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EVENITY (romosozumab-aqqg) injection, for subcutaneous use 

Amgen

INDICATION FOR USE: Treatment of osteoporosis in postmenopausal women at high risk for fracture, defined as a history of osteoporotic fracture, or multiple risk factors for fracture; or patients who have failed or are intolerant to other available osteoporosis therapy
Limitations of Use : Anabolic effect wanes after 12 monthly doses of therapy. Therefore, the duration of EVENITY use should be limited to 12 monthly doses. If osteoporosis therapy remains warranted, continued therapy with an anti-resorptive agent should be considered

ADDRESSING UNMET NEED:
  • > 10 million US women osteoporosis; weakened bones likely to fracture
  • Approval provides women with postmenopausal osteoporosis with high risk of fracture with a new treatment

MECHANISM OF ACTION: Monoclonal antibody that inhibits action of sclerostin, a regulatory factor in bone metabolism; increases bone formation and, to a lesser extent, decreases bone resorption

EFFICACY:
  • 2 randomized, double-blind studies, placebeo and alendronate controlled respectively, n>11,000 women with postmenopausal osteoporosis
  • Endpoints: Risk of a new fracture in spine (vertebral fracture): 73% lowered risk vs. placebo, maintained over the second year of trial when Evenity.  50% lowered risk vs. two years of alendronate alone
  • Evenity followed by alendronate reduced risk of fractures in other bones (nonvertebral fractures) vs. alendronate alone
SAFETY:
  • Boxed Warning: Potential risk for myocardial infarction, stroke and cardiovascular death
  • Common side effects: Joint pain, headache, injection site reactions
REGULATORY PATHWAY: BLA
  • Pediatric assessments: Waived; do not apply to population
  • Postmarketing requirements: Cardiovascular safety

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DENGVAXIA (Dengue Tetravalent Vaccine, Live) Suspension for Subcutaneous Injection

Sanofi Pasteur

INDICATION FOR USE: indicated for the prevention of dengue disease caused by dengue virus serotypes 1, 2, 3 and 4. DENGVAXIA is approved for use in individuals 9 through 16 years of age with laboratory-confirmed previous dengue infection and living in endemic areas
Limitations of use: Not approved for use in individuals not previously infected by any dengue virus serotype or for whom this information is unknown; Safety and effectiveness have not been established in individuals living in dengue non-endemic areas who travel to dengue endemic areas

ADDRESSING UNMET NEED:

  • FDA committed to working with CDC and WHO to combat public health threats
  • Approval is important step toward reducing impact of virus in endemic US regions

MECHANISM OF ACTION: Elicits dengue-specific immune responses against four dengue virus serotypes

EFFICACY:

  • Three randomized, placebo-controlled studies, n=35,000 individuals in dengue-endemic areas, including Puerto Rico, Latin America and Asia Pacific region, 9-16 years of age
  • Endpoint: Symptomatic virologically-confirmed dengue (VCD); DENGVAXIA 76%  effective in preventing VCD in individuals who previously had laboratory- confirmed dengue disease

SAFETY: 

  • Most commonly reported side effects: Headache, muscle pain, joint pain, fatigue, injection site pain and low-grade fever

REGULATORY PATHWAY: BLA

  • Priority Review and a Tropical Disease Priority Review Voucher
  • Postmarketing Study Requirement/Commitments: Safety and effectiveness in children 2 to < 9 years of age

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MAVYRET (glecaprevir and pibrentasvir) tablets

AbbVie

INDICATION FOR USE: Treatment of adult and pediatric patients 12 years and older or weighing at least 45 kg with chronic hepatitis C virus (HCV) genotype 1, 2, 3, 4, 5 or 6 infection without cirrhosis or with compensated cirrhosis (Child-Pugh A)

ADDRESSING UNMET NEED:  First treatment for all genotypes of hepatitis C in pediatric patients

MECHANISM OF ACTION: Fixed-dose combination of glecaprevir and pibrentasvir, which are direct-acting antiviral agents against the hepatitis C virus

EFFICACY:

  • Open-label study, n=47  adolescent subjects 12 -< 18 years  with genotype 1, 2, 3 or 4 HCV infection without cirrhosis or with mild cirrhosis, 8 or 16 weeks.
  • 100% had no virus detected in blood 12 weeks after finishing treatment, suggesting  infection had been cured

SAFETY:

  • Adverse reactions observed were consistent with those observed in adults
  • Most commonly reported adverse reactions: Headache and fatigue

REGULATORY PATHWAY: Supplemental NDA

  • Initial US approval (NDA) in adult patients in 2017

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BENLYSTA (belimumab) intravenous infusion

GlaxoSmithKline

INDICATION FOR USE: Treatment of patients aged 5 years and older with active, autoantibody-positive, systemic lupus erythematosus (SLE) who are receiving standard therapy

ADDRESSING UNMET NEED:

  • First treatment for pediatric patients with Systemic Lupus Erythematosus (SLE)
    • Benlysta has been approved for use in adult patients since 2011

EFFICACY:

  • International, randomized, doubleblind, placebo-controlled, 52-week, n=93  pediatric patients with a clinical diagnosis of SLE
  • Primary efficacy endpoint: SLE Responder Index (SRI-4) at Week 52; numerically higher patients achieving a response in SRI-4 with BENLYSTA vs. placebo

SAFETY:

  • Warning for mortality, serious infections, hypersensitivity and depression
  • Most common side effects: Nausea, diarrhea and fever, infusion reactions

REGULATORY PATHWAY: sBLA, Priority review

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TIBSOVO (ivosidenib) tablets

Agios Pharmaceuticals

RealTime IDH1 Assay

Abbott

INDICATION FOR USE: Treatment of newly-diagnosed acute myeloid leukemia (AML)  with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in adult patients who are ≥ 75 years old or who have comorbidities that preclude use of intensive induction chemotherapy

ADDRESSING UNMET NEED: First-line treatment for AML with IDH1 mutation

EFFICACY:

  • Open-label, single-arm, multicenter clinical trial, n=28 (newly-diagnosed AML with IDH1 mutation detected by Abbott RealTimeTM IDH1 Assay
  • Endpoints: Rate of complete remission (CR) or complete remission with partial hematologic recovery (CRh), the duration of CR+CRh, conversion rate from transfusion dependence to transfusion independence
  • CR+CRh :42.9%, 41.2% achieved transfusion independence lasting at least 8 weeks

SAFETY:

  • Boxed Warning for Differentiation Syndrome which may be life-threatening or fatal
  • Adverse reactions: Diarrhea, fatigue, edema, decreased appetite, leukocytosis, nausea, arthralgia, abdominal pain, dyspnea, differentiation syndrome and myalgia

REGULATORY PATHWAY: sNDA

  • Used Real-Time Oncology Review pilot program
  • Priority Review and Orphan Product designation
  • Postmarketing requirement: Determine safe dose in mild, moderate and severe hepatic impairment

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VYNDAQEL (tafamidis meglumine) and VYNDAMAX (tafamidis) capsules 

FoldRx (Pfizer subsidiary)

INDICATION FOR USE: Treatment of cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization

ADDRESSING UNMET NEED:

  • Transthyretin-mediated amyloidosis is rare, debilitating, often fatal disease
  • Approval an important advancement in treatment of cardiomyopathy caused by transthyretin-mediated amyloidosis

MECHANISM OF ACTION: Selective stabilizer of TTR;  slows dissociation into monomers, the rate-limiting step in the amyloidogenic process

EFFICACY:

  • Multicenter, international, randomized, double-blind, placebo-controlled study
    n=441 patients with wild type or hereditary ATTR-CM
  • Endpoints: All-cause mortality and frequency of cardiovascular-related hospitalizations
  • Significant reduction (p=0.0006) in all-cause mortality and frequency of
    cardiovascular-related hospitalizations with Vyndaqel / Vyndamax group

SAFETY:

  • No drug-associated side effects identified
  • May cause fetal harm when administered to pregnant woman

REGULATORY PATHWAY: NDA

  •  Fast Track, Priority Review and Breakthrough Therapy designations
  • Orphan Drug designation

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RUZURGI (amifampridine) tablets 

Jacobus Pharmaceutical Company

INDICATION FOR USE: Treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to less than 17 years of age

ADDRESSING UNMET NEED:

  • LEMS is a rare autoimmune disorder affecting connection between nerves and muscles
  • First approved treatment for LEMS specifically in children

MECHANISM OF ACTION: Broad spectrum potassium channel blocker

EFFICACY:

  • Randomized, double-blind, placebo-controlled withdrawal study
  • Primary measure of efficacy: Change in Triple Timed Up and Go test (3TUG)  and self-assessment scale for LEMS-related weakness
  • Less impairment with Ruzurgi; greater perceived weakening with placebo

SAFETY: 

  • Most common side effects: Burning or prickling sensation (paresthesia), abdominal pain, indigestion, dizziness and nausea
  • Seizures in patients without a history of seizures

REGULATORY PATHWAY: NDA,  Priority Review and Fast Track designations, Orphan Drug designation

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Image credits: Amgen, Sanofi, AbbVie, Agios, GSK, Pfizer, Jacobus

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