The Committee for Medicinal Products for Human Use (CHMP) is recommending the conditional approval of Adakveo (crizanlizumab) for sickle cell disease (SCD) be revoked in Europe. According to the therapy’s developer Novartis, a final decision from the European Commission is expected in about two months. In the…

Treatment with Adakveo (crizanlizumab) did not reduce the yearly occurrence of painful vaso-occlusive crises (VOCs) leading to a medical visit, as compared with a placebo, in people with sickle cell disease (SCD), according to preliminary data from an ongoing Phase 3 trial. The study, called STAND (NCT03814746), is…

Precision BioSciences and Novartis are teaming up to develop and commercialize a custom gene therapy as a potential treatment approach for certain inherited blood disorders, including beta thalassemia and sickle cell disease (SCD). Under their new exclusive license and collaboration agreement, the companies will combine Precision’s proprietary ARCUS genome…

Novartis is partnering with the American Society of Hematology (ASH) to roll out an app in six sub-Saharan African countries to track babies who receive a diagnosis of sickle cell disease (SCD) through newborn screening. The app is already in use in Ghana, where it works to collect…

Adakveo (crizanlizumab), by Novartis, is now available on the U.K.’s National Health Service (NHS) for sickle cell disease (SCD) patients 16 and older. The new treatment, which is delivered by an into-the-vein infusion, can be used alone or as an add-on to hydroxyurea to help reduce the frequency of…

Novartis and the Bill and Melinda Gates Foundation are collaborating to develop a single-shot gene therapy that would cure sickle cell disease (SCD) without the need to engineer cells outside the body. The partnership aligns with Novartis’ efforts to better treat SCD, and the Gates Foundation‘s…

Iron overload is a side effect that some sickle cell patients suffer from as a result of blood transfusion programs being used as long-term treatment plans. Back in September, I wrote about trying a new medication to address my iron overload problem. For the past two years,…

The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent vaso-occlusive crises (VOCs) in people, 16 and older, with sickle cell disease (SCD). The approval covers the use of Adakveo in combination with hydroxyurea (also known as hydroxycarbamide) — another approved therapy…

Iron overload is a common side effect for sickle cell patients participating in blood transfusion programs. When this happens, the body’s excessive iron levels can cause an accumulation of iron in the body’s organs, which can lead to serious organ damage. I was diagnosed with iron overload…

A European Medicines Agency (EMA) committee has adopted a positive opinion on Novartis‘s Adakveo (crizanlizumab) for the prevention of vaso-occlusive crises (VOCs) in people with sickle cell disease (SCD). The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended the conditional approval of…