NEW DRUG APPROVALS Blincyto for Rare Form of ALL

NEW DRUG APPROVALS Blincyto for Rare Form of ALL The FDA has approved blinatumomab (Blincyto, Amgen) to treat patients with Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia (B-cell ALL). Precursor B-cell ALL is a rapidly growing cancer in which the bone marrow makes too many B-cell lymphoblasts. The Philadelphia chromosome is an abnormality that sometimes occurs in the bonemarrow cells of leukemia patients. Blinatumomab is the first FDAapproved drug that engages the body’s T cells to destroy leukemia cells. The drug acts as a connector between the CD19 protein, which is found on the surface of most B-cell lymphoblasts, and CD3, a protein on T-cell lymphocytes. The treatment is meant for patients with relapsed or refractory cancer. The FDA granted Blincyto breakthrough therapy, priority review, and orphan drug status. Its efficacy and safety were evaluated in a clinical study in which 185 adults with Philadelphia chromosome-negative relapsed or refractory precursor B-cell ALL were treated with blinatumomab for at least four weeks via intravenous infusion. Thirty-two percent of the participants experienced complete remission for approximately 6.7 months. The product has a boxed warning noting that some clinical trial participants experienced cytokine-release syndrome at the start of the first treatment and brief encephalopathy or other adverse nervous system effects. The most common adverse events with blinatumomab included fever, headache, peripheral edema, febrile neutropenia, nausea, hypokalemia, fatigue, constipation, diarrhea, and tremor. The FDA approved Blincyto with a risk evaluation and mitigation strategy. Source: FDA, December 3, 2014 Lemtrada for Relapsing MS Alemtuzumab (Lemtrada, Genzyme/ Sanofi) has won FDA approval for the treatment of patients with relapsing forms of multiple sclerosis (MS). Because of its safety profile, however, it should generally be reserved for patients who have shown an inadequate response to two or more drugs indicated for MS treatment. The approval was based on two pivotal, randomized, phase 3, open-label, raterblinded studies that compared alemtuzumab with high-dose subcutaneous interferon (IFN) beta-1a (Rebif, EMD Serono/Pfizer) in patients with relapsing/remitting MS who were either new to treatment (CARE-MS I) or had relapsed on prior therapy (CARE-MS II). In both trials, alemtuzumab was significantly more effective than IFN beta-1a at reducing annualized relapse rates, which were 0.18 versus 0.39, respectively, in CARE-MS I—a 55% relative reduction— and 0.26 versus 0.52 in CARE-MS II—a 49% relative reduction. Alemtuzumab has a boxed warning for a risk of serious, sometimes fatal autoimmune conditions and a risk of serious and life-threatening infusion reactions. The label also notes that it may cause an increased risk of malignancies. The drug is available only through the Lemtrada Risk Evaluation and Mitigation Strategy. Lemtrada is administered via intravenous infusion on five consecutive days, with a second course of treatment on three consecutive days 12 months later. Alemtuzumab is a monoclonal antibody that targets CD52, a protein on T and B cells, which are thought to cause the damaging inflammatory process in MS. Common adverse effects include rash, headache, pyrexia, nasopharyngitis, nausea, urinary tract infection, fatigue, insomnia, upper respiratory tract infection, herpes viral infection, urticaria, pruritus, thyroid gland disorders, fungal infection, arthralgia, pain in an extremity, back pain, diarrhea, sinusitis, oropharyngeal pain, paresthesia, dizziness, abdominal pain, flushing, and vomiting. Serious side effects include autoimmune thyroid disease, autoimmune cytopenias, infections, and pneumonitis. Source: Genzyme Corporation, November 14, 2014