Medication Monitor

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  • 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.

  • 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.

  • 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 22, 2015

    FDA approved coagulation Factor X (human) for hereditary Factor X (10) deficiency under the trade name Coagadex. Until today’s orphan drug approval, no specific coagulation factor replacement therapy was available for patients with hereditary Factor X deficiency.

    In healthy individuals, the Factor X protein activates enzymes to help with normal blood clotting in the body. Factor X deficiency is an inherited disorder, affecting men and women equally, in which the blood does not clot as it should. Patients with the disorder are usually treated with fresh-frozen plasma or plasma-derived prothrombin complex concentrates (plasma products containing a combination of vitamin K–dependent proteins) to stop or prevent bleeding. The availability of a purified Factor X concentrate increases treatment options for patients with this rare bleeding disorder.

    Coagadex, which is derived from human plasma, is indicated for individuals aged 12 and older with hereditary Factor X deficiency for on-demand treatment and control of bleeding episodes, and for perioperative management of bleeding in patients with mild hereditary Factor X deficiency.

    Safety and efficacy of the product were evaluated in a multicenter, nonrandomized study involving 16 participants (208 bleeding episodes) for treatment of spontaneous, traumatic, and heavy menstrual bleeding episodes. Coagadex was demonstrated to be effective in controlling bleeding episodes in participants with moderate to severe hereditary Factor X deficiency.

    Coagadex was also evaluated in five participants with mild to severe Factor X deficiency who were undergoing surgery. The five individuals received Coagadex for perioperative management of seven surgical procedures. Coagadex was demonstrated to be effective in controlling blood loss during and after surgery in participants with mild deficiency. No individuals with moderate or severe Factor X deficiency received Coagadex for perioperative management of major surgery, and no safety concerns were identified in either study.

    Coagadex was also granted fast track designation and priority review.

  • 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.