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Pegvaliase (Palynziq®) – Enzyme therapy for uncontrolled PKU

The US FDA has approved pegvaliase (Palynziq®, BioMarin Inc) for the treatment of phenylketonurea (PKU) uncontrolled by conventional therapy.

Pegvaliase

  • Pegylated recombinant bacterial Anabaena variabilis phenylalanine ammonia lyase
  • Subcutanous daily injection, 5-60 mg
  • 51.1% decrease in blood Phe levels after 12 months of therapy
  • Half of patients on treatment achieve blood Phe level < 120 µmol/L, the upper limit of normal range, within 2 years
  • Adverse events (common): arthralgia (70.5%), injection-site reaction (62.1%), headache (47.1%)
  • Adverse events (severe): anaphylaxis (4.6%)
  • Current treatment: dietary phenylalanine restriction, sapropterin in THB-responsive PKU
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The US FDA has approved the thrombopoietin receptor agonist avatrombopag (Doptelet®, AkaRx Inc) for the prevention of bleeding events in patients with chronic liver disease and thrombocytopenia undergoing a planned medical or dental procedure.

Avatrombopag

  • Thrombopoietin receptor agonist, stimulates platelet production
  • Decreases serious bleed risk 2-3 fold in patients w/ severe thrombocytopenia (<50*109/L) and chronic liver disease undergoing invasive procedures for up to 7 days after procedure
  • Most common adverse events: fever, abdominal pain, fatigue, edema, nausea
  • Current treatment: pre-procedural platelet transfusion 
  • May increase risk of thromboembolism in some patients

The US FDA has approved the cation chelator sodium zirconium cyclosilicate (ZS-9, Lokelma®) for the treatment of hyperkalemia.

Sodium Zirconium Cyclosilicate

  • Oral potassium cation-exchange agent, not systemically absorbed
  • Onset of action 1 hour, median 2.2 hours until normokalemic
  • Not approved for life-threatening potassium elevations at this time
  • Daily dosing for 1 year twice as likely as placebo to maintain normokalemia
  • Most common adverse effects: GI distress, hypokalemia, UTI, edema
  • Adverse effects less frequent than patiromer (alternative chelating agent for hyperkalemia)

The US FDA has approved the alpha-adrenergic agonist lofexidine HCl (Lucemyra®) for the treatment of opioid withdrawal symptoms.

Key Points

  • First non-opioid approved for treating opioid withdrawal symptoms
  • Approved for up to 14 days after discontinuation of opioids
  • Alpha-2 adrenergic agonist, suppresses sympathetic outflow
  • Originally developed in 1980s for management of hypertension
  • Most common side effects: hypotension, bradycardia, sedation and dizziness
  • Alternative treatments for opioid withdrawal: gradual opioid dose taper, switch to less addictive opioids (e.g., methadone, buprenorphine), use opioid receptor antagonists (naltrexone)

The US FDA has approved the first epoetin alfa biosimilar drug, Retacrit, (epoetin alfa – epbx) for the treatment of anemia.

Key Points

  • First biosimilar drug approved for epoetin alfa (Epogen/Procrit), a recombinant human erythropoetin
  • Approved to treat anemia due to CKD, chemotherapy, zidovudine use, or surgical hemorrhage
  • Epogen/Procrit currently accounts for significant % of Medicare spending, weekly treatments can cost $2,000

fingolimod

The US FDA has approved fingolimod (Gilenya by Novartis) for the treatment of multiple sclerosis (MS) in children aged 10 years or older.

Key Points

  • Once-daily, taken orally
  • Inhibits lymphocyte migration to CNS (downregulates sphingosine receptor)
  • Approved since 2010 for relapsing MS in adults
  • Current first-line: Interferon beta/glatiramer acetate injections (off-label in pediatric MS)