Medication Approvals

Medication Approvals

Information about U.S. Food and Drug Administration medication approvals.

Specialty Drug Pipeline 2015

by Ryan Chandanais, M.S., emerging therapeutics analyst, Diplomat Specialty Pharmacy

In 2015, the specialty drug pipeline is expected to continue to produce valuable new treatments for patients across a variety of disease states. The oncology and rare disease pipelines remain particularly strong, with several approvals expected in each area. Chronic hepatitis C (CHC) still has a few promising drugs in development, but the time of greatest innovation in that area may have already peaked. Biosimilar developments will be interesting to follow as well. We’ve seen only the tip of the iceberg regarding biosimilars, with the recent Food and Drug Administration (FDA) approval of the Neupogen® biosimilar, Zarxio™ (filgrastim-sndz). The issue of increasing cost of drugs and payors’ ability to sustain those costs is certainly not going away and will only intensify as additional high-cost drugs become available. The percentage of drugs approved that have earned special FDA designations during their development is expected to remain high as well.

In the remainder of 2015, oncology is forecasted to see approvals for approximately seven or eight new agents over the course of the year, along with some significant expanded indications for a few currently available therapies. Rare disease drugs are estimated to gain a total of about five or six new approvals for diseases, including cystic fibrosis, Duchenne muscular dystrophy, hemophilia, von Willebrand disease, hypophosphatasia and lysosomal lipase deficiency. Immunology is expected to see relatively few approvals. However, additional agents in the interleukin-17 (IL-17) class of drugs, similar to Cosentyx® (secukinumab) such as brodalumab, ixekizumab and guselkumab are expected to have a significant impact on the psoriasis market, but probably not until 2016 or later.

Biosimilars will continue to be a developing area of the pipeline. Zarxio™, the first FDA-approved biosimilar, earned the agency’s stamp of approval in March 2015. However, the product has not yet launched due to legal issues. Legal challenges will be a concern for other biosimilars in the pipeline as well. Other factors, including uncertainty surrounding biosimilar regulations in the U.S., questions of interchangeability with the brand name reference products, and patient and prescriber comfort level, make it difficult to determine the impact these agents will have or when they will launch. Due to the number of highly prescribed biologics with recently expired or soon-to-be expiring patents, biosimilars will be worth watching in 2015 and beyond.

Table 1. Expected FDA Decisions for the Remainder of 2015

Second Quarter 2015

Raltegravir (Isentress®) + Lamivudine (Epivir®) – MERCK

  • HIV (new combination)
  • Possible approval in the second quarter

Sirolimus (Rapamune®) – PFIZER

  • Lymphangioleiomyomatosis (LAM) (expanded indication)
  • June 15, 2015

Drisapersen – PROSENSA

  • Duchenne muscular dystrophy
  • June 30, 2015

Asfotase alfa – ALEXION

  • Hypophosphatasia
  • June 30, 2015

Third Quarter 2015

Lumacaftor – VERTEX

  • Cystic fibrosis
  • July 5, 2015

Gefitinib (Iressa™) – ASTRAZENECA

  • Non-small cell lung cancer (attempting to re-acquire approval)
  • July 2015


  • Hypercholesterolemia
  • July 24, 2015

Trabectedin (Yondelis®) – PHARMAMAR

  • Soft tissue sarcoma
  • Aug. 3, 2015

Cobimetinib – EXELIXIS

  • Melanoma
  • Aug. 11, 2015

Evolocumab – AMGEN

  • Hypercholesterolemia
  • Aug. 27, 2015

Obeticholic acid – INTERCEPT

  • Primary biliary cirrhosis
  • Aug. 31, 2015


  • Eosinophilic asthma
  • Sept. 5, 2015

Sebelipase alfa – SYNAGEVA

  • Lysosomal acid lipase deficiency
  • Sept. 8, 2015

Daclatasvir + Sofosbuvir – BRISTOL-MYERS SQUIBB, GILEAD

  • Hepatitis C, genotype 3
  • Sept. 15, 2015

Filgrastim (Neupogen® biosimilar) – APOTEX

  • Same indications as reference product
  • Mid-to-late 2015

Infliximab (Remicade® biosimilar) – CELLTRION

  • Same indications as reference product
  • Possible approval in the third quarter

Fourth Quarter 2015


  • Hemophilia A
  • Oct. 1, 2015

Pembrolizumab (Keytruda®) – MERCK

  • Non-small cell lung cancer (expanded indication)
  • Oct. 19, 2015 (likely early approval)

Vonicog alfa (BAX 111) – BAXTER

  • Von Willebrand Disease
  • Oct. 22, 2015

Selexipag – ACTELION

  • Pulmonary arterial hypertension
  • Oct. 23, 2015

Viekira Pak™ – ABBVIE

  • Hepatitis C, genotype 4 (expanded indication)
  • Oct. 24, 2015

Talimogene Laherparepvec – AMGEN

  • Melanoma
  • Oct. 27, 2015

Nivolumab (Opdivo®) – BRISTOL-MYERS SQUIBB

  • Melanoma, first-line treatment (expanded indication)
  • Oct. 27, 2015 (likely early approval)

Tofacitinib (Xeljanz®) – PFIZER

  • Psoriasis (expanded indication)
  • Oct. 31, 2015

Epoetin alfa (Epogen®, Procrit® biosimilar) – HOSPIRA

  • Anemia
  • October 2015

Tenofovir alafenamide – GILEAD

  • HIV
  • Nov. 6, 2015

Carfilzomib (Kyprolis®) – AMGEN

  • Multiple myeloma (expanded indication)
  • Nov. 27, 2015


  • Colorectal cancer
  • Dec. 9, 2015 (possible early approval)

Telotristat Etiprate – LEXICON

  • Carcinoid syndrome
  • Late 2015

Adalimumab (Humira®) – ABBVIE

  • Hidradenitis suppurativa (expanded indication)
  • Late 2015

Binimetinib – ARRAY

  • Melanoma
  • Late 2015


Posted in: Medication Approvals
An Overview of the Antimicrobial Zerbaxa

by Paul Brumley, Pharm.D., PGY1 pharmacy resident, St. Joseph Mercy Ann Arbor 

According to the Infectious Diseases Society of America (IDSA), antimicrobial resistance has become an imminent threat in the health care world, and the tangible evidence of this is becoming more apparent every year. Antibiotic-resistant pathogens lead to devastating consequences, including nearly 99,000 deaths per year resulting in $21 billion in health care costs. To help combat the insufficient rate at which new antibiotics are being brought to market, the IDSA implemented the “10 X ‘20 Initiative,” a global movement to develop 10 new agents by the year 2020. One new agent that has made its way to market is Zerbaxa®

On Dec. 19, 2014, Cubist Pharmaceuticals’ new product Zerbaxa® (ceftolozane/ tazobactam) became the latest Food and Drug Administration (FDA)-approved antibiotic in this battle against growing antimicrobial resistance. With its antipseudomonal coverage, ceftolozane now joins the ranks of ceftazidime and cefepime in cephalosporins with this property; the addition of the β-lactamase inhibitor tazobactam was found to increase activity against both gram-positive and gram-negative organisms in vitro. In regard to resistant strains of Pseudomonas aeruginosa, ceftolozane/tazobactam has demonstrated in vitro activity against isolates with up-regulation of efflux pumps, presence of chromosomal AmpC and loss of outer membrane porin. Other resistant bugs, which have been shown to have in vitro susceptibility to ceftolozane/tazobactam, include some strains of β-lactamase-producing E. coli and K. pneumoniae. To what extent this activity holds up in vivo will be an important aspect to monitor over time. Although studies for other indications are under way, Cubist’s product is currently only indicated for complicated urinary tract infections (cUTIs) and complicated intra-abdominal infections (cIAIs). 

For use in cUTI’s, ceftolozane/tazobactam 1,500 mg IV q8h was compared with levofloxacin 750 mg IV q24h in a phase III, multicenter, prospective, randomized, double-blind, double-dummy study. Included patients were required to have two or more symptoms of a UTI (dysuria, fever, suprapubic/flank pain, nausea or vomiting) plus at least one complicating factor (males with documented history of urinary retention, an indwelling catheter, obstructive uropathy, or any urogenital tract functional or anatomical abnormality). After seven days of therapy, ceftolozane/tazobactam was found to be non-inferior to levofloxacin, with composite microbiological and clinical cure rates (the primary outcome) of 76.9 percent and 68.4 percent, respectively. Safety profiles were comparable between the two groups, with treatment-related adverse events occurring in 10.3 percent of the ceftolozane/tazobactam group and 12 percent of the levofloxacin group. Studies have shown ceftolozane/tazobactam to be effective against cUTI’s caused by the following bugs: E. coli, K. pneumoniae, P. mirabilis and P. aeruginosa

With a similar non-inferiority study design for patients with cIAIs, ceftolozane/tazobactam 1,500 mg IV q8h plus metronidazole 500 mg IV q8h was compared with meropenem 1,000 mg IV q8h. For study inclusion, patients had to require surgical intervention (e.g., laparotomy, laparoscopic surgery or percutaneous draining of an abscess) within 24 hours of the first dose of study drug. After four to 14 days of therapy, ceftolozane/tazobactam plus metronidazole achieved non-inferiority to meropenem with clinical cure rates (the primary outcome) of 83 percent and 87.3 percent, respectively. Adverse event rates for ceftolozane/tazobactam were comparable with the cUTI study, with the most common observed adverse effects being nausea (7.9 percent) and diarrhea (6.2 percent). Studies have shown ceftolozane/tazobactam plus metronidazole to be effective against cIAI’s caused by the following microorganisms: E. cloacae, E. coli, K. oxytoca, K pneumoniae, P mirabilis, P. aeruginosa, B. fragilis, S. anginosus, S. constellatus and S. salivarius

With its recent FDA approval, Zerbaxa® is the fifth new antibiotic available since the IDSA’s introduction of the 10 X ‘20 Initiative. In a December 2014 progress statement, the organization indicated that it “applauds Cubist and other public and private industry enterprises dedicated to helping combat the rise of antibiotic-resistant bacteria – a true public health crisis – for their efforts to mitigate the sharp decline in the development of new antibiotics.” Exactly what the new product’s place in therapy will be is yet to be determined, but the incorporation of a new weapon in the arsenal against resistant bacteria is an exciting advancement. 

References available upon request from MPA office.

Posted in: Medication Approvals
FDA Approves First Biosimilar in the U.S.
On March 6, 2015, the U.S. Food and Drug Administration (FDA) announced the approval of Zarxio® (filgrastim-sndz) as a biosimilar to Neupogen®. The formulation of Zarxio differs from that of U.S.-licensed Neupogen in one inactive component, and a review of evidence demonstrated that “there are no clinically meaningful differences between Zarxio and U.S.-licensed Neupogen,” according to an FDA announcement. In addition, the agency notes that more biosimilar treatment options are expected to be approved in the future.
Posted in: Medication Approvals
FDA Advisory Panel Votes for Approval of First U.S. Biosimilar

On Wednesday, Jan. 7, 2015, a U.S. Food and Drug Administration (FDA) Advisory Panel recommended approval of the first U.S. biosimilar medication Zarxio, which is Sandoz’s (a unit of Novartis) equivalent to Neupogen currently manufactured by Amgen. According to the advisory panel, Zarxio should be approved for the same five conditions Neupogen is approved to treat. The FDA will most likely make a decision on the biosimilar medication Zarxio in March, and will consider the advisory panel’s recommendation at that time.

One of MPA’s legislative priorities this year is ensuring that when pharmacists dispense a FDA-approved interchangeable biosimilar medication, there isn’t undue burden placed on them to notify the prescriber of the change. MPA advocates that biosimilar medications should be treated as other generic medications are currently, with no notification to the prescriber that a switch to or from a brand name to interchangeable generic medication has occurred. MPA will continue to monitor any legislation and keep you up-to-date on the biosimilar guidance documents the FDA will release this year.

Posted in: Medication Approvals
Three New Medications Approved for Influenza, Melanoma and Weight Management

The U.S. Food and Drug Administration (FDA) recently approved three new medications. Read more below and click on the links provided to access the full FDA press release for each medication online.

  • The agency approved BioCryst Pharmaceuticals’ Rapivab (peramivir), the first neuraminidase inhibitor approved for intravenous administration to treat influenza in adults. The treatment, made by Durham, N.C.-based BioCryst Pharmaceuticals, is intended for patients 18 years and older who have acute uncomplicated flu and who have shown symptoms for no more than two days.
  • Bristol-Myers Squibb won accelerated FDA approval for Opdivo (nivolumab), a new treatment for patients with unresectable or metastatic melanoma who no longer respond to other drugs.
  • The agency also gave the nod to Saxenda (liraglutide [rDNA origin] injection), a treatment option for chronic weight management in addition to reduced calorie intake and physical activity. The drug, approved with a Risk Evaluation and Mitigation Strategy (REMS), is manufactured by Novo Nordisk.
Posted in: Medication Approvals