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The European Union's health regulator on Friday said it had recommended granting a conditional marketing authorisation for a gene therapy by Australian drugmaker CSL Ltd and partner uniQure N.V. , to treat haemophilia B, a rare bleeding disorder which is caused by genetic anomalies. About one in 40,000 people are affected by the inherited disorder, caused by a gene mutation that hampers the body's ability to make clotting protein factor IX. If approved, the treatment, branded Hemgenix, will be the first gene therapy in the European Union for the condition that is usually treated by regular injections of factor IX, the European Medicines Agency said. CSL had acquired exclusive global rights to Hemgenix in May last year from uniQure N.V.

The new deal struck by the NHS will enable provision of a life-saving treatment for babies and young children who suffer with a rare and fatal genetic disease, metachromatic leukodystrophy (MLD). The revolutionary gene therapy treatment, known by its brand name Libmeldy, is used to treat MLD, which causes severe damage to the child's nervous system and organs, leading to a life expectancy of just five to eight years. Having a reported list price of more than £2.8 million, it is the most expensive drug in the world, but can now be offered to young patients on the NHS in England after the health service negotiated a significant confidential discount. the drug works by removing the patient's stem cells and replacing the faulty gene that causes MLD before then re-injecting the treated cells into the patient. The most common form of MLD usually develops in babies younger than 30 months and can cause loss of sight, speech and hearing, as well as difficulty moving, brain impairment, seizures, and eventually death.

The Medicines and Healthcare Products Regulatory Agency (MHRA) has authorised an innovative and first-of-its-kind gene-editing treatment for sickle-cell disease and transfusion-dependent β-thalassemia. Known as Casgevy (exagamglogene autotemcel), the treatment is based on the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. Casgevy is the first medicine to be licensed that uses CRISPR, and it is to be used for treating patients aged 12 and over. To date, a bone marrow transplant has been the only permanent treatment option for these life-long conditions, which in some cases can be fatal. The MHRA's authorisation came after a rigorous assessment of its safety, quality and effectiveness.

The Department of Health and Social Care (DHSC) launched a new Innovative Medicines Fund on Tuesday (June 7) under which £340 million has been made available to purchase potentially life-saving drugs early. This will allow NHS patients in England to have early access to potentially life-saving and cutting-edge treatments Health secretary Sajid Javid said: "I want NHS patients to be the first in the world to access the most promising and revolutionary treatments that could extend or save their lives. "The launch of the Innovative Medicines Fund delivers another manifesto pledge and will fast-track cutting-edge medicines to adults and children to give people renewed hope for a better future." A total of £680 million has been ringfenced for the Innovative Medicines Fund and Cancer Drugs Fund - £340 million each - to fast-track medicines to NHS patients. DHSC said: "The Innovative Medicines Fund will provide quick access to novel treatments, including potentially lifesaving gene therapies for serious conditions with few treatment options. It often takes longer for pharmaceutical companies to collect data on a medicine's clinical and cost effectiveness for rare diseases due to the smaller patient cohort.

Hundreds of children in England are set to benefit from a treatment for cystic fibrosis - Kaftrio, after the UK's Medicines and Healthcare products Regulatory Agency (MHRA) confirmed an extension to its licence. With the licence extension, more than 1,300 children in England with cystic fibrosis, aged six to 11, are newly eligible for this treatment, which improves lung function and improves overall quality of life of patients. Earlier, Kaftrio was only licensed for those aged 12 and above. British patients were the first in Europe to benefit from Kaftrio, when NHS England secured a landmark deal in June 2020. NHS chief executive Amanda Pritchard said: "Since NHS staff delivered one of the fastest rollouts of Kaftrio in the world just over a year ago, the lives of thousands of patients with cystic fibrosis have been transformed. "Innovative treatments like Kaftrio are life-changing for patients and their families, and that is why the NHS has done all it can since we secured the deal for Kaftrio to ensure patients benefit as soon as possible.

The drug, alpelisib, which is the 100th cancer drug that has being fast-tracked to patients under the NHS Cancer Drugs Fund (CDF) will be used in combination with the hormone therapy, fulvestrant, to target the gene that causes fast-growing tumours. Up to 3,000 people a year with a certain type of secondary breast cancer will benefit from the treatment. The approach has contributed to people in England having access to nearly one third more cancer drugs compared to the European average. The drug which is manufactured by pharmaceutical company Novartis, is part of a growing number of precision treatments that target a tumour based on mutations in its DNA and that the NHS is rolling out. John Stewart, NHS National Director for Specialised Commissioning said, "In just over five years, more than 80,000 people have benefitted from earlier access to a range of cancer drugs, with people in England having access to nearly one third more cancer drugs compared to the European average, and this latest innovative new treatment will help up to 3,000 more to live a better quality of life.

The government on Wednesday (March 2) announced an investment of £260 million to support research, development and manufacturing of new drugs, devices and diagnostics. Of the total funding, up to £200m has been allocated for research to better access NHS data through Trusted Research Environments and digital clinical trial services, enabling availability of crucial data with the highest levels of privacy. This will allow the NHS to deliver new life-saving treatments to patients, tackle health inequalities and improve patient care, a government release stated. The remaining £60m will support commercial-scale manufacturing investments by companies at the leading-edge of innovation, from cell and gene therapies and earlier and better diagnostic technologies, to medical devices. The funding for manufacturing investments will be distributed through the new Life Sciences Innovative Manufacturing Fund (LSIMF), following the success of the earlier Medicines and Diagnostics Manufacturing Transformation Fund.