FDA Market Authorizations: AEMCOLO for travelers’ diarrhea, YULPERI for COPD, GAMIFANT for rare immune disease, DAURISMO for AML
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
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
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
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
Image credits: Cosma, Theravance, Novimmune, Pfizer