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Volume 31, Issue 3
Biologic new molecular entities (NMEs) accounted for 26% of total NME approvals in 2017.
FDA had another banner year for new molecular entity (NME) drug approvals with 46 NMEs approved by the agency’s Center for Drug Evaluation and Research (CDER) in 2017 (1), compared to 22 NME approvals in 2016 (2) and surpassing the 45 NMEs approved in 2015 (3). Biologics continued to be strongly represented, accounting for 26% of the total NMEs approved in 2017, a consistent level of biologics license application (BLA) NMEs approved in recent years. By number, CDER approved nearly double the number of biologic NMEs, going from seven in 2016 to 12 in 2017 (see Table I) (1,2).
Pharma majors led the approvals, accounting for eight of the 12 biologic NMEs approved by FDA in 2017 (see Table II) (1). These companies included AstraZeneca, Roche, Johnson & Johnson (J&J), Merck KGaA, and Sanofi. The other four biologic NMEs came from BioMarin Pharmaceutical, headquartered in Novato, CA; Regeneron Pharmaceuticals, headquartered in Tarrytown, NY; Valeant Pharmaceuticals International, based in Laval, Quebec, Canada; and Ultragenyx Pharmaceutical, also headquartered in Novato, CA.
AstraZeneca and Roche each received two biologic NME approvals in 2017. AstraZeneca received approval for Fasenra (benralizumab), for treating severe asthma in patients with an eosinophilic phenotype, and Imfinzi (durvalumab), for treating locally advanced or metastatic urothelial carcinoma.
Roche’s biologic NME approvals included Hemlibra (emicizumab), a hemophila A therapy, and Ocrevus (ocrelizumab), for treating relapsing and progressive forms of multiple sclerosis (MS).
The other pharma-major biologic NME approvals included J&J’s Tremfya (guselkumab), for treating moderate-to-severe plaque psoriasis; Merck KGaA’s Bavencio (avelumab), for treating metastatic Merkel cell carcinoma; and Sanofi’s Kevzara (sarilumab), for treating rheumatoid arthritis.
The biologic NME approvals garnered by smaller pharma companies included candidates from BioMarin, Regeneron, Valeant, and Ultragenyx.
BioMarin’s Brineura (cerliponase alfa) is the first enzyme replacement therapy for treating a specific form of Batten disease (4). Regeneron’s Dupixent (dupilumab), which the company is co-developing with Sanofi under a global collaboration agreement (5), is indicated for treating moderate-to-severe eczema, and Valeant’s Siliq (brodalumab), in indicated for treating moderate-to-severe plaque psoriasis. Ultragenyx’s Mepsevii (vestronidase alfa-vjbk) is indicated for treating mucopolysaccharidosis type VII (MPS VII) (Sly syndrome), a rare genetic enzyme disorder (6).
Oncology was a specific therapeutic focus for some of the biologic NMEs approved in 2017. Other major therapeutic categories that were targeted for biologic development included respiratory and autoimmune and inflammatory diseases.
Three of the 12 biologic approvals target cancer treatment: Bavencio treats skin cancer (Merkel cell carcinoma); Besponsa treats blood cancer (relapsed or refractory acute lymphoblastic leukemia); and Imfinzi treats urothelial cancer (locally advanced or metastatic urothelial carcinoma).
Four biologic NMEs targeted the autoimmune and inflammatory disease category: Dupixent treats eczema; Ocrevus treats MS; and Siliq and Tremfya both treat plaque psoriasis. The respiratory therapeutic category was represented by Fasenra, which targets the treatment of severe asthma.
Another trend in biologic drug development has centered around the treatment of genetically specific populations. For instance, the asthma therapy, Fasenra, is specifically indicated for severe asthma in patients that have an eosinophilic phenotype. Eosinophils are a type of white blood cell that are native to the body’s immune system, but elevated levels of these cells impact airway inflammation and airway hyper-responsiveness, which results in increased asthma severity and symptoms, decreased lung function, and increased risk of exacerbations, according to AstraZeneca (7). Elevated levels of eosinophils are seen in about half of severe asthma patients, as stated by the company.
Brineura and Mepsevii are also biologics that were approved to treat genetically specific populations. Brineura is specifically indicated for treating late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency. CLN2 disease is part of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), collectively referred to as Batten disease. CLN2 is a rare inherited disorder that primarily affects the nervous system (4).
Mepsevii is also specifically indicated to treat a rare genetic condition, MPS VII. MPS VII is an inherited genetic condition that typically involves progressive skeletal abnormalities due to a lysosomal storage disorder caused by deficiency of the enzyme beta-glucuronidase. The absence of this enzyme causes an abnormal buildup of toxic materials in the body’s cells. Mepsevii is an enzyme replacement therapy that works by replacing the missing beta-glucuronidase enzyme (6).
Several of the 2017 biologic NMEs have blockbuster potential: Ocrevus, Dupixent, Imfinzi, and Bavencia. Sales of these products are forecast to surpass the $1-billion revenue mark by 2021, according to market data by Thomson Reuters (8).
Ocrevus, Roche’s MS biologic, is forecast to be the top-ranking blockbuster. Sales for the drug are expected to reach more than $3.3 billion in 2021. Ocrevus was expected to “materially disrupt” the MS market with its launch in 2017. It is a first-in-class anti-CD20 antibody that is said to be the first to have proven itself effective in primary progressive MS (8). Dupixent is forecast to reach more than $2.8 billion; Imfinizi is forecast to reach more than $2 billion; and Bavencia is forecast to reach more than $1.2 billion, all in 2021 (8).
The pipeline for upcoming biologics shows robust development. Among the pharma majors that have a strong pipeline in biologics is Roche. The company has five biologic NMEs in Phase III development: etrolizumab for ulcerative colitis; gantenerumab and crenezumab for Alzheimer’s disease; satralizumab for neuromyelitis optica; and RG6206, an anti-myostatin adnectin IgG1-Fc fusion protein Duchenne muscular dystrophy. The company plans to file for regulatory approval in 2020 or beyond, except with satralizumab, which is expected to be filed in 2019 (9).
1. FDA, “Novel Drug Approvals for 2017,” accessed Feb. 20, 2018.
2. FDA, “Novel Drug Approvals for 2016,” accessed Feb. 20, 2018.
3. FDA, “Novel Drug Approvals for 2015,” accessed Feb. 20, 2018.
4. FDA, “FDA Approves First Treatment for a Form of Batten Disease,” Press Release, Apr. 27, 2017.
5. Regeneron Pharmaceuticals, “Regeneron and Sanofi Announce Approval of Dupixent (dupilimab) to Treat Adult Patients with Moderate-to-Severe Atopic Dermatitis in the European Union,” Press Release, Sep. 28, 2017.
6. FDA, “FDA Approves Treatment for Rare Genetic Enzyme Disorder,” Press Release, Nov. 15, 2017.
7. AstraZeneca, “Fasenra (benralizumab) Receives US FDA Approval for Severe Eosinophilic Asthma,” Press Release, Nov. 14, 2017.
8. Thomson Reuters (Cortellis Competitive Intelligence), “Drugs to Watch 2017,” accessed Feb. 23, 2018.
9. Roche, “Product Development Portfolio,” accessed Feb. 23, 2018.
Volume 31, Number 3
When referring to this article, please cite as F. Mirasol, “Biologic NMEs Maintain Strong Presence in 2017 Drug Approvals,” BioPharm International 31 (3) 2018.