New FDA Approvals
Several new drugs have been approved since April of this year including treatments for migraine, liver disease, kidney disease, and those facing opioid withdrawal symptoms.
Aimovig (erenumab-aooe) from Amgen has been approved for the preventive treatment of episodic or chronic migraine in adults. Aimovig is supplied as an injection with a recommended dosage of 70 mg once per month. Some patients may benefit from two injections of 70 mg once per month. If a dose of Aimovig is missed, another dose should be administered as soon as possible.
Adverse reactions associated with the use of Aimovig may include, but are not limited to, the following:
- injection site reactions
- constipation
For those with a rare, inherited, disorder known as X-linked hypophosphatemia (XLH) Crysvita (burosumab-twza) from Ultragenyx has recently been approved.
XLH is characterized by low levels of phosphate in the blood. Phosphate levels are low because phosphate is abnormally processed in the kidneys, which causes a loss of phosphate in the urine and leads to soft, weak bones (rickets).This genetic disorder affects about one in 20,000 people and is typically, it is passed from one generation to the next, but sometimes appears in an individual with no family history of XLH.
Crysvita is specifically indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older and is supplied as an injection every two to four weeks depending on the age of the patient and the length of treatment.
Side effect may include, but are not limited to:
Pediatrics:
- headache
- injection site reaction
- vomiting
- pyrexia
- pain in extremity
- vitamin D decreased
Adults:
- back pain
- headache
- tooth infection
- restless leg syndrome
- vitamin D decreased
- dizziness
- constipation
- blood phosphorus increased
AndexXa (coagulation factor Xa (recombinant), inactivated-zhzo) by Portola Pharmaceuticals has recently been approved for the reversal of factor Xa inhibitors. AndexXa is administered via intravenous injection.
This is the first and only antidote indicated for patients treated with drugs such as rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding. Factor Xa inhibitors are anticoagulants used to prevent deep vein thrombosis and pulmonary embolism in patients undergoing hip or knee replacement surgery.
Side effects include:
- urinary tract infections
- pneumonia
Two new drugs have been approved for the treatment of thrombocytopenia (low blood platelet count).
Doptelet (avatrombopag) from AkaRx has been approved for the treatment of thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a procedure that would typically require platelet transfusion. Doptelet is supplied as a tablet for oral administration with dosing beginning 10-13 days prior to the scheduled procedure.
Side effects may include, but are not limited to:
- pyrexia
- abdominal pain
- nausea
- headache
- fatigue
- edema peripheral
Tavalisse (fostamatinib disodium hexahydrate) from Rigel Pharmaceuticals has been approved for the treatment of chronic immune thrombocytopenia (ITP) in adult patients who have had an insufficient response to a previous treatment.
Tavalisse (fostamatinib disodium hexahydrate) is a tyrosine kinase inhibitor with demonstrated activity against spleen tyrosine kinase (SYK).
Tavalisse is supplied as a tablet for oral administration with an initial dose of 100 mg taken orally twice daily for a month. Depending on platelet county, dosage may be increased. Tavalise may be taken with or without food.
Side effects may include:
- diarrhea
- hypertension
- nausea
- respiratory infection
- dizziness
- ALT/AST increased, rash
- abdominal pain
- fatigue
- chest pain
- neutropenia
For those with higher than normal potassium levels—known as hyperkalemia—Lokelma (sodium zirconium cyclosilicate) from AstraZeneca has been approved. Lokelma is a highly-selective, oral potassium-removing agent.
Lokelma is administered orally as a suspension in water for oral administration. The recommended dose is 10 g administered three times a day for up to 48 hours. For continued treatment, the recommended dose is 10 g once daily. The most common adverse reaction associated with the use of Lokelma is mild to moderate edema.
Palynziq (pegvaliase-pqpz) from BioMarin has been approved for the treatment of phenylketonuria. Phenylketonuria (commonly known as PKU) is an inherited disorder that increases the levels of a substance called phenylalanine in the blood. Phenylalanine is a building block of proteins (an amino acid) that is obtained through the diet. It is found in all proteins and in some artificial sweeteners.Without treatment, phenylalanine builds up in the blood and causes health problems.
Playnziq is specifically indicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.
Side effects of Palynziq may include:
- injection site reactions
- arthralgia
- hypersensitivity reactions
- headache
- generalized skin reactions lasting at least 14 days
- pruritus
- nausea
- abdominal pain
- oropharyngeal pain
- vomiting
- cough
- diarrhea
- fatigue
Jynarque (tolvaptan) by Otsuka America has been approved for the treatment of autosomal dominant polycystic kidney disease (PKD). PKD is a genetic disorder characterized by the growth of numerous cysts filled with fluid in both kidneys. The progressive expansion of PKD cysts slowly replaces much of the normal mass of the kidneys, and can reduce kidney function and lead to kidney failure.
Jynarque is a selective vasopressin V2-receptor antagonist that is specifically indicated to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease.
Jynarque is supplied as a tablet for oral administration. The recommended initial dose is 60 mg orally per day as 45 mg taken on waking and 15 mg taken 8 hours later. Titrate to 60 mg plus 30 mg then to 90 mg plus 30 mg per day if tolerated with at least weekly intervals between titrations. Patients may down-titrate based on tolerability.
Adverse effects associated with the use of Jynarque may include, but are not limited to:
- thirst
- polyuria
- nocturia
- pollakiuria
- polydipsia
Jynarque (tolvaptan) is not without risk and can cause serious and potentially fatal liver injury. Acute liver failure requiring liver transplantation has been reported.
Lucemyra(lofexidine) by US Worldmeds has been approved for the mitigation of opioid symptoms to facilitate abrupt opioid discontinuation in adults.
Lucemyra is supplied as a tablet for oral administration. The usual starting dosage is three 0.18 mg tablets taken orally 4 times daily during the period of peak withdrawal symptoms (generally the first 5 to 7 days following last use of opioid) with dosing guided by symptoms and side effects. There should be 5 to 6 hours between each dose. The total daily dosage of Lucemyra should not exceed 2.88 mg (16 tablets) and no single dose should exceed 0.72 mg (4 tablets).
Lucemyra treatment may be continued for up to 14 days with dosing guided by symptoms. Discontinue Lucemyra with a gradual dose reduction over a 2- to 4-day period to mitigate Lucemyra withdrawal symptoms.
Adverse effects associated with the use of Lucemyra may include, but are not limited to:
- orthostatic hypotension
- bradycardia
- hypotension
- dizziness
- somnolence
- sedation
- dry mouth
Lucemyra may cause low blood pressure or slower heart rate and should be used with caution in patients diagnosed with low blood pressure, slow heart rate, any other cardiac abnormality (including prior diagnosis or family history of long QT syndrome), or prior heart attack.
