Ibuprofen is a non-steroidal anti-inflammatory drug (NSAID) and non-selective cyclooxygenase inhibitor indicated for the treatment of mild-moderate pain, fever, and arthritis. Its analgesic, antipyretic, and anti-inflammatory properties are thought to be mediated through inhibition of the cyclooxygenase-2 (COX-2) enzyme. COX-2 is upregulated by cells during the inflammatory response, resulting in increased production of PGE2 and leading to tissue inflammation, fluid extravasation, pyrexia, pain, and vasodilation. Inhibition of prostaglandin synthesis by ibuprofen blocks inflammatory signaling pathways and decreases pain and body temperature.
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.
- 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
Epidiolex® – investigational cannabidiol formulation
- Study: GWPCARE3
- Authors: Devinsky, Zuberi et al
- Journal: New England Journal of Medicine
- Date: May 17th, 2018
- Cannabidiol add-on to conventional antiepileptic therapy reduced drop seizure frequency in patients with Lennox–Gastaut syndrome
- Both 10 mg/kg and 20 mg/kg cannabidiol effective in reducing number of seizures
- Dose-dependent increase in adverse events relative to placebo
Levothyroxine is a thyroid receptor agonist commonly prescribed for hypothyroidism. It is a synthetically manufactured levo-isomer of the endogenous thyroxine (T4) hormone, and is converted peripherally to triiodothyronine (T3) hormone. T3 binds to thyroid hormone receptors (TR) and can induce either transcriptional repression or derepression depending on the molecular and cellular context. These transcriptional changes give rise to an increased sensitivity to catecholamines (e.g., raising heart rate and blood pressure) and increasing metabolic activity. Pituitary thyroid-stimulating hormone (TSH) is downregulated by levothyroxine administration.
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.
- 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
Atorvastatin is statin-class drug commonly prescribed for the treatment of dyslipidemias and for the prevention of ischemic cardiovascular events. It works by inhibiting hydroxymethylglutaryl-coenzyme A (HMG-CoA) reductase, a key enzyme in the cholesterol biosynthetic pathway. Inhibition of HMG-CoA reductase decreases hepatic cholesterol synthesis, increases hepatic cholesterol re-uptake, and decreases serum cholesterol levels.