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 PALYNZIQ (pegvaliase-pqpz) 

BioMarin

INDICATION:  Reduce blood phenylalanine concentrations in adult patients with  phenylketonuria (PKU) who have uncontrolled blood phenylalanine (Phe) concentrations greater than 600 micromol/L on existing management

ADDRESSING UNMET NEED:

  • PKU affects about 1 in 10,000 to 15,000 people in US
  • Can cause chronic intellectual, neurodevelopmental, psychiatric disabilitie
  • Novel enzyme substitution therapy for PKU patients unable to control blood Phe levels with current treatment options

MECHANISM OF ACTION: PEGylated phenylalanine ammonia lyase (PAL) enzyme that substitutes for deficient PAH enzyme activity in PKU patients

EFFICACY:

  • Two clinical trials, n>100 PKU patients, unrestricted diet prior to and during the trial
  • First trial- Randomized, open-label, increasing doses of subcutaneous Palynziq
  • Second trial – 8-week, placebo-controlled, randomized withdrawal trial in patients previously treated with Palynziq
  • Statistically significant reductions in blood phenylalanine concentrations from their pre-treatment baseline blood Phe concentrations

SAFETY: 

  • Most common adverse events:  Injection site reactions, joint pain, hypersensitivity reactions, headache, generalized skin reactions lasting at least 14 days, pruritus (itchy skin), nausea, dizziness, abdominal pain, throat pain, fatigue, vomiting, cough and diarrhea\
  • Most serious adverse reaction: Anaphylaxis
  • Boxed Warning and restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Palynziq REMS Program

REGULATORY PATHWAY: BLA

  • Orphan Drug Designation
  • Postmarketing Studies: Prospective, longitudinal, observational study to assess long-term risks of severe immune-mediated adverse reactions, Pre-/Postnatal development study in rats, Assay developments

LABEL


Capture.JPGAIMOVIG (erenumab-aooe)

Amgen

INDICATION: Preventive treatment of migraine in adults

ACCRESSING UNMET NEED:

  • Migraine is 3X more common in women than in men; affects > 10% worldwide
  • Novel option for reducing the number of days with migraine

MECHANISM OF ACTION:  Binds to calcitonin gene-related peptide (CGRP) receptor and antagonizes CGRP receptor function

EFFICACY:

  • Three clinical trials
  • First study, n= 955 participants with history of episodic migraine, Aimovig vs. placebo, 6 mo.- average one to two fewer monthly migraine days vs. placebo
  • Second study, n=577 patients with history of episodic migraine, Aimovig vs. placebo, 3 mo.,- average one fewer migraine day per month vs. placebo
  • Third study, n=667 patients with history of chronic migraine, Aimovig vs. placebo, 3 mo. – average, 2 ½ fewer monthly migraine days vs. placebo

SAFETY: 

  • Most common side effects: Injection site reactions and constipation

REGULATORY PATHWAY: BLA

  • Deferred pediatric studies

LABEL


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LUCEMYRA (lofexidine hydrochloride) 

US WorldMeds LLC

INDICATION:  Mitigation of opioid withdrawal symptoms to facilitate abrupt
opioid discontinuation in adults

ADDRESSING UNMET NEED:

  • First FDA-approved non-opioid treatment for management of opioid withdrawal symptoms
  • New option for providers to work with patients to select the treatment best suited to an individual’s needs

MECHANISM OF ACTION:  Central alpha-2 adrenergic agonist binds to receptors on adrenergic neurons; reduces release of norepinephrine and decreases sympathetic tone

EFFICACY:

  • Two randomized, double-blind, placebo-controlled clinical trials, n=866 adults meeting DSM -IV criteria for opioid dependence, physically dependent on opioids, undergoing abrupt opioid discontinuation
  • Primary endpoint: Short Opiate Withdrawal Scale of Gossop (SOWS-Gossop) – Paient-reported outcome instrument for opioid withdrawal symptoms including feeling sick, stomach cramps, muscle spasms/twitching, feeling of coldness, heart pounding, muscular tension, aches and pains, yawning, runny eyes, insomnia
  • Patients rated symptom severity: Nnone, mild, moderate and severe; higher score indicates greater withdrawal symptom severity.
  • SOWS-Gossop scores lower with Lucemyra vs. placebo
  • More patients completed treatment period with Lucemyra vs. placebo

SAFETY:

  • Most common side effects: Hypotension, bradycardia, somnolence, sedation and dizziness
  • Also associated: Syncope,  increase risk of abnormal heart rhythms

REGULATORY PATHWAY: NDA

  • Fast Track Designation, Priority Review
  • 15 postmarketing studies, including both animal and human safety studies and to support longer term use, use in pediatrics

LABEL


Capture.JPGRETACRIT(epoetin alfa-epbx) 

Pfizer

INDICATION:

  • Anemia Due to Chronic Kidney Disease
  • Anemia Due to Zidovudine in Patients with HIV-infection\
  • Anemia Due to Chemotherapy in Patients with Cancer
  • Reduction of Allogeneic Red Blood Cell Transfusions in Patients Undergoing Elective, Noncardiac, Nonvascular Surgery

ADDRESSING UNMET NEED:

  • First epoetin alfa biosimilar for the treatment of anemia
  • Biosimilars can provide greater access to treatment options, increasing competition and potentially lowering costs

EFFICACY:

  • Biosimilar approved based on data showing that it is highly similar to a marketed biological product
  • Has no clinically meaningful differences in terms of safety, purity and potency (i.e., safety and effectiveness) from the reference product
  • Approval based on a review of evidence that included extensive structural and functional characterization, animal study data, human pharmacokinetic and pharmacodynamic data, clinical immunogenicity data and other clinical safety and effectiveness data that demonstrates Retacrit is biosimilar to Epogen/Procrit

SAFETY:

  • Most common side effects:  High blood pressure, joint pain, muscle spasm, fever, dizziness, medical device malfunction, blood vessel blockage, respiratory infection, cough, rash, injection site irritation, nausea, vomiting, muscle pain, inflammation of the mouth and lips, weight decrease, reduction in white blood cells, bone pain, high blood sugar, insomnia, headache, depression, difficulty swallowing, low blood potassium, blood clots, itching, headache, injection site pain and chills
  • Must be dispensed with patient Medication Guide that provides information about the drug’s uses and risks

LABEL


Image credits: Biomarin, Amgen, US WorldMeds, Pfizer

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