FDA and Pfizer announced the approval of bosutinib (Bosulif), an Abl kinase and Src kinase inhibitor, for the treatment of adult patients with chronic, accelerated, or blast-phase Philadelphia chromosome–positive (Ph+) chronic myelogenous leukemia (CML) with resistance or intolerance to prior therapy. This new oral medication is taken once daily with food at a dose of 500 mg. Approval was based on data from Study 200, a global, open-label, multicohort, Phase I/II study of more than 500 patients with imatinib (Gleevec—Novartis)–resistant or –intolerant Ph+ CML previously treated with one or more tyrosine kinase inhibitors. For patients with chronic phase CML who had been previously treated with imatinib and/or at least one other tyrosine kinase inhibitor, the major cytogenetic response at 24 weeks and at the long-term follow-up visit was increased with bosutinib treatment.
FDA announced the approval of adalimumab (Humira—Abbott) for the treatment of moderate to severe ulcerative colitis in adults who have had an inadequate response to conventional therapies such as corticosteroids, azathioprine, and 6-mercaptopurine. The dosing regimen for adalimumab for ulcerative colitis begins with an initial dose of 160 mg, a second dose 2 weeks later of 80 mg, and a maintenance dose of 40 mg every other week thereafter. Approval was based on data from two clinical studies involving a total of 908 patients with ulcerative colitis who were treatment naive to tumor necrosis factor (TNF) blockers or who lost response or were intolerant to TNF blockers. A higher percentage of patients treated with adalimumab achieved clinical remission in both trials (16.5%–18.5%) compared with placebo (9.2%–9.3%).
Regeneron announced the FDA approval of aflibercept injection (Eylea) for the treatment of macular edema following central retinal vein occlusion. The recommended dose for this new indication is 2 mg administered by intravitreal injection once every 4 weeks. Approval was based on data from two randomized, multicenter, double-masked, sham-controlled, Phase III studies. In the first study, 56% of patients receiving aflibercept gained at least 15 letters of best corrected visual acuity from baseline to week 24, compared with 12% of patients who received sham injections (P < 0.01). In the second study, 60% of patients who received aflibercept gained at least 15 letters from baseline at week 24, compared with 22% of patients who received sham injections (P < 0.01). The drug is already indicated for the treatment of neovascular (wet) age-related macular degeneration.
Amgen announced FDA’s approval of denosumab (Prolia) as a treatment to increase bone mass in men with osteoporosis at high risk for fracture. Approval is based on data from ADAMO, a Phase III, multicenter, randomized, double-blind, placebo-controlled, 12-month trial which compared the efficacy and safety of denosumab 60 mg given once every 6 months with placebo in 242 men with osteoporosis. Treatment with denosumab resulted in significantly greater gains in bone mineral density at the lumbar spine compared with placebo (5.7% vs. 0.9%). The most common adverse reactions included back pain, arthralgia, and nasopharyngitis.
Acton announced approval of flunisolide HFA inhalation aerosol 80 µg (Aerospan) for the maintenance treatment of asthma as prophylactic therapy in adult and pediatric patients 6 years and older. This new inhalation product was developed with an integrated spacer device that has been studied in clinical trials involving more than 1,700 patients. Data from both adult and pediatric trials showed that use of flunisolide inhalation resulted in significant improvements in change from baseline in the primary endpoint of forced expiratory volume in 1 second compared with placebo. Acton plans to launch this product in early 2013.
NextWave Pharmaceuticals announced the FDA approval of methylphenidate extended-release oral suspension (Quillivant XR) for the treatment of ADHD. This new liquid formulation is the first once daily, extended-release liquid methylphenidate available for this patient population. The product is expected to be available in January 2013 in a 25 mg/5 mL strength suspension.
Cell Therapeutics announced that FDA has granted paclitaxel poliglumex (Opaxio) orphan drug designation for the treatment of glioblastoma multiforme. This new formulation is a biologically enhanced chemotherapeutic that links paclitaxel to a biodegradable polyglutamate polymer. When bound to the polymer, paclitaxel is inactive, potentially sparing normal tissue’s exposure to high levels of paclitaxel and its associated toxicities. However, blood vessels in tumor tissue, unlike blood vessels in normal tissue, are porous to macromolecules such as paclitaxel poliglumex. Based on preclinical studies, it appears that paclitaxel poliglumex is preferentially distributed to tumors due to their leaky blood vessels and trapped in the tumor bed. Once inside the tumor cell, enzymes metabolize the protein polymer, releasing active paclitaxel.