Medication Monitor

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  • June 11, 2019

    On May 6, 2019, FDA approved amifampridine tablets for treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients aged 6 years to younger than 17 years. This is the first FDA approval of a treatment specifically for pediatric patients with LEMS. The only other treatment approved for LEMS is approved for use in adults.

    LEMS is a rare autoimmune disorder in which the body’s immune system attacks the neuromuscular junction and disrupts the ability of nerve cells to send signals to muscle cells. LEMS may be associated with other autoimmune diseases, but more commonly occurs in patients with cancer, such as small cell lung cancer, where its onset precedes or coincides with the diagnosis of cancer.

    LEMS can occur at any age. The prevalence of LEMS specifically in pediatric patients is not known, but the overall prevalence of LEMS is estimated to be three per million individuals worldwide.

    Use of amifampridine in patients aged 6 years to younger than 17 years is supported by evidence from adequate and well-controlled studies of the drug in adults with LEMS, pharmacokinetic data in adult patients, pharmacokinetic modeling and simulation to identify the dosing regimen in pediatric patients, and safety data from pediatric patients in that age group.

    Effectiveness for treatment of LEMS was established by a randomized, double-blind, placebo-controlled withdrawal study of 32 adult patients in which patients were taking amifampridine for at least 3 months before entering the study. The study compared patients continuing on amifampridine to patients switched to placebo. Effectiveness was measured by the degree of change in a test that assessed the time it took the patient to rise from a chair, walk 3 meters, and return to the chair for three consecutive laps without pause. The patients who continued on amifampridine experienced less impairment than those on placebo.

    Effectiveness was also measured with a self-assessment scale for LEMS-related weakness that evaluated the feeling of weakening or strengthening. The scores indicated greater perceived weakening in the patients switched to placebo.

    The most common adverse effects experienced by pediatric and adult patients taking amifampridine were burning or prickling sensation, abdominal pain, indigestion, dizziness, and nausea. Adverse effects reported in pediatric patients were similar to those seen in adult patients. Seizures have been observed in patients without a history of seizures. Patients should inform their health professional immediately if they have signs of hypersensitivity reactions such as rash, hives, itching, fever, swelling, or trouble breathing.

  • May 29, 2019

    FDA approved tafamidis meglumine and tafamidis capsules for treatment of heart disease caused by transthyretin mediated amyloidosis (ATTR-CM) in adults. These are the first FDA-approved treatments for ATTR-CM. Although both treatments have the same active moiety, tafamidis, they are not substitutable on a milligram to milligram basis, and their recommended doses differ.

    Efficacy of tafamidis meglumine and tafamidis in treating ATTR-CM was shown in a clinical trial of 441 patients randomized to receive tafamidis meglumine or a placebo. After an average of 30 months, the survival rate was higher in the tafamidis meglumine group than in the placebo group was also shown to reduce the number of hospitalizations for cardiovascular problems.

    The number of patients in clinical studies was small, but no drug-associated adverse effects have been identified. Tafamidis may cause fetal harm when administered to a pregnant woman. Women taking either treatment should discuss pregnancy planning and prevention with their health professional.

    ATTR is caused by the buildup of abnormal deposits of specific proteins known as amyloid in the body's organs and tissues, interfering with their normal functioning. These protein deposits most frequently occur in the heart and the peripheral nervous system. Heart involvement can result in shortness of breath, fatigue, heart failure, loss of consciousness, abnormal heart rhythms and death. Involvement of the peripheral nervous system can result in a loss of sensation, pain, or immobility in the arms, legs, hands and feet. Amyloid deposits can also affect the kidneys, eyes, GI tract, and central nervous system.

  • May 29, 2019

    FDA approved alpelisib tablets for use in combination with the endocrine therapy fulvestrant to treat men and postmenopausal women with hormone receptor (HR)-positive, HER2-negative, PIK3CA-mutated, advanced or metastatic breast cancer (as detected by an FDA-approved test) following progression on or after an endocrine-based regimen.

    FDA also approved the companion diagnostic test, therascreen PIK3CA RGQ PCR Kit, to detect the PIK3CA mutation in a tissue and/or a liquid biopsy. Patients who are negative by the therascreen test using the liquid biopsy should undergo tumor biopsy for PIK3CA mutation testing. When breast cancer is HR positive, patients may be treated with antihormonal treatment (also called endocrine therapy), alone or in combination with other medicines, or chemotherapy.

    Common adverse effects of alpelisib are high blood glucose levels, increase in creatinine, diarrhea, rash, decrease in lymphocyte count in the blood, elevated liver enzymes, nausea, fatigue, low red blood cell count, increase in lipase, decreased appetite, stomatitis, vomiting, weight loss, low calcium levels, prolonged aPTT, and hair loss.

    Health professionals are advised to monitor patients taking alpelisib for severe hypersensitivity reactions and not to initiate treatment in patients with a history of severe skin reactions such as Stevens-Johnson Syndrome, erythema multiforme, or toxic epidermal necrolysis. Patients receiving alpelisib have reported severe hyperglycemia, and the safety of alpelisib in patients with type 1 or uncontrolled type 2 diabetes has not been established. Before initiating treatment with alpelisib, health professionals are advised to check fasting glucose and A1C and to optimize glycemic control. Patients should be monitored for pneumonitis/interstitial lung disease and diarrhea during treatment. Alpelisib must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.

    Efficacy was studied in the SOLAR-1 trial, a randomized trial of 572 men and postmenopausal women with HR-positive, HER2-negative, advanced or metastatic breast cancer whose cancer had progressed while on or after receiving an aromatase inhibitor. Results from the trial showed the addition of alpelisib to fulvestrant significantly prolonged progression-free survival (median of 11 mo vs. 5.7 mo) in patients whose tumors had a PIK3CA mutation.

  • May 28, 2019

    FDA has approved onasemnogene abeparvovec-xioi, the first gene therapy approved to treat children younger than 2 years who have spinal muscular atrophy (SMA). SMA is the leading genetic cause of infant mortality.

    SMA is a rare genetic disease caused by a mutation in the survival motor neuron 1 (SMN1) gene. SMA caused by mutations in the SMN1 gene is generally classified into several subtypes that are based on age of onset and severity. Infantile-onset SMA is the most severe and most common subtype. Children with this condition have problems holding their head up, swallowing, and breathing. These symptoms may be present at birth or may present by the age of 6 months.

    A one-time I.V. administration of onasemnogene abeparvovec-xioi results in expression of the SMN protein in a child’s motor neurons, which improves muscle movement and function, and survival of a child with SMA. Dosing is determined on the basis of the patient's weight.

    Safety and effectiveness of the drug are based on an ongoing clinical trial and a completed clinical trial involving a total of 36 pediatric patients with infantile-onset SMA between the ages of approximately 2 weeks and 8 months at study entry. The primary evidence of effectiveness is based on results from the 21 patients treated with onasemnogene abeparvovec-xioi a in the ongoing clinical trial.

    The drug's most common adverse effects are elevated liver enzymes and vomiting. A boxed warning cautions that acute serious liver injury can occur. Patients with preexisting liver impairment may be at higher risk of experiencing serious liver injury.

    Certain vaccines are contraindicated for patients on a substantially immunosuppressive steroid dose. Therefore, caregivers should consult with their health professional to determine if adjustments to the patient’s vaccination schedule are necessary to accommodate concomitant corticosteroid administration.

  • May 2, 2019

    FDA has approved Dengvaxia, the first vaccine for prevention of dengue disease caused by all dengue virus serotypes (1, 2, 3, and 4) in people aged 9 through 16 years who have laboratory-confirmed previous dengue infection and who live in endemic areas. Dengvaxia is a live, attenuated vaccine that is administered as three separate injections, with the initial dose followed by two additional shots given 6 and 12 months later.

    Dengue disease is the most common mosquito-borne viral disease in the world and is endemic in the U.S. territories of American Samoa, Guam, Puerto Rico, and the U.S. Virgin Islands. The first infection with dengue virus typically results in either no symptoms or a mild illness that can be mistaken for the flu or another viral infection. A subsequent infection can lead to severe dengue, including dengue hemorrhagic fever, a more severe form of the disease that can be fatal. Symptoms may include stomach pain, persistent vomiting, bleeding, confusion, and difficulty breathing. Approximately 95% of all severe/hospitalized cases of dengue are associated with second dengue virus infection. Because there are no specific drugs approved for treatment of dengue disease, care is limited to managing the symptoms.

    Safety and effectiveness of the new vaccine were determined in three randomized, placebo-controlled studies involving approximately 35,000 individuals in dengue-endemic areas, including Puerto Rico, Latin America, and the Asia Pacific region. The vaccine was determined to be approximately 76% effective in preventing symptomatic, laboratory-confirmed dengue disease in individuals aged 9 through 16 years who previously had laboratory-confirmed dengue disease. Dengvaxia has already been approved in 19 countries and the European Union.

    The most commonly reported adverse effects by those who received Dengvaxia were headache, muscle pain, joint pain, fatigue, injection-site pain, and low-grade fever. The frequency of adverse effects was similar across Dengvaxia and placebo recipients and tended to decrease after each subsequent dose of the vaccine.

    Dengvaxia is not approved for use in individuals not previously infected by any dengue virus serotype or for whom this information is unknown. This is because in people who have not been infected with dengue virus, Dengvaxia appears to act like a first dengue infection—without actually infecting the person with wild-type dengue virus—such that a subsequent infection can result in severe dengue disease. Health professionals should evaluate individuals for prior dengue infection to avoid vaccinating individuals who have not been previously infected by dengue virus. This can be assessed through a medical record of a previous laboratory-confirmed dengue infection or through serological testing prior to vaccination.