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Complete response letters; recommendation for approval; fast-track designations; orphan drug designations; first-time generic approvals; FDA actions in brief


Complete response

Proprietary formulation of mannitol (Bronchitol, Pharmaxis) administered as a dry powder in a hand-held inhaler for the treatment of cystic fibrosis. In the complete response letter (CRL), FDA recommended that Pharmaxis conduct an additional clinical trial to obtain an approval for Bronchitol. The CRL stated that: “The submitted data do not provide a favorable benefit-risk balance to support the use of inhaled mannitol in patients with cystic fibrosis aged 6 years and older. The determination of efficacy based on the 2 clinical trials are not adequate because of the treatment-related frequent early dropouts in trial 301 for which the primary statistical analyses did not account and the lack of statistical significance in trial 302 for the primary end point.” In relation to safety, FDA stated its concern with the occurrence of hemoptysis, particularly in pediatric patients. Pharmaxis will follow up with FDA next quarter.


Hepatitis B vaccine (Heplisav, Dynavax Technologies) for the immunization against infection caused by all known subtypes of hepatitis B virus in adults aged 18 through 70 years. In the CRL, FDA specified that the indication in adults 18 through 70 years of age cannot be approved without further evaluation of safety in this broad age group. FDA also continues to express concern that novel adjuvants may cause rare autoimmune events. However, FDA indicated its willingness to continue discussions regarding a more restricted use of Heplisav. Furthermore, FDA requested additional data from Dynavax’s process validation program and clarifying information on the manufacturing controls and facilities related to the assurance of the quality of the commercial product. Dynavax plans to meet with FDA.

Recommended for approval

Probuphine (Titan Pharmaceuticals) for treatment of opioid dependence.

Fast-track designations

TTP488, (TransTech Pharma), a new small-molecule chemical compound for the treatment of Alzheimer's disease. 

CMX001 (Chimerix) for the prevention of cytomegalovirus infection.


Ceftolozane/tazobactam and surotomycin (Cubist Pharmaceuticals) have been issued Qualified Infectious Disease Product (QIDP) and fast-track designations. Ceftolozane/tazobactam was granted QIDP for the indications of hospital-acquired bacterial pneumonia/ventilator-associated bacterial pneumonia, and complicated urinary tract infections. In addition, ceftolozane/tazobactam and surotomycin, have been granted fast-track status in previously granted QIDP indications, complicated Intra-abdominal infections, and Clostridium difficile-associated diarrhea, respectively.

Avarofloxacin (Furiex), has been granted QIDP and fast-track designations for treatment of acute bacterial skin and skin-structure infections, community-acquired pneumonia and has proven to be effective in treating methicillin-resistant staphylococcus aureus infections.

Orphan drug designations

Plasma purified human plasminogen drug (ProMetic BioTherapeutics for the treatment of hypoplasminogenemia, or type I plasminogen deficiency.

G-202, (GenSpera) for the treatment of hepatocellular carcinoma (HCC), the most common form of primary liver cancer.

Elafin (Proteo, Inc./Proteo Biotech AG), a copy of a naturally occurring human anti-inflammatory protein, for the prevention of inflammatory complications of transthoracic esophagectomy.

First-time generic approvals

Flvoxamine maleate extended-release capsules in 100-mg and 50-mg strengths

(equiv to Luvox CR)

Par Pharmaceuticals Cos.

Levalbuterol inhalation solution in 0.31 mg/3 mL, 0.63 mg/3 mL, and 1.25 mg/3 mL strengths (equiv to Xopenex inhalation solution)



Actions in brief


Gadoterate meglumine (Dotarem, Guerbet LLC) was approved for use in magnetic resonance imaging of the brain, spine and associated tissues of patients aged 2 years and older.

Aripiprazole (Abilify Maintena, Otsuka and Lundbeck) was approved for extended-release injectable suspension for the treatment of schizophrenia.

Ospemifene (Osphena, Shionogi) tablets were approved for the treatment of moderate to severe dyspareunia (painful intercourse), a symptom of vulvar and vaginal atrophy (VVA) due to menopause.

Phenylephrine Hydrochloride Ophthalmic Solution (Paragon BioTeck), USP 2.5% and 10%, was approved to dilate the pupil.

Technetium Tc 99m tilmanocept
(Lymphoseek, Navidea Biopharmaceuticals) Injection, a radioactive diagnostic imaging agent, was approved to help locate lymph nodes in patients with breast cancer or melanoma who are undergoing surgery to remove tumor-draining lymph nodes.

Cefixime (Suprax, Lupin Pharmaceuticals) for oral suspension, 500 mg/5 mL was approved for the treatment of otitis media, acute exacerbation of chronic bronchitis, uncomplicated urinary tract infections, uncomplicated gonorrhea (cervical/urethral), and pharyngitis/tonsillitis.

A new vial size for immune globulin intravenous [human] (Privigen, CSL Behring) was approved to treat primary immunodeficiency and chronic immune thrombocytopenic purport.

Expanded use of regorafenib (Stivarga, Bayer HealthCare Pharmaceuticals) was approved to treat patients with advanced gastrointestinal stromal tumors that cannot be surgically removed and no longer respond to other FDA-approved treatments.

Next-generation viral load test (COBAS AmpliPrep/COBAS TaqMan HCV Test, v2.0, Roche) was approved for the management of patients with chronic hepatitis C virus (HCV) infection. The test is designed to accurately determine the amount of hepatitis C virus ribonucleic acid in order to assess a patient’s response to antiviral therapy.


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