CSL889 (CSL Behring) was granted orphan drug designation from the European Commission and the U.S. FDA Office of Orphan Products Development for the treatment of sickle cell disease (SCD). CSL889 is an investigational, plasma-derived hemopexin for the treatment of acute vaso-occlusive crisis (VOC), a common painful complication of SCD. Hemopexin scavenges free heme from the vasculature, conferring protection against inflammation and oxidative stress. Hemopexin levels are often depleted in SCD patients and low levels of hemopexin have been associated with increased risk for acute VOCs. An open-label Phase 1 clinical study has been initiated to assess key safety and pharmacokinetic variables of CSL889 (NCT04285827). Enrollment is currently open at two treatment sites in the U.K. More info: CSL Behring press release

Novartis announced the approval of Adakveo (crizanlizumab) from the European Commission (EC), on the 30th of October 2020. Adakveo, is the first targeted sickle cell disease therapy for the prevention of recurrent vaso-occlusive crises (VOCs), or pain crises, in patients with sickle cell disease aged 16 years and older available for use in Europe. It can be given as an add-on therapy to HU/HC or as monotherapy in patients for whom HU/HC is inappropriate or inadequate. Clinical data showed that use of Adakveo led to a significant reduction in the rate of VOCs and decrease the duration of hospitalization. VOCs disrupt patients’ lives physically, socially, emotionally and can increase risk of organ damage and early death. Therefore, preventing the sudden, unpredictable and life-threatening VOCs is hugely important, said Kees Roks, Head Region Europe, Novartis Oncology, emphasizing the importance of Adakveo approval from EC. Adakveo, is now approved in 36 countries around the world including the United States and European Union member states For more information, see here.

Bluebird's LentiGlobinTM for sickle cell disease (SCD) gene therapy (bb1111) was granted eligibility to the Priority Medicines (PRIME) program by the European Medicines Agency (EMA). LentiGlobinTM is an investigational therapy. PRIME provides early and enhanced scientific and regulatory support to medicines that have the potential to significantly address patients’ unmet medical needs. Clinical data from the completed Phase 1/2 HGB-205 study, the ongoing Phase 1/2 HGB-206 study and ongoing long-term safety and efficacy follow-up study LTF-303 supported the PRIME application for LentiGlobinTM for SCD. The U.S. FDA granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobinTM for SCD. LentiGlobinTM for SCD also received orphan medicinal product designation from the European Commission for the treatment of SCD. For more information, see here.

Imara's IMR-687 is an oral disease-modifying treatment for sickle cell disease (SCD) and β-thalassaemia. IMR-687 is a highly selective and potent small molecule inhibitor of PDE9. Blocking PDE9 acts to increase cyclic GMP levels, which is associated with reactivation of foetal haemoglobin, or HbF, a well-established modifier of disease severity. Having received Orphan Drug Designation by the FDA for IMR-687 for treatment of β-thalassaemia earlier in the year (June 2020 press release), Imara announces dosing of the first β-thalasaemia patient in the Forte Phase 2b clinical trial (NCT04411082). Multiple preclinical studies show that treatment with IMR-687 enhances both the maturation and production of red blood cells in β-thalassaemia. This is exciting news towards the development of β-thalassaemia therapeutics as there are currently no approved oral therapies to increase HbF in β-thalasaemia. Read more on Imara's press release.

The Sickle Cell Disease (SCD) Implementation Consortium (SCDIC) was created to address the gap in the implementation of evidence-based interventions for SCD into clinical care and to develop a longitudinal registry of patients with SCD. The SCDIC is a cooperative research program composed of eight academic/clinical sites and one data coordinating center, and is supported by the National Institutes of Health through the National Heart, Lung, and Blood Institute (NHLBI) and the National Institute on Minority Health and Health Disparities (NIMHD). An article published in the July 15 issue of the Orphanet Journal of Rare Diseases describes the development of the SCDIC Registry, making available the registry baseline for SCD and first follow-up data collection forms and REDCap data dictionary for other SCD research efforts. Source: OJRD