Medication Monitor

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  • 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.

  • October 23, 2015

    FDA approved irinotecan liposome injection, administered in combination with fluorouracil (5FU) and leucovorin (LV), for the treatment of patients with metastatic adenocarcinoma of the pancreas whose disease has progressed following gemcitabine-based therapy. Irinotecan liposome is not approved for use as a single agent for the treatment of patients with metastatic adenocarcinoma of the pancreas.  

    Approval was based on the demonstration of improved overall survival (OS) in a multicenter, randomized, open-label, active-controlled, three-arm trial enrolling 417 patients with metastatic pancreatic adenocarcinoma with documented disease progression after gemcitabine-based therapy. Patients were randomly allocated (1:1:1) to receive irinotecan liposome in combination with 5FU and LV (n = 117), irinotecan liposome (n = 151), or 5FU and LV (n = 149) until disease progression or unacceptable toxicity. Patients homozygous for the UGT1A1*28 allele initiated treatment with irinotecan liposome at a reduced dose in the two irinotecan liposome–containing arms.  

    The primary study endpoint was OS, with comparisons of each of the two irinotecan liposome–containing arms with the 5FU/LV control arm; progression-free survival (PFS) and overall response rate (ORR) were secondary endpoints. The trial showed a statistically significant improvement in OS for patients randomized to receive irinotecan liposome in combination with 5FU and LV compared with those randomized to receive 5FU/LV; the median OS was 6.1 and 4.2 months, respectively. 

    PFS was also significantly longer in patients randomized to receive irinotecan liposome plus 5FU/ LV compared with those randomized to receive 5FU/LV, with median PFS of 3.1 and 1.5 months, respectively. The ORR was low in both arms (7.7% vs. 0.8%). There was no improvement in OS for patients randomized to receive irinotecan liposome alone compared with those randomized to receive 5FU/LV.  

    Serious risks of irinotecan liposome injection include neutropenic fever or sepsis, severe diarrhea, and interstitial lung disease. Severe hypersensitivity reactions have occurred with irinotecan hydrochloride; irinotecan liposome injection is contraindicated in patients with severe allergic reactions to irinotecan liposome or irinotecan hydrochloride. 

    The most common adverse drug reactions were diarrhea, fatigue/asthenia, vomiting, nausea, decreased appetite, stomatitis, and pyrexia. The most common severe (Grade 3–-4) laboratory abnormalities were lymphopenia and neutropenia. The most frequent adverse reactions resulting in discontinuationof irinotecan liposome were diarrhea, vomiting, and sepsis.

    The most frequent adverse reactions leading to dose reductions or delays were neutropenia, diarrhea, nausea/vomiting, anemia, fatigue, and thrombocytopenia.  

    The recommended dose and schedule of irinotecan liposome is 70 mg/m2 administered by I.V. infusion over 90 minutes, prior to leucovorin and fluorouracil, every 2 weeks. For patients homozygous for the UGT1A1*28 allele, the recommended starting dose of irinotecan liposome is 50 mg/m2 every 2 weeks.

  • February 2, 2016

    FDA has expanded the approval of nivolumab in combination with ipilimumab for the treatment of patients with BRAF V600 wild type and BRAF V600 mutation–positive unresectable or metastatic melanoma.

    FDA also expanded the use of nivolumab as a single agent to include previously untreated BRAF mutation–positive advanced melanoma patients. Use of nivolumab as a single agent in patients with BRAF V600 mutation–positive unresectable or metastatic melanoma was approved under accelerated approval based on progression-free survival.

  • August 4, 2015

    Aprecia Pharmaceuticals Co. announced that FDA has approved levetiracetam for oral use as a prescription adjunctive therapy in the treatment of partial onset seizures, myoclonic seizures, and primary generalized tonic–clonic seizures in adults and children with epilepsy. 

    Aprecia's ZipDose Technology platform uses three-dimensional printing (3DP) to produce a porous formulation that rapidly disintegrates with a sip of liquid. While 3DP has been used previously to manufacture medical devices, this approval marks the first time a drug product manufactured with this technology has been approved by FDA.

    ZipDose Technology enables the delivery of a high drug load, up to 1,000 mg in a single dose. In addition, no measuring is required, as each dose is individually packaged, making it easy to carry this treatment on the go.

    The product is expected to be available in the first quarter of 2016.

  • June 3, 2015

    FDA approved sirolimus to treat lymphangioleiomyomatosis (LAM), a rare, progressive lung disease that primarily affects women of childbearing age. This is the first drug approved to treat the disease.

    LAM is characterized by an abnormal growth of smooth muscle cells that invade lung tissues (including the airways) and blood/lymph vessels, causing destruction of the lung. LAM is a very rare disease. According to the National Library of Medicine, only between two and five women per million women worldwide are known to have the disease.

    Available as both a tablet and an oral solution, sirolimus was originally approved in 1999 as an immunosuppressive agent to help prevent organ rejection in patients 13 years and older receiving kidney transplants. Because sirolimus’s sponsor demonstrated that the drug may offer a substantial improvement over available therapies, it received breakthrough therapy designation. It also received a priority review and orphan product designation.

    Development of this drug was also supported in part 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.

    Safety and efficacy of sirolimus for the treatment of LAM were studied in a clinical trial that compared sirolimus with placebo in 89 patients for a 12-month treatment period, followed by a 12-month observation period. The primary endpoint was the difference between the groups in forced expiratory volume in one second (FEV1). The difference in the average decrease in FEV1 during the 12-month treatment period was approximately 153 mL. After discontinuation of sirolimus, the decline in lung function resumed at a rate similar to the placebo group.

    The most commonly reported adverse effects were mouth and lip ulcers, diarrhea, abdominal pain, nausea, sore throat, acne, chest pain, leg swelling, upper respiratory tract infection, headache, dizziness, muscle pain, and elevated cholesterol. 

    Serious adverse effects including hypersensitivity and edema have been observed in renal transplant patients.