2015 FDA New Drug Approvals – 13 Biologics Receive Approval

The FDA just released its report titled “Novel New Drugs 2015 Summary,” in which they discuss 2015 FDA new drug approvals. In 2015, the FDA’s Center for Drug Evaluation and Research (CDER) approved 45 novel new medicines, called new molecular entities or NMEs. The number of approvals in 2015 was up from 2014 with 41 approvals and up from 2013 with 27 approvals. In fact, in looking at approvals over the past ten years, 2015 had the most approvals overall.

Other highlights from the report include:

  • 16 of the 45 NMEs were considered “first in class” which means that they utilize a novel or unique mechanism of action over existing therapies.
  • 21 of the 45 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 45 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.” (14 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.” (10 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. (24 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.” (6 NMEs had this designation)

  • 39 of the 45 were approved on the first cycle, which means that they were approved without the need for additional information that would delay approval.
  • 29 of the 45 were approved first in the United States before any other country.

Biologics Approved in 2015

Included in the report were 13 biotech drugs or biologics that were approved by CDER. This number was up from last year where there were 11 approved. In addition, 7 of the 13 approved biologics participated in at least one expedited pathway designation. Seven of the thirteen biologics identified their expression system as CHO Cells. Please see Table 1 below for more details.

Table 1: Biologics approved by FDA (CDER) in 2015, in order of approval date (latest to earliest)
Drug NameActive IngredientsFDA Expedited PathwayExpression SystemCompanyIndications
Kanumasebelipase alfaFast Track, Breakthrough, Priority ReviewProduced using recombinant DNA technology in the egg white of eggs laid by genetically engineered chickensAlexion PharmaceuticalsIndicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.
EmplicitielotuzumabBreakthrough, Priority ReviewProduced in NS0 cells by recombinant DNA technology.Bristol-Myers SquibbA SLAMF7-directed immunostimulatory antibody indicated in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received one to three prior therapies.
PortrazzanecitumumabFast TrackProduced in genetically engineered mammalian NS0 cells.Eli Lilly and CompanyAn epidermal growth factor receptor (EGFR) antagonist indicated, in combination with gemcitabine and cisplatin, for first-line treatment of patients with metastatic squamous non-small cell lung cancer.
DarzalexdaratumumabFast Track, Breakthrough, Priority Review, Accelerated ApprovalProduced in a mammalian cell line (Chinese Hamster Ovary [CHO]) using recombinant DNA technology.Janssen BiotechA human CD38-directed monoclonal antibody indicated for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.
Nucalamepolizumabn/aProduced by recombinant DNA technology in Chinese hamster ovary cells.GSKAn interleukin-5 antagonist monoclonal antibody (IgG1 kappa) indicated for add-on maintenance treatment of patients with severe asthma aged 12 years and older, and with an eosinophilic phenotype.
Strensiqasfotase alfaFast Track, Breakthrough, Priority ReviewProduced by recombinant DNA technology in a Chinese hamster ovary cell line.Alexion PharmaceuticalsA tissue nonspecific alkaline phosphatase indicated for the treatment of patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP).
PraxbindidarucizumabBreakthrough, Priority Review, Accelerated ApprovalProduced in a well characterized recombinant (mammalian) CHO cell line and is purified using standard technology.Boehringer Ingelheim PharmaceuticalsA humanized monoclonal antibody fragment (Fab) indicated in patients treated with Pradaxa® when reversal of the anticoagulant effects of dabigatran is needed: For emergency surgery/urgent procedures or in life-threatening or uncontrolled bleeding
Tresibainsulin degludec injectionn/aProduced by a process that includes expression of recombinant DNA in Saccharomyces cerevisiae followed by chemical modification.Novo NordiskA long-acting human insulin analog indicated to improve glycemic control in adults with diabetes mellitus
Repathaevolocumabn/aProduced in genetically engineered mammalian (Chinese hamster ovary) cells.Amgen, Inc.A human monoclonal immunoglobulin G2 (IgG2) directed against human proprotein convertase subtilisin kexin 9 (PCSK9). To treat certain patients with high cholesterol
Praluentalirocumabn/aProduced by recombinant DNA technology in Chinese Hamster Ovary cell suspension culture.Sanofi and Regeneron PharmaceuticalsA human monoclonal antibody (IgG1 isotype) that targets proprotein convertase subtilisin kexin type 9 (PCSK9). Alirocumab is a PCSK9 inhibitor to treat certain patients with high cholesterol.
UnituxindinutuximabPriority ReviewA chimeric monoclonal antibody composed of murine variable heavy and light chain regions and the human constant region for the heavy chain IgG1 and light chain kappa. Unituxin binds to the glycolipid disialoganglioside (GD2). It is produced in the murine myeloma cell line, SP2/0.United TherapeuticsA GD2-binding monoclonal antibody indicated, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2), and 13-cis-retinoic acid (RA), for the treatment of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to prior first-line multiagent, multimodality therapy.
Natparaparathyroid horomonen/aThe active ingredient in NATPARA, parathyroid hormone, is produced by recombinant DNA technology using a modified strain of Escherichia coli.ShireA parathyroid hormone indicated as an adjunct to calcium and vitamin D to control hypocalcemia in patients with hypoparathyroidism.
Cosentyxsecukinumabn/aExpressed in a recombinant Chinese Hamster Ovary (CHO) cell line.Novartis PharmaceuticalsA human interleukin-17A antagonist indicated for the treatment of moderate to severe plaque psoriasis in adult patients who are candidates for systemic therapy or phototherapy. Also for adults with active psoriatic arthritis (PsA) and adults with active ankylosing spondylitis (AS).
Source: FDA and Prescribing Information

Biologics approved by CBER

Eight biologic drugs produced using biotechnology were approved in 2015 by FDA’s Center for Biologics Evaluation and Research (CBER) and thereby not included in the above referenced report.

Vonvendi manufactured by Baxalta is a purified recombinant von Willebrand factor expressed in Chinese Hamster Ovary (CHO) cells to treat bleeding episodes in adults diagnosed with von Willebrand disease.

Fluad the first seasonal vaccine containing an adjuvant approved by the FDA. It is a trivalent, inactivated influenza vaccine prepared from virus propagated in hen’s eggs

Adynovate manufactured by Baxalta is a recombinant full length human coagulation factor VIII covalently conjugated with one or more molecules of polyethylene glycol. It is produced using recombinant DNA technology from the CHO cell line. It is indicated for adolescent (12 to less than 18 years) and adult (greater than or equal to 18 years) patients with hemophilia A (congenital factor VIII deficiency) for: control and prevention of bleeding episodes and routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

Imlygic manufactured by Amgen is a live attenuated HSV-1 that has been genetically modified to express huGM-CSF. The parental virus for IMLYGIC was a primary isolate, which was subsequently altered using recombinant methods to result in gene deletions and insertions. IMLYGIC is a genetically modified oncolytic viral therapy indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery.

Nuwiq manufactured by Octapharma is a is a recombinant antihemophilic factor [blood coagulation factor VIII (Factor VIII)] produced by recombinant DNA technology in genetically modified human embryonic kidney (HEK) 293F cells with no animal or human derived materials added during the manufacturing process or to the final product. It is indicated in adults and children with Hemophilia A for on demand treatment and control of bleeding episodes, perioperative management of bleeding and routine prophylaxis to reduce the frequency of bleeding episodes.

Ixinity manufactured by Cangene (subsidiary of Emergent Biosolutions) is a coagulation factor IX (recombinant) that is secreted by a genetically engineered mammalian cell line derived from Chinese Hamster Ovary (CHO) cells. It is indicated in adults and children ≥ years of age with hemophilia B for control and prevention of bleeding episodes, and for perioperative management.

Quadracel manufactured by Sanofi Pasteur, is a vaccine indicated for active immunization against diphtheria, tetanus, pertussis and poliomyelitis. Poliovirus Type 1, Type 2, and Type 3 are each grown in separate cultures of MRC-5 cells, a line of normal human diploid cells, by the microcarrier method.

Bexsero manufactured by GSK, is a vaccine indicated for active immunization to prevent invasive disease caused by Neisseria meningitidis sero group B.

For previous year’s coverage, please see:

2016 FDA CDER Novel Biologics Approval

2014 FDA New Drug Approvals – 11 Biologics Receive Approval

2013 FDA New Drug Approvals

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