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The European Commission has authorized the marketing of Hizentra (human normal immunoglobulin) as the first and only subcutaneous immunoglobulin (SCIg) for maintenance therapy in patients with chronic inflammatory demyelinating polyneuropathy (CIDP), after stabilization with immunoglobulin by intravenous (IVIg). The approval is based on data obtained from the phase III PATH study (Polyneuropathy And Treatment with Hizentra), the largest randomized placebo-controlled clinical trial in patients with CIDP. The results of the PATH study showed that, following the switch from intravenous to subcutaneous therapy, the rate of disease recurrence and exit from the study for any other reason was significantly lower among patients treated with subcutaneous (39% in the low dose therapy group of Hizentra [0.2 g / kg per week], 33% in the high dose therapy group [0.4 g / kg per week]) compared to the placebo group (63%).

Gemtuzumab ozogamicin given in combination with daunorubicin and cytarabine, has been shown to prolong the life span of patients for up to 8 months before the cancer has returned to manifest. This is the motivation that led the European Commission to authorize the marketing of the drug for the treatment of acute myeloid leukemia (AML). This is the European opinion although some side effects of gemtuzumab ozogamicin may be serious. Gemtuzumab ozogamicin is authorized for patients over the age of 15 without prior treatment for AML. The drug is used in patients whose tumor cells have CD33 protein on the surface (as is the case for most patients with acute myeloid leukemia). It is not used instead for acute promyelocytic leukemia (APL). AML is “rare” and gemtuzumab ozogamicin has been defined as an “orphan medicinal product” on 18 October 2000. Gemtuzumab ozogamicin should be administered by intravenous infusion over 2 hours. The patient usually receives 3 infusions over a week along with daunorubicin and cytarabine. If the tumor responds to the initial treatment, the patient may receive an additional “consolidation” treatment to prevent the tumor from recurring. The active substance consists of two parts: a cytotoxic substance and a monoclonal antibody. As the antibody binds to CD33 on leukemic cells, the cells absorb the antibody and the cytotoxic substance attached to it. Once inside the cells, calicheamicin is released exerting its cytotoxic effect. A main study involving 271 patients with acute myeloid leukemia (AML) has shown that the addition of gemtuzumab ozogamicin to daunorubicin and to citarabine can prolong the life span of patients by approximately 8 months without the tumor returning. For patients who received gemtuzumab ozogamicin in combination with daunorubicin and cytarabine, an average of 17.3 months passed before treatment failed, before the tumor returned or before the patient died, compared to 9.5 months for patients who received a combination of only daunorubicin and cytarabine. The most common side effects with Mylotarg given in combination with daunorubicin and cytarabine are bleeding and serious infections.

Recently have been published the results of two separate clinical trials of two years each showing the potential of gene therapy with LentiGlobina to eliminate or reduce chronic blood transfusions in patients with β-thalassemia transfusion-dependent. Both studies, Northstar (HGB-204), recently completed, and HGB-205, currently underway, are aimed at evaluating the safety and efficacy of a single-dose treatment of gene therapy based on LentiGlobin and whose interim results showed that most of the 22 patients in the two Phase 1/2 trials followed for two or more years remained transfusion-free. From a safety point of view, the collected profile was consistent with myeloablative conditioning and the busulfan chemotherapeutic agent. The recently completed Northstar study is a multicentre, open-dose, single-dose, non-randomized, Phase 1/2 study designed to assess the safety and efficacy of LentiGlobin in the treatment of patients with TDT. HGB-205 is an ongoing, single-dose, non-randomized, monocentric, single-dose, open-label, 1/2 study designed to assess the safety and efficacy of LentiGlobin in the treatment of patients with TDT and severe sickle cell anemia (SCD). The HGB-205 Phase 1/2 study is conducted in a single center in France. It is designed to evaluate the safety and efficacy of LentiGlobin in the treatment of subjects with TDT and SCD. Seven subjects were enrolled in the study. The intention of the manufacturer is to present, at the end of this year, an authorization request for the LentiGlobina for β-thalassemia transfusion-dependent to the EU.