2014 FDA New Drug Approvals – 11 Biologics Receive Approval

Last month, the FDA released its report titled “Novel New Drugs 2014 Summary,” in which they discuss approvals that occurred in calendar year 2014. In 2014, the FDA’s Center for Drug Evaluation and Research (CDER) approved 41 novel new medicines, called new molecular entities or NMEs. The number of approvals in 2014 was up from 2013 with 27 approvals and up from 2012 with 39 approvals. In fact, in looking at approvals over the past ten years, 2014 had the most approvals overall.

Other highlights from the report include:

  • 17 of the 41 NMEs were considered “first in class” which means that they utilize a novel or unique mechanism of action over existing therapies.
  • 17 of the 41 NMEs were approved to treat orphan diseases. Orphan diseases are considered rare diseases that affect 200,000 or fewer Americans. This is more approvals for orphan drugs than in any previous year.
  • 27 of the 41 NMEs were designated in one or more expedited pathway categories – Fast Track, Breakthrough, Priority Review, and Accelerated Approval
    • Fast Track designation is identified by FDA as drugs with the potential to address unmet medical needs. “Fast Track speeds new drug development and review, for instance, by increasing the level of communication FDA allocates to developers and by enabling developers to use a “rolling review” process such that CDER can review portions of an application ahead of the submission of the full application.” (15 NMEs had this designation)
    • Breakthrough designation is identified by FDA as drugs with preliminary clinical evidence that shows the potential of substantial improvement over at least one clinically significant endpoint compared with current therapy. “A breakthrough therapy designation conveys all of the fast track program features as well as more intensive FDA guidance on an efficient drug development program.” (9 NMEs had this designation)
    • Priority Review is determined by FDA that the drug has the potential to provide a significant advance on medical care. With priority review, FDA sets a target to review the drug within six months instead of the standard that is 10 months. (25 NMEs had this designation)
    • Accelerated Approval allows early approval of a drug for a serious or life-threatening illness that offers benefits over current treatments. “This approval is based on a “surrogate endpoint” (e.g., a laboratory measure) or other clinical measure that FDA considers reasonable likely to predict clinical benefit. After this approval, the drug must undergo additional testing to confirm that benefit; this speeds the availability of the drug.” (8 NMEs had this designation)
  • 32 of the 41 were approved on the first cycle, which means that they were approved without the need for additional information that would delay approval.
  • 26 of the 41 were approved first in the United States before any other country.

Biologics Approved in 2014Included in the report were 11 biotech drugs or biologics that were approved by CDER. This number was up from last year where there were just 2 approved. In addition, 9 of the 11 approved biologics participated in at least one expedited pathway designation. Five of the eight biologics identified their expression system as CHO Cells. Please see Table 1 below for more details.

Table 1: Biologics approved by FDA (CDER) in 2014, in order of approval date
Drug NameActive IngredientsFDA Expedited PathwayExpression SystemCompanyIndications
Vimizimelosulfase alfaFast Track, Priority ReviewPurified elosulfase alfa produced by recombinant DNA technology in a Chinese hamster ovary cell line.Biomarin Pharmaceutical Inc.Indicated as a treatment for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome)
Myaleptmetreleptin for injectionFast Track, Priority ReviewMetreleptin (recombinant methionyl human leptin) is produced in E. coli and differs from native human leptin by the addition of a methionine residue at its amino terminus.Aegerion PharmaceuticalsIndicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.
Tanzeumalbiglutiden/aTANZEUM is produced by a strain of Saccharomyces cerevisiae modified to express the therapeutic protein.GlaxoSmithKlineIndicated as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus
CyramzaramucirumabFast Track, Priority ReviewCYRAMZA is produced in genetically engineered mammalian NS0 cellsEli Lilly and CompanyFor treatment in advanced stomach cancer or gastroesophageal junction carcinoma
SylvantsiltuximabPriority ReviewSYLVANT (siltuximab) is a human-mouse chimeric monoclonal antibody produced by Chinese hamster ovary cellsJanssen BiotechFor the treatment of patients with multicentric Castleman’s disease (MCD) who are human immunodeficiency virus (HIV) negative and human herpesvirus-8 (HHV-8) negative.
EntyviovedolizumabFast Track, Priority ReviewENTYVIO (vedolizumab) is a humanized IgG1 monoclonal antibody produced in Chinese hamster ovary cellsTakeda PharmaceuticalsFor the treatment of adults with moderate to severe ulcerative colitis and adults with moderate to severe Crohn’s disease
Plegridypeginterferon beta-1aThe interferon beta-1a portion of PLEGRIDY is produced as a glycosylated protein using genetically-engineered Chinese hamster ovary cells into which the human interferon beta gene has been introducedBiogen IdecFor the treatment of patients with relapsing forms of multiple sclerosis
KeytrudapembrolizumabBreakthrough, Priority Review, Accelerated ApprovalNot listedMerck & Co., Inc.For treatment of advanced or unresectable melanoma no longer responding to other drugs
Trulicitydulaglutiden/aNot listedEli Lilly and CompanyIndicated as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus.
BlincytoblinatumomabBreakthrough, Priority Review, Accelerated ApprovalBLINCYTO is produced in Chinese hamster ovary cells.Amgen, Inc.Indicated for the treatment of Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).
OpdivonivolumabFast Track, Breakthrough, Priority Review, Accelerated ApprovalNot listedBristol-Myers SquibbIndicated for the treatment of patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor.
Source: FDA and Prescribing Information

Biologics approved by CBER Seven biologic drugs were approved in 2014 by FDA’s Center for Biologics Evaluation and Research (CBER) and thereby not included in the above referenced report.

Aprolix manufactured by Biogen Idec is Recombinant Coagulation Factor IX Fc Fusion Protein expressed in human embryonic kidney (HEK) cells to treat Hemophilia B.

Eloctate manufactured by Biogen Idec is a B domain deleted recombinant factor VII, FC fusion protein expressed in human embryonic kidney (HEK) cells to treat Hemophilia A.

Ruconest manufactured by Salix Pharmaceuticals is a recombinant C1 esterase inhibitor purified from the milk of transgenic rabbits. It is indicated for the treatment of acute attacks in patients with hereditary angioedema.

Hyqvia manufactured by Baxter Healthcare is an immune globulin with a recombinant human hyaluronidase expressed in Chinese Hamster Ovary (CHO) Cells. It is indicated for the treatment of Primary Immunodeficiency (PI).

Obizur manufactured by Baxter Healthcare is a recombinant analogue of porcine factor VIII (p FVIII) expressed in baby hamster kidney (BHK) cells. It is indicated for the treatment of bleeding episodes in adults with acquired Hemophilia A.

Trumenba manufactured by Wyeth Pharmaceuticals (subsidiary of Pfizer) is a sterile suspension composed of two recombinant lipidated factor H (fHBP) binding protein variants from N. meningitides serogroup B, one from fHBP subfamily A and one from subfamily B. The proteins are produced in E. coli. Trumemba is a Meningococcal Group B vaccine, the first approved for use in the United States.

Gardasil 9 manufactured by GlaxoSmithKline, is a Human Papillomavirus 9-valent vaccine. It is manufactured using recombinant Saccharomyces cerevisiae.

For other year’s coverage, please see:

2016 FDA CDER Novel Biologics Approval

2015 FDA New Drug Approvals – 13 Biologics Receive Approval

2013 FDA New Drug Approvals