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AEMCOLO (rifamycin) AEMCOLO (rifamycin) delayed-release tablets

Cosmo Technologies, Ltd

INDICATION: Treatment of travelers’ diarrhea (TD) caused by non-invasive strains of Escherichia coli in adults

  • Limitations of Use: Not recommended for use in patients with diarrhea complicated by fever and/or bloody stool or due to pathogens other than noninvasive strains of E. coli.

ADDRESSING UNMET NEED: 

  • Travelers’ diarrhea affects 10–40 % travelers worldwide each year
  • Highest-risk destinations in most of Asia as well as the Middle East, Africa, Mexico, and Central and South America

MECHANISM OF ACTION: Antibacterial drug

EFFICACY:

  • Multi-center, randomized, double-blind, placebo (Guatemala, Mexico)  and active-controlled (India, Guatemala and Ecuador) trials
  • Primary endpoint: Time to last unformedstool (TLUS) before achieving clinical cure
  • AEMCOLO significantly reduced the TLUS compared to placebo (p=0.0008); supportive outcome ina ctive controlled trial.

SAFETY: 

  • Most common adverse reactions (incidence > 2%): Headache and constipation

REGULATORY PATHWAY: NDA

  • Qualified Infectious Disease Product (QIDP) designation under the Generating Antibiotic Incentives Now (GAIN) title of the FDA Safety and Innovation Act
  • Priority Review
  • Deferred pediatrkc studies: Treatment of travelers’ diarrhea in children from 6 – 11 years of age and 12-17 years of age

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YUPELRI™ (revefenacin) inhalation solution

Theravance and Mylan

INDICATION: Mtreatment of patients with chronic obstructive pulmonary disease (COPD)

MECHANISM OF ACTION: Long-acting muscarinic antagonist, inhibition of M3 receptor at the smooth muscle leading to bronchodilation

EFFICACY:

  • Two dose-ranging trials, two replicate 12-week, Phase 3 confirmatory clinical trials, and a 52-week safety trial
  • Confirmatory Trials: n=1,229 subjects, YUPELRI vs. placebo once daily
  • Primary endpoint: Change from baseline in trough (predose) Forced Expiratory Volume in One Second or FEV1  at Day 85
  • Significant improvement in lung function vs placebo

SAFETY:

  • Most common adverse reactions (incidence greater than or equal to 2% and more common than placebo):  Cough, nasopharyngitis, upper respiratory tract infection, headache, back pain
  • Worsening of narrow-angle glaucoma may occur

REGULATORY PATHWAY: NDA

  • Pediatric requirements waived

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Novimmune SA

INDICATION: Treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or
progressive disease or intolerance with conventional HLH therapy

ADDRESSING UNMET NEED:

  • Primary HLH is rare and life-threatening condition typically affecting children
  • Approval fills an unmet medical need for these patients

MECHANISM OF ACTION: Monoclonal antibody that binds to and neutralizes interferon gamma

EFFICACY:

  • Clinical trial of 27 pediatric patients,  with suspected or confirmed primary HLH,  intolerant of conventional HLH therapy
  • 63% experienced response and 70 % were able to proceed to stem cell transplant

SAFETY:

  • Common side effects: Infections, hypertension, infusion-related reactions, low potassium and fever
  •  Patients should not receive any live vaccines and should be tested for latent tuberculosis
  • Patients should be closely monitored and treated promptly for infections

REGULATORY PATHWAY: BLA

  • Orphan designation, Priority Review and Breakthrough Therapy designation

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DAURISMO (glasdegib) tablets

Pfizer

INDICATION: In combination with low-dose cytarabine, for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients who are ≥75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.

  • Limitation of Use: Has not been studied in patients with the comorbidities of severe renal impairment or moderate-to-severe hepatic impairment

MECHANISM OF ACTION: Hedgehog pathway inhibitor, binds to and inhibits Smoothened, a transmembrane protein involved in hedgehog signal transduction

EFFICACY:

  • Multicenter, openlabel, randomized study, n=115 patients with newly-diagnosed AML, DAURISMO with low-dose Cytarabine va. low-dose Cytarabine
  • Primary Endpoint: Overall Survival; 8.3 mo. vs 4.3 mo, p=0.0002

SAFETY:

  • Boxed warning: Can cause embryo-fetal death or severe birth defects
  • Most common adverse reactions (incidence ≥20%): Anemia, fatigue, hemorrhage, febrile neutropenia, musculoskeletal pain, nausea, edema, thrombocytopenia, dyspnea, decreased appetite, dysgeusia, mucositis, constipation, rash

REGULATORY PATHWAY: NDA

  • Orphan and Priority designations

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Image credits: Cosma, Theravance, Novimmune, Pfizer

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