The U.S. Food and Drug Administration (FDA) has recently granted Orphan Drug Designation to Naproxcinod, a CINOD (Cyclooxygenase-Inhibiting Nitric Oxide-Donating) anti-inflammatory candidate, for the treatment of sickle cell disease (SCD) in the United States (US). The drug is developed by Nicox SA and exclusively licensed to Fera Pharmaceuticals in the US. Naproxcinod is an anti-inflammatory treatment that works by releasing nitric oxide and at the same time suppressing the activity of the enzyme cyclooxygenase, which is responsible for producing pro-inflammatory molecules. The treatment is designed to reduce pain and inflammation, and improve blood flow.
Nicox already completed a broad clinical program for naproxcinod in osteoarthritis, including three phase 3 studies with over 2,700 patients. Fera has conducted pre-clinical work on naproxcinod in models of SCD with promising findings furthering the development of the treatment for vaso-occlusive crises (VOCs), a frequent complication of SCD. More info: Fera press release, Sickle Cell Disease News


Be Aware, Share, Care: Working with the global community as one to improve thalassaemia knowledge’’, is the theme that Thalassaemia International Federation (TIF) announced for the International Thalassaemia Day (ITD) on 8 May 2022. The theme is an open call to action to all supporters to promote awareness about thalassaemia and its global impact and share key information and knowledge to support the best possible health, social and other care of patients with this disease. Furthermore. It seeks to inspire every individual to contribute, at the personal level, to the fight against thalassaemia and serves as a powerful reminder that everyone has a substantial role to play and a responsibility to act. More information about theme can be found here.

Global Blood Therapeutics, Inc. (GBT) announced the approve of Marketing Authorization for Oxbryta® (voxelotor) for the treatment of hemolytic anemia due to sickle cell disease (SCD) in adult and pediatric patients 12 years of age and older from the European Commission (EC). Oxbryta, given as a once-daily oral treatment, is the first medicine approved in Europe that directly suppresses the sickle hemoglobin (HbS) polymerization, the molecular basis of sickling and destruction of red blood cells in SCD. Eligible patients may take the therapy as monotherapy or in combination with hydroxycarbamide (hydroxyurea), a standard treatment to reduce the frequency of vaso-occlusive crises — episodes of acute pain caused by sickled red blood cells blocking blood flow — and the need for blood transfusions. Oxbryta demonstrated a favorable safety profile with limited and transitory adverse events compared with placebo groups. For more information: Global Blood Therapeutics, press release.

28 February 2018 marks the 14th edition of Rare Disease Day, coordinated by EURORDIS, aiming to raise awareness about rare diseases and their impact on patients' lives. The central message of this year's campaign "Rare is many. Rare is strong. Rare is proud" focuses on reframing the word 'rare', representing over 6000 rare diseases and affecting an estimated 300 million people worldwide. This year's campaign seeks to relay patient stories across diseases and borders through the dissemination of multilingual promotional materials for social media and the Rare Disease Day 2021 animated video, translated in thirty-seven languages. You can get involved on social media by using #RareDiseaseDay to share your colours with the rare disease community. More information: Rare Disease Day 2022

Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for Oxbryta® (voxelotor) tablets for the treatment of hemolytic anaemia due to sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older as monotherapy or in combination with hydroxyurea. Based on this CHMP opinion, a decision by the European Commission (EC), which authorizes marketing approval in the European Union, is expected in the first quarter of 2022. If approved by the EC, Oxbryta will receive marketing authorization in all EU member states and Iceland, Liechtenstein and Norway. Furthermore, GBT announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval of a supplemental New Drug Application (sNDA) for Oxbryta tablets for the treatment SCD in children ages 4 to less than 12 years. This approval expands the previously approved use of Oxbryta to treat SCD in patients ages 12 years and older in the United States. The FDA also approved GBT’s separate New Drug Application (NDA) for Oxbryta tablets for oral suspension, a new dispersible, once-daily tablet dosage form suitable for patients ages 4 to less than 12 years as well as for older patients who have difficulty swallowing whole tablets. Oxbryta is the first and only approved medicine that directly targets sickle hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. For more information see press releases here, here, and here.