FDA drug approvals-November 2014


FDA drug approvals, fast-track designations

FDA actions in brief

Hydrocodone bitartrate (Hysingla ER, Purdue Pharma), an extended-release (ER) opioid analgesic was approved to treat pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate.

Alemtuzumab (Lemtrada, Genzyme) was approved for the treatment of patients with relapsing forms of multiple sclerosis (MS). Because of its safety profile, the use of Lemtrada should generally be reserved for patients who have had an inadequate response to 2 or more drugs indicated for the treatment of MS.

Bevacizumab (Avastin, Roche) in combination with chemotherapy for the treatment of women with platinum-resistant, recurrent ovarian cancer.

A new indication was approved for paliperidone palmitate (Invega Sustenna, Janssen), the once-monthly atypical long-acting antipsychotic to treat schizoaffective disorder as either monotherapy or adjunctive therapy.

Simeprevir (Olysio, Janssen) was approved in combination with sofosbuvir as an oral, interferon- and ribavirin-free treatment option for genotype 1 chronic hepatitis infection in adults.

Ramucirumab (Cyrazma, Eli Lilly) in combination with paclitaxel (a type of chemotherapy) was approved as a treatment for people with advanced or metastatic gastric or gastroesophageal junction adenocarcinoma whose cancer has progressed on or after prior fluoropyrimidine- or platinum-containing chemotherapy. 

Flublok (Protein Sciences) influenza vaccinewas approved for all adults aged 18 years and older, granting approval for use in people 50 and older under the accelerated approval of biological products regulations, 21 CFR 601.40-46. Flublok is the only licensed flu vaccine that is made using modern recombinant technology and the only flu vaccine that is 100% egg-free and highly purified. 

A first-of-its-kind, personalized blood test (Pleximmune) was approved to predict the likelihood of organ rejection in children with liver or intestine transplants. The test was developed by researchers at Children’s Hospital of Pittsburgh of UPMC to determine a personalized rejection-risk index with cell-based technology. The technology was licensed by the University of Pittsburgh to Plexision, a Pittsburgh-based biotech company, for development.

Complete response

AVP-825 (Avanir Pharmaceuticals) drug-device combination product consisting of low-dose sumatriptan powder delivered intranasally utilizing a novel Breath Powered delivery technology for the treatment of migraine. In the complete response letter, FDA has asked Avanir to evaluate the primary reasons that led to erroneous use of the device as was observed in the human factors testing conducted earlier. Additionally, the agency stated that Avanir should conduct a new human factors validation study to evaluate the usability of the device following improvements.

Daclatasvir (Bristol-Myers Squibb), an NS5A complex inhibitor, in combination with other agents for the treatment of hepatitis C (HCV). The initial daclatasvir NDA submitted to FDA focused on its use in combination with asunaprevir, an NS3/4A protease inhibitor. Given the withdrawal of asunaprevir by Bristol-Myers Squibb in October, FDA is requesting additional data for daclatasvir in combination with other antiviral agents for the treatment of HCV. Bristol-Myers Squibb is in discussions with FDA about the scope of these data.

Macimorelin (MacrilenÆterna Zentaris), a novel orally-active ghrelin agonist, for use in evaluating adult growth hormone deficiency (AGHD). FDA's complete response letter (CRL) mentions that the planned analysis of the company's pivotal trial did not meet its stated primary efficacy objective as agreed to in the Special Protocol Assessment agreement letter between the company and FDA. The CRL further mentioned issues related to the lack of complete and verifiable source data for determining whether patients were accurately diagnosed with AGHD. FDA concluded that, "in light of the failed primary analysis and data deficiencies noted, the clinical trial does not by itself support the indication." To address the deficiencies identified above, the CRL states that the company will need to demonstrate the efficacy of macimorelin as a diagnostic test for growth hormone deficiency in a new, confirmatory clinical study. The CRL also outlined that a serious event of electrocardiogram QT interval prolongation occurred for which attribution to drug could not be excluded. Therefore a dedicated thorough QT study to evaluate the effect of macimorelin on the QT interval would be necessary.

Priority review

QIPD designation was granted to two Bayer HealthCare Pharmaceuticals Inc. respiratory drugs: Amikacin Inhale, aerosolized solution in addition to intravenous antibiotics for adult pneumonia patients on breathing machines and ciprofloxacin dry powder for prevention or delay of lung complications in bronchiectasis, a chronic dilation of airway passages.


Fast-track designations

Protein nanoparticle vaccine candidate (Novavax) for protection of infants via maternal immunization

Dupilumab (Sanofi and Regeneron) as breakthrough therapy in the treatment of atopic dermatitis, a chronic form of eczema, granting the product a fast-track development and review 

GC021109 (GliaCure) for Alzheimer's disease.

TH-302 (Threshold Pharmaceuticals), an investigational anticancer drug, for the treatment of previously untreated patients with metastatic or locally advanced unresectable soft tissue sarcoma.

RHB-105 (RedHill Biopharma) Qualified Infectious Disease Product (QUIP) designation for the treatment of Helicobacter pylori (H pylori), a major cause of chronic gastritis, peptic ulcer disease and gastric cancer.

lPRM-151 (Promedior Inc.) as a potential treatment for patients with myelofibrosis. PRM-151 us a recombinant form of an endogenous human protein, Pentraxin-2 and acts as a monocyte/macrophage differentiation factor that may inhibit and potentially reverse fibrosis. The designation encompasses three indications: Primary myelofibrosis, pos-polycythemia vera myelofibrosis and post-essential thrombocythemia myelofibrosis.

MOR208 (MorphoSys) program, a humanized Fc engineered monoclonal antibody against CD19, for the treatment of relapsed or refractory diffuse large B-cell lymphoma. 

Orphan drug designations

SD-809 (Auspex Pharmaceuticals) for its investigational compoundto treat Huntington's disease, a neurodegenerative movement disorder that impacts cognition, behaviour and movements.

Soluble glucagon (Xeris Pharmaceuticals), an investigational new drug, for the prevention of severe, persistent hypoglycemia in patients with congenital hyperinsulinism.

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