Apadamtase alfa /Cinaxadamtase alfa

The CRISPR/Cas9 gene-editing therapy Casgevy offers a one-time curative option for beta-thalassemia patients. Image: Shutterstock/Perception7 Following positive guidance issued by the National Institute for Health and Care Excellence (NICE), the CRISPR-based gene therapy Casgevy has been made available to patients with transfusion-dependent beta thalassemia (TDT) in England. Today (8 August), NICE released their final draft guidance recommending Casgevy coverage via the NHS. Casgevy was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. The recommendation concerns patients aged 12 and older with severe beta-thalassemia needing blood transfusion, for whom blood and bone marrow transplant is suitable, but no matching donor is available. Consequently, Vertex announced a reimbursement agreement with NHS England making the treatment available beginning today. Casgevy will be accessible through the Innovative Medicines Fund for up to 460 eligible patients, as per NICE. The NICE decision came after an independent committee heard statements on how TDT impacts the lives of patients. TDT is a life-threatening inherited disease in which genetic mutation reduces or prevents production of healthy red blood cells and haemoglobin. The condition often manifests as intense fatigue; infants may suffer delayed development, misshapen bones, and enlarged organs. Treatment involves lifelong blood transfusions and iron chelation therapy. See Also: Takeda’s ADZYNMA gains EU approval for cTTP treatment Amneal’s CREXONT gains FDA approval for Parkinson’s treatment Stem cell transplant is a potentially curative option, but is limited by matched donor scarcity. This is especially true for people of South Asian ethnicity who are disproportionally affected and for whom matching donors are particularly rare, according to Dr. Diana Hernandez, a group leader at the medical nonprofit Anthony Nolan. Hernandez added that Casgevy also has potential against sickle cell disease – “Sickle cell largely affects patients from African and African-Caribbean backgrounds, who are less likely to have a matching stem cell donor, and this community has been waiting years for a new, effective treatment.” The Medicines and Healthcare products Regulatory Agency (MHRA) granted Casgevy the world-wide first authorisation for a CRISPR-based therapy in November 2023. The approval was for both TDT and sickle cell disease. At a list price of £1.65mn (~$2mn) per course, this reimbursement agreement has significantly increased availability for patients in England. Casgevy is a one-time therapy in which a patient’s haematopoietic stem cells are removed and edited through clustered regularly interspaced short palindromic repeats (CRISPR) technology that uses the Cas9 enzyme at the BCL11A gene. Modified cells are transfused into the patient, increasing foetal haemoglobin production in red blood cells without the risk of rejection. Vertex is also working with reimbursement authorities in the EU to make their therapy available to TDT and SCD patients, as has been done in the US , Saudi Arabia, and Bahrain. The company is poised to compete with bluebird Bio’s CRISPR-based SCD therapy Lyfgenia (lovotibeglogene autotemcel). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

cTTP is an ultra-rare, life-threatening blood clotting disorder caused by the ADAMTS13 enzyme deficiency. Credit: Kateryna Kon/Shutterstock. The European Commission (EC) has granted approval to Takeda for ADZYNMA (recombinant ADAMTS13) – the first and only enzyme replacement therapy in the European Union (EU) for congenital thrombotic thrombocytopenic purpura (cTTP). cTTP is an ultra-rare, life-threatening blood clotting disorder caused by the ADAMTS13 enzyme deficiency. ADZYNMA is approved to treat ADAM metallopeptidase with thrombospondin type 1 motif 13 (ADAMTS13) deficiency in children and cTTP in adults. The approval follows a positive opinion from the Committee for Medicinal Products for Human Use, announced on 31 May 2024. The EC’s decision was informed by comprehensive data from a Phase III trial and subsequent continuation study, demonstrating the efficacy and safety of ADZYNMA. See Also: Takeda’s ADZYNMA gains EU approval for cTTP treatment Amneal’s CREXONT gains FDA approval for Parkinson’s treatment In the pivotal Phase III trial, 45 patients treated with ADZYNMA did not experience any acute TTP events. One acute event was reported in 46 patients receiving plasma-based therapies. Additionally, one patient experienced a subacute TTP event when treated with ADZYNMA, compared to seven events in six subjects on plasma-based treatments. These findings were consistent in the continuation phase of the trial. ADZYNMA’s safety profile was also favourable when compared to plasma-based therapies, with the most common adverse reactions being diarrhoea, headache, dizziness, nausea, upper respiratory tract infection and migraine. Takeda is further evaluating recombinant ADAMTS13’s potential in treating immune-mediated thrombotic thrombocytopenic purpura (iTTP) in an ongoing Phase IIb trial. ADZYNMA also received approval from the US Food and Drug Administration (FDA) and the Japanese Ministry of Health, Labour and Welfare (MHLW) for the prophylactic and on-demand treatment of cTTP patients. Takeda Europe and Canada Business Unit president Ricardo Marek stated: “This approval marks the first treatment specifically indicated to address the root cause of the disease – ADAMTS13 deficiency. “Building on our 70-year legacy of innovation in rare blood disorders, we’re proud to offer ADZYNMA to cTTP patients in the EU and remain committed to bringing innovative medicines to rare disease patients with high unmet need.” In March 2024, Takeda secured approval from the MHLW for ADZYNMA Intravenous Injection 1500 (apadamtase alfa/cinaxadamtase alfa) to treat cTTP.