Medication Monitor



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  • November 13, 2015

    FDA granted accelerated approval to osimertinib once-daily tablets for the treatment of patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non–small cell lung cancer (NSCLC), as detected by an FDA-approved test, who have progressed on or after EGFR tyrosine kinase inhibitor (TKI) therapy.  

    Approval was based on two multicenter, single-arm, open-label clinical trials in patients with metastatic EGFR T790M mutation-positive NSCLC who had progressed on prior systemic therapy, including an EGFR TKI (Study 1 and 2).  All patients were required to have EGFR T790M mutation-positive NSCLC as detected by the cobas EGFR mutation test and received osimertinib 80 mg once daily. The major efficacy outcome measure was objective response rate (ORR). Duration of response (DOR) was an additional outcome measure.   Safety data were evaluated in 411 patients who received osimertinib at a dose of 80 mg daily. The most common adverse events were diarrhea, rash, dry skin, nail toxicity, eye disorders, nausea, decreased appetite, and constipation.  

    The most common nonfatal serious adverse events (SAEs) included pneumonia and pulmonary embolus. The most frequent adverse reaction leading to dose reductions or interruptions were prolonged QTc and neutropenia. Adverse events leading to discontinuation included ILD/pneumonitis  and cerebrovascular accident.  

    Fatal adverse events occurred in 3.2% of patients, including four cases of pneumonitis attributed to osimertinib.  

    The recommended dose and schedule for osimertinib is 80 mg given orally once daily.

  • October 26, 2015

    FDA approved asfotase alfa as the first approved treatment for perinatal, infantile-onset, and juvenile-onset hypophosphatasia (HPP).  

    HPP is a rare, genetic, progressive, metabolic disease in which patients experience devastating effects on multiple systems of the body, leading to severe disability and life-threatening complications. It is characterized by defective bone mineralization that can lead to rickets and softening of the bones that result in skeletal abnormalities. It can also cause complications such as profound muscle weakness with loss of mobility, seizures, pain, respiratory failure, and premature death. Severe forms of HPP affect an estimated 1 in 100,000 newborns, but milder cases, such as those that appear in childhood or adulthood, may occur more frequently.

    Asfotase alfa received a breakthrough therapy designation because it is the first and only treatment for perinatal, infantile-onset, and juvenile-onset HPP. FDA also granted asfotase alfa orphan drug designation because it treats a disease affecting fewer than 200,000 patients in the United States, as well as priority review and a rare pediatric disease priority review voucher.

    Development of this drug was also in part supported by the FDA Orphan Products Grants Program, which provides grants for clinical studies on safety and/or effectiveness of products for use in rare diseases or conditions.

    Asfotase alfa is administered via injection three or six times per week. The medication works by replacing the enzyme (known as tissue-nonspecific alkaline phosphatase) responsible for formation of an essential mineral in normal bone, which has been shown to improve patient outcomes.

    Safety and efficacy of the drug were established in 99 patients with perinatal, infantile, or juvenile-onset HPP who received treatment for up to 6.5 years during four prospective, open-label studies. Study results showed that patients with perinatal- and infantile-onset HPP treated with asfotase alfa had improved overall survival and survival without the need for a ventilator.

    Ninety-seven percent of treated patients were alive at 1 year of age, compared with 42% of control patients selected from a natural history study group. Similarly, the ventilator-free survival rate at 1 year of age was 85% for treated patients, compared with less than 50% for the natural history control patients.  

    Patients with juvenile-onset HPP treated with asfotase alfa showed improvements in growth and bone health compared with control patients selected from a natural history database. All treated patients had improvement in low weight or short stature or maintained normal height and weight. In comparison, approximately 20% of control patients had growth delays over time, with shifts in height or weight from the normal range for children their age to heights and weights well below normal for age.

    Juvenile-onset patients also showed improvements in bone mineralization, as measured on a scale that evaluates the severity of rickets and other HPP-related skeletal abnormalities based on x-ray images. All treated patients demonstrated substantial healing of rickets on x-rays, while some natural history control patients showed increasing signs of rickets over time.

    The most common adverse effects in patients treated with asfotase alfa include injection site reactions, hypersensitivity reactions (such as difficulty breathing, nausea, dizziness and fever), lipodystrophy at the injection site, and ectopic calcifications of the eyes and kidney.

  • November 18, 2015

    FDA has approved antihemophilic factor (recombinant), polyethylene glycol (PEGylated), for use in adults and adolescents aged 12 years and older who have hemophilia A. The new therapy has been approved under the trade name Adynovate.

    Adynovate is modified to last longer in the blood and potentially require less-frequent injections than unmodified antihemophilic factor when used to reduce the frequency of bleeding.

    Adynovate is approved for on-demand treatment and control of bleeding episodes and to reduce the frequency of bleeding episodes in patients with hemophilia A. Adynovate consists of the full-length coagulation Factor VIII molecule (historically known as antihemophilic factor) linked to other molecules, known as polyethylene glycol (PEGylated). This link makes the product last longer in the patient’s blood.

    Safety and efficacy of adynovate were evaluated in a clinical trial of 137 adults and adolescents aged 12 years and older, which compared the recommended routine prophylactic treatment regimen to on-demand therapy. The trial demonstrated that Adynovate was effective in reducing the number of bleeding episodes during routine care. In addition, Adynovate was effective in treating and controlling bleeding episodes. No safety concerns were identified during the trial.

  • December 11, 2018

    Fresenius Kabi announced that fish oil triglycerides injectable emulsion, approved under the trade name Omegaven, is now commercially available in the United States. This novel lipid had previously been available only for compassionate use in the United States.

    Omegaven is an I.V. lipid emulsion that provides calories and fatty acids for pediatric patients with parenteral nutrition-associated cholestasis, or PNAC. It is the first and only FDA-approved fish oil lipid emulsion for this condition.

    The product is available as a 5 g/50 mL and 10 g/100 mL (0.1 g/mL) injectable emulsion in a single-dose bottle.  

    Cholestasis is a condition in which bile is not released from the liver. PNAC may occur following long-term parenteral nutrition administration in pediatric patients with temporary or permanent intestinal failure. Development of PNAC is associated with increased morbidity and mortality and can progress to liver fibrosis, hepatic failure, and death.

    In clinical trials, the most common adverse drug reactions (>15%) were vomiting, agitation, bradycardia, apnea, and viral infection.

  • August 30, 2015

    FDA approved eltrombopag to treat low blood platelet count in pediatric patients aged 1 year and older with chronic immune thrombocytopenic purpura (ITP), a rare blood disorder. The agent can be used in these children when they have not achieved an appropriate response using other ITP medicines or surgery to remove the spleen.

    ITP is a disorder that results in an abnormally low number of platelets. Without enough platelets, bleeding can occur under the skin, in mucous membranes (such as in the mouth) or in other parts of the body.

    Eltrombopag helps increase blood platelet production and is available as a tablet taken once-daily or as a powder that is mixed with liquid for children aged 1 to 5 to take orally. It was first approved in 2008 to treat adult patients with the same condition as the new pediatric indication.

    The agent should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.

    Efficacy and safety of eltrombopag in pediatric patients aged 1 to 17 years with chronic ITP was evaluated in two double-blind, placebo-controlled trials of 159 participants where the primary endpoint was an increase in platelet counts.

    In the first trial (n = 67), patients were randomly assigned to receive either eltrombopag or placebo daily for 7 weeks. Of those taking eltrombopag, 62% had an improvement in platelet counts without rescue therapy between weeks 1 and 6, compared with 32% in the placebo group.

    In the second trial (n = 92), patients received either eltrombopag or placebo daily for 13 weeks. In those treated with eltrombopag, 41% experienced increased platelet counts for at least 6 out of 8 weeks between weeks 5 to 12, compared with 3% of patients receiving placebo.

    In both trials, patients taking eltrombopag also had less need for other treatments to increase their platelet counts, such as corticosteroids or platelet transfusions. Among patients taking one or more ITP medications at the start of the trials, about one-half were able to reduce or discontinue their use of these medications, primarily corticosteroids.

    The most common adverse effects of treatment in children aged 1 and older were infections of the upper respiratory tract or nose and throat (symptoms including fever, cough, nasal congestion, runny nose, and sore throat), diarrhea, abdominal pain, rash, and increase in liver enzymes.

    Safety and efficacy of eltrombopag in pediatric patients younger than 1 year with ITP, or in pediatric patients with thrombocytopenia associated with chronic hepatitis C and severe aplastic anemia, have not been established.

    FDA granted eltrombopag orphan drug designation because it treats a rare disease.

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