September 2023
Crisantaspase	Enrylaze^®	ALL and lymphoblastic lymphoma	Recombinant Erwinia asparaginase or crisantaspase	Jazz Pharmaceuticals	July 2023^e
Dabrafenib	Finlee^®	Glioma	B-Raf inhibitor	Novartis	September 2023^e
Decitabine- cedazuridine	Inqovi^®	AML	Hypomethylating agent	Otsuka Pharmaceuticals	July 2023
Degarelix acetate (generic)	Degarelix Accord	Prostate cancer	GnRH antagonist	Accord	July 2023
Elacestrant	Orserdu™	ER+, HER2– breast cancer with ESR1 mutation	ER antagonist	Stemline Therapeutics	July 2023^e
Epcoritamab	Tepkinly^®	DLBCL	Bispecific antibody	AbbVie	July 2023^e
Glofitamab	Columvi^®	DLBCL	Bispecific antibody	Roche	July 2023^d
Melphalan flufenamide	Pepaxti	Multiple myeloma	XPO1 inhibitor	Oncopeptides AB	September 2023
Nivolumab	Opdivo	Melanoma	PD-1 inhibitor	Bristol Myers Squibb	July 2023^e
Pembrolizumab	Keytruda	NSCLC (adjuvant therapy)	PD-1 inhibitor	Merck Sharp & Dohme	September 2023
Pembrolizumab	Keytruda	HER2+ gastric or gastroesophageal junction adenocarcinoma	PD-1 inhibitor	Merck Sharp & Dohme	July 2023^e
Piflufolastat (18F)	Pylclari	Prostate cancer diagnosis	Diagnostic solution	Curium	July 2023
Sacituzumab govitecan	Trodelvy	HR+, HER2– metastatic breast cancer	Trop-2–directed ADC	Gilead	July 2023
Tislelizumab	Tevimbra	Esophageal squamous cell carcinoma	PD-1 inhibitor	Novartis	July 2023^e
Trastuzumab (biosimilar)	Herwenda^®	Breast cancer and gastric cancer	Monoclonal antibody	Sandoz GmbH	September 2023^e
Trastuzumab deruxtecan	Enhertu	NSCLC with HER2 mutation	Antibody-drug conjugate	Daiichi Sankyo	September 2023
+, positive; –, negative; AKT, protein kinase B; ALL, acute lymphocytic leukemia; BRAF, v-Raf murine sarcoma viral oncogene homolog B; BTK, Bruton tyrosine kinase; CLL, chronic lymphocytic leukemia; CML, chronic myeloid leukemia; EMA, European Medicines Agency; FDA, US Food and Drug Administration; G-CSF, granulocyte colony-stimulating factor; GEJ, gastroesophageal junction; HER2, human epidermal growth factor receptor 2; HR, hormone receptor; IDH1, isocitrate dehydrogenase 1; JAK, Janus kinase; KRAS, Kirsten rat sarcoma viral proto-oncogene; MDS, myelodysplastic syndrome; MEK, mitogen-activated ERK kinase; MOA, mechanism of action; NSCLC, non-small cell lung cancer; PARP, poly(ADP-ribose) polymerase; PD-1, programmed cell death protein 1; PD-L1, PD-1 ligand 1; PDGFRA, platelet-derived growth factor receptor alpha; Ph, Philadelphia chromosome; RCC, renal cell carcinoma; ROS1, ROS proto-oncogene 1; SLL, small lymphocytic lymphoma; TKI, tyrosine kinase inhibitor; TRK, tropomyosin receptor kinase; VEGF, vascular endothelial growth factor. ^aPlease see each product’s prescribing information and summary of product characteristics for full indications and labeling information. ^bBiologic. ^cAccelerated approval. ^dConditional marketing authorization. ^ePositive opinion.

Sources:

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

https://www.ema.europa.eu/en/committees/chmp/chmp-agendas-minutes-highlights

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

https://www.raps.org/News-and-Articles/News-Articles/2023/11/FDA-notes-continued-growth”-of-Orbis-collaborative

Matthew Gordon

VP, Real-World Evidence

Matthew has more than 25 years of experience in real-world evidence and observational, non-interventional research. He has led studies across the full life cycle—from startup through publication—supporting objectives that range from understanding a disease’s natural history to fulfilling global safety surveillance requirements. Matthew brings deep expertise in orphan disease programs, having overseen more than 25 long-term, global initiatives, as well as in disease and product registries, prospective pharmacoeconomic studies, and systematic literature reviews. Matthew leads the RWE Registries team, responsible for building the business and team. Prior to joining Aptitude Health, he held senior leadership roles at Parexel, Worldwide Clinical Trials, inVentiv Health Clinical, Quintiles Outcome, and ICON Clinical Research. Matthew holds a BA in sociology from Boston University, is a long-standing member of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), and is a frequent speaker at ISPOR, the Center for Business Intelligence, and related industry conferences.

Gerald Stanvitch, PhD

VP, Scientific Content

Cate Browning, PhD

VP, Global Medical Affairs

Erin Zingales Rau

VP, Account Services

Kelly Kocor

VP, People & Culture

Kelly leads both the human resources and talent acquisition teams, ensuring that Aptitude Health attracts, retains, and develops top personnel to drive our continued success. With over 17 years of experience transforming global HR initiatives, Kelly is an expert in harmonizing HR policies and fostering a culture of engagement and partnership. She is committed to partnering with all areas of the business to ensure full regulatory compliance and delivering value-added services to our organization and its people. Kelly is passionate about developing and implementing HR strategies that help support our employees’ professional and personal growth. She is dedicated to fostering a culture that encourages innovation, collaboration, and inclusivity, helping Aptitude Health continue to be a great place to work.

Bart Zygmond

VP, Finance

Bart brings a wealth of experience to the organization, having worked in the life sciences, pharmaceuticals, manufacturing, and service industries. With his expertise in financial reporting, US GAAP, SOX, cash flow modeling, and financial analysis, he plays a crucial role in the company’s financial management and strategy. Prior to joining Aptitude Health as VP, Finance, Bart held several controller positions: at Q2 Solutions, he oversaw the global finance team and financial operations, ensuring the accurate and timely financial reporting of the company. He also held controller positions at Domtar Inc and Veristat.

Eugene Vissers, MD

Senior VP, Global Scientific Content

Eugene is a seasoned medical doctor with over 20 years of international experience in pharma, consultancy, and agency environments. Eugene leads the US team of dedicated experts responsible for developing high-quality medical content. Prior to joining Aptitude Health, Eugene served as medical director at Ipsen and AstraZeneca. With both his medical degree and an MBA, Eugene brings a unique perspective to his role. His clinical background, combined with his business acumen, allows him to develop innovative strategies that drive results. Under Eugene’s leadership, the medical content team delivers scientific information of the highest quality, providing valuable insights to our clients around the world.

Adrian Barfield

VP, US Business Development

Kelley Hernandez

Executive VP, US Business Development

Kelley has over 18 years of experience in the oncology space. Kelley joined Aptitude Health after working with Cardinal Health, where she was part of the Healthcare and Analytics Division, and finished her tenure there with VitalSource™ (GPO division). As the leader of the strategic business development team for the US, Kelley brings a wealth of expertise to the organization. Her experience in the healthcare industry, combined with her ability to identify and capitalize on new business opportunities, is invaluable in driving the company’s growth and success. Kelley’s dedication to building strong relationships with life science partners is a testament to her commitment to delivering exceptional value to the healthcare industry.

Adam Sinensky, MBA

Chief Technology Officer

Adam has over 20 years of experience in the healthcare industry and an MBA in healthcare management. After 10 years as a strategy consultant to life science companies, Adam has spent the last decade as a product and strategy leader focused on bringing technology products to market across the payor, provider, and life sciences segments. By combining his business acumen and experience working directly with software developers, engineers, and data scientists, Adam has successfully led numerous product launches and enhancements from ideation to development and go-to-market initiatives. His product and change management expertise has led organizational shifts from services to technology at companies such as Change Healthcare and Datavant/Ciox. At Aptitude Health, Adam is responsible for growing our portfolio of product offerings by leveraging real-world data and artificial intelligence with our existing solutions and industry-leading Axess Network of healthcare providers. He also oversees our IT and cybersecurity teams.

Stefanie Daniels

Chief Commercial Officer

Stefanie is a seasoned healthcare executive with over 20 years of experience in oncology. She brings a wealth of knowledge and expertise to the organization. Stefanie joined Aptitude Health after spending over a decade as a senior director at Physicians’ Education Resource, an oncology CME vendor. During her tenure, she led and managed teams responsible for grant development/acquisition, program creation/execution, and faculty management. Stefanie’s deep understanding of the oncology industry and her ability to lead teams through complex projects make her a vital part of the organization’s success. Her dedication to providing high-quality solutions to our life science partners is a testament to her commitment to improving cancer patient care.

Jason Cash

Chief Financial Officer

Jason is an accomplished finance professional with over 20 years of experience in the pharmaceutical services industry. Throughout his career, he has demonstrated a keen ability to navigate high-growth organizations, delivering exceptional results. Before joining Aptitude Health, Jason served as the CFO of Veristat International, a global contract research organization. In this role, he led the financial strategy and played a pivotal role in driving the company’s growth and success. Jason’s wealth of experience and expertise in financial management make him an essential member of the leadership team. His strategic thinking and ability to drive results are highly respected within the industry.

Jez Moulding

Chief Executive Officer

Jez is a seasoned leader with over 20 years of experience in general management and regional president roles. He has a proven track record of success in the healthcare industry, having worked in the US, Japan, Australia, Korea, South Africa, France, and the UK for Sanofi, where he supported the launch of 10 new drugs across various therapeutic areas. As chief commercial officer at UDG Healthcare and EVP at Ashfield, Jez demonstrated his expertise in developing and implementing successful business strategies. He joined Aptitude Health from Pharmaspectra, an IQVIA business, where he served as CEO since 2018. Jez’s extensive experience in the pharmaceutical industry and his leadership skills make him an invaluable asset to the organization.

News & Resources

Broad Scope of Unique Targets and Malignancies for New FDA and EMA Drug Approvals as 2023 Concluded

FDA and EMA Approvals

Generic Name

Brand Name

Tumor Typea

MOA

Manufacturer

Approval

Pembrolizumabb

Keytruda®

Gastric or GEJ adenocarcinoma

PD-1 inhibitor

Merck & Co

811/16/23 (FDA) and October 2023 (EMA)e

FDA Approvals

Generic Name

Brand Name

Tumor Typea

MOA

Manufacturer

Approval

Avelumabb

Bavencio

Merkel cell carcinoma

PD-L1 inhibitor

EMD Serono

9/6/23

Bosutinib monohydrate

Bosulif

Pediatric Ph+ CML

Tyrosine kinase inhibitor

PF Prism CV

9/26/23

Dabrafenib

Tafinlar

Tumor agnostic

B-Raf inhibitor

Novartis

8/31/23

Dostarlimab-gxlyb

Jemperli

dMMR endometrial cancer

PD-1 inhibitor

GlaxoSmithKline

7/31/23

Elranatamab-bcmmb

Elrexfio

Multiple myeloma

Bispecific antibody

Pfizer

8/14/23c

Fosaprepitant

Focinvez

Chemotherapy-induced nausea and vomiting

Antiemetic

Spes Pharmaceuticals

8/22/23

Luspatercept

Reblozyl

Anemia in patients with MDS

Recombinant fusion protein

Bristol Myers Squibb

8/28/23

Melphalan

Hepzato Kit

Uveal melanoma with unresectable hepatic metastases

Alkylating agent

Delcath Systems

8/14/23

Momelotinib

Ojjaara

Myelofibrosis and anemia

JAK1/JAK2 and activin A receptor type 1 inhibitor

GlaxoSmithKline

9/15/23

Motixafortide

Aphexda™

Stem cell mobilization in patients with multiple myeloma

CXCR4 antagonist

Tumor Type^a

Pembrolizumab^b

Keytruda^®

811/16/23 (FDA) and October 2023 (EMA)^e

Tumor Type^a

Avelumab^b

Dostarlimab-gxly^b

Elranatamab-bcmm^b

8/14/23^c

Gavreto^®

Tumor Type^a

Enrylaze^®

July 2023^e

Finlee^®

September 2023^e

Inqovi^®