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The National Institute for Health and Care Excellence (NICE) has recommended elosulfase alfa for routine use in the NHS for the treatment of mucopolysaccharidosis type 4A (also known as MPS 4A and Morquio A syndrome). Elosulfase alfa, also called Vimizin and made by BioMarin, becomes the first disease modifying treatment recommended by NICE for routine NHS use for people with this rare, severely life-limiting condition. NICE's final draft guidance follows the collection of 'real-world' data from 69 people who received NHS-funded treatment since 2015 as part of a managed access agreement. Clinical trial evidence and data collected as part of the managed access agreement, along with expert clinical opinion, shows some long-term benefits with elosulfase alfa treatment which suggest it slows progression of MPS 4A.

The National Institute for Health and Care Excellence (NICE)'s independent committee has called for more research to ensure liposuction for treatment of chronic lipoedema is safe and effective enough for use on the NHS. Lipoedema is more prevalent in women and very rarely affects men. It is characterised by an abnormal, usually symmetrical, accumulation of fat in the legs, hips, buttocks, and occasionally arms. It is a separate condition to obesity and lymphoedema. The cause of lipoedema is unknown, but hormonal changes, weight gain and genetics are thought to be involved. The size and shape of legs, and the resultant mobility issues and pain, can have a profoundly negative effect on quality of life, and physical and mental health. The interventional procedures advisory committee reviewed evidence from several sources, including eight before-and-after studies. The committee was informed that several different liposuction techniques are used and that they may have different safety and efficacy profiles.

Amgen Inc on Monday agreed to buy Horizon Therapeutics Plc in a deal valued at $27.8 billion (£22.67bn), fortifying its rare diseases portfolio in the biggest buyout in the sector this year. The company will pay $116.50 in cash, a premium of nearly 20 per cent to the stock's last close, for each Horizon share. Horizon shares closed up 15 per cent on Monday at $112.36. They had climbed 23.5 per cent through Friday since the company disclosed in late November it was in preliminary talks with Amgen, Sanofi and Johnson & Johnson for potential offers. Amgen shares dipped less than 1 per cent to close at $276.78. Sanofi said Sunday it ended discussions with Horizon after concluding the transaction prices "do not meet our value criteria." J&J also said in early December that it had dropped its pursuit of the deal.

he Healthcare Distribution Association (HDA) and the Pharmaceutical Services Negotiation Committee (PSNC) have urged the NHS England and the Department of Health and Social Care (DHSC) to issue urgent communications requesting that all those involved in medicines supply do not hoard, stockpile or over-prescribe Strep A antibiotics. In a statement HDA said that the sudden spike in demand for antibiotics used for the treatment of Strep A has meant that there is not enough of these medicines in the supply chain currently to meet this increased demand. As a result, wholesalers are working extremely hard with manufacturers to increase the supply of antibiotics. It added: "As regards pricing, the prices charged to pharmacies by HDA wholesale distributors will directly reflect the increase in prices wholesalers are having to pay for these medicines from manufacturers at the moment, in order to be able to continue supplying these medicines to pharmacies. This will be the case until supply and demand are more in sync."

The Department of Health and Social Care (DHSC) has allowed pharmacists to supply alternative penicillin to treat Strep A. It has issued Serious Shortage Protocols (SSPs) for three penicillin medicines. "The SSPs will help mitigate local supply issues of oral penicillin and allow pharmacists to supply alternative forms of the medicine if they do not have the specific formulation stated on the prescription," said DHSC. SSPs have been issued across the UK for 3 penicillin medicines on Thursday (December 15. "Issuing an SSP allows pharmacists to legally supply a specified alternative medicine, removing the need for the patient to return to the prescriber - which saves time in GP practices and inconvenience for patients." "Demand for penicillin has risen recently as it is used to treat strep A and scarlet fever, and the increased demand means that some pharmacists are experiencing temporary and localised supply issues and may not have the specific formulation listed on the prescription."

Erectile dysfunction is a very common form of medical condition that occurs in males over time. Usually, this problem is found in people who take too much medication, and ED is probably a side effect of any one of them. However, in seventy-five percent of males, the cause of erectile dysfunction is unknown, and finding the accurate reason for it is very complex. It can be a result of any neural complications, diabetes, or any surgeries that include the prostate of our body. ED can also be treated by using a vacuum device and surgeries on the private parts. However, it can be eradicated completely without any surgeries, and here are some tips that will help you in overcoming erectile dysfunction without surgery and lead a happy life. DO EXERCISE Exercise is a great way to overcome any health issues, and also to safeguard yourself from health issues in the future. Physical activity also helps you in controlling obesity, which is one of the reasons behind ED. By not doing regular exercises, you expose yourself to various cardiovascular problems, which contribute to ED. Studies show that light aerobic exercise for 30-40 minutes a day for at least four times a week for at least 6 months has considerably reduced ED in people. STAY SLIM Studies have shown that a person who has a waist near 42 inches is more likely to suffer from ED than a person with a waistline of 32 inches. Therefore, if you are obese it's time that you lose some weight. Excessive fat in the body is the birthplace of various diseases. Obesity is also responsible for vascular problems and diabetes which are two primary causes of ED.

British pharmaceutical giant GSK said on Saturday (April 15) its oral antibiotic drug to treat uncomplicated urinary tract infections (uUTI) in female adults and adolescents met the main goals in late-stage trials. The drug, gepotidacin, in phase III trials, met its primary goals of being on a par or better than nitrofurantoin, the current standard of care for the treatment of uUTIs, the company said. GSK plans to submit results of the trials to the United States Food and Drug Administration for review later in the second quarter. The drug, if approved, could become the first new type of antibiotic, which is critically important for countering drug-resistant infections, to treat uUTIs in 20 years, the company said. "Gepotidacin, if approved, will offer a much-needed additional oral treatment option for patients at risk of treatment failure associated with resistance or recurrence of uUTI," Chris Corsico, senior vice president of development at GSK, said.

British drugmaker AstraZeneca says it's confident that its new version of COVID-19 antibody treatment could protect immunocompromised patients against all known virus variants. Laboratory studies show the antibody, called AZD3152, neutralises all known variants of COVID-19 and AstraZeneca has support from regulators to make the treatment available by the end of this year, the company's vaccines head Iskra Reic said on Tuesday (April 18). AstraZeneca plans, pending more positive data and regulatory approval, to make the antibody available by the end of 2023. These types of therapies are most needed for people with compromised immune systems, either because of underlying conditions or because they are undergoing immune suppressing treatments. They account for nearly 2% of the global population. AstraZeneca's AZD3152, it new COVID-19 antibody, was acquired through a $157 million deal last year with British biotech start-up RQ Bio. The British drugmaker will likely make future investments like its current partnerships with RQ Bio but did not have any deals to announce, said Reic, a long-time AstraZeneca executive who has led the company's vaccines and immune therapies unit since it was formed in late 2021, during the pandemic.

Hospitals and healthcare providers across the UK have been notified about the shortage of an anti-inflammatory medicine caused by a manufacturing failure. The Department of Health and Social Care (DHSC) on Friday (3 May) issued a National Patient Safety Alert stating that Erelzi 50mg solution for injection in pre-filled pen will be out of stock from early May until mid-July 2024. "The supply issues have been caused by a manufacturing failure resulting in delays in production and the implementation of global allocations," it said. However, it noted that homecare providers (Alcura, Healthnet and Sciensus) have sufficient stock to supply existing patients until mid/late May 2024. Although Erelzi 50mg pre-filled syringes (PFS) are still available, they cannot support a full increase in demand, the DHSC warned.

American pharmaceutical giant Pfizer has received marketing authorisation from the Medicines and Healthcare products Regulatory Agency (MHRA) for its medication Etrasimod, marketed as Velsipity, used to treat people with moderately to severely active ulcerative colitis. Ulcerative colitis (UC) is a type of inflammatory bowel disease (IBD) that causes inflammation and ulcers (sores) in the lining of the large intestine (colon) and rectum. The approved recommended dose for Velsipity is one 2 mg tablet to be taken once daily. For the first three days, it is advised to take the medication with food, and subsequently, it can be taken daily either with or without food. According to the regulator, the approval of the medication is based on evidence from two clinical studies, which showed that 26 per cent of patients taking etrasimod achieved clinical remission after 12 weeks of treatment compared with 11 per cent of those receiving placebo. The studies included more than 740 patients aged 16 years and over for whom standard treatment or other treatments did not work well enough or could not be used.

NHS England's decision to forgo commissioning puberty suppressing hormones (PSH) for children and young people, citing insufficient evidence of their safety and clinical effectiveness, follows a comprehensive review conducted by the National Institute for Health and Care Excellence (NICE) in 2020. The clinical report underscores that "PSH treatment options will not be part of routine commissioning" for people under the age of 18 in England grappling with gender incongruence or dysphoria. The NHS England review analysed nine observational studies on Gonadotrophin Releasing Hormone Analogues (GnRHa) and found no statistically significant difference in gender dysphoria, mental health, body image, and psychosocial functioning among children and adolescents treated with GnRHa. Duncan Rudkin, chief executive of the General Pharmaceutical Council (GPhC), acknowledged the challenges faced by pharmacy professionals regarding prescriptions for puberty suppressing hormones, stating:

London's Great Ormond Street Hospital and Alder Hey Children's Hospital in Liverpool are poised to introduce groundbreaking gender-related services in England, following the closure of the Gender Identity Development Service (Gids) operated by the Tavistock and Portman NHS Foundation Trust. Amid concerns expressed by hospital executives regarding the closure of Gids, with leaked emails revealing apprehensions about appointment cancellations and inadequate communication with the new services, the National Health Services England (NHSE) has confirmed "its commitment to ensuring continuity of care for patients already accessing endocrine care in GIDS." NHSE's decision to transition to regional services stems from a 2022 interim review, led by Dr. Hilary Cass, which highlighted the need for improved support structures for gender-distressed youth. It emphasized a significant increase in referrals to Gids, surpassing 5,000 in 2021/22, compared to less than 250 a decade ago.

Kanabo Group plc, a medical cannabis research and development company, has entered into a partnership with City Dock Pharmacy in London to introduce a walk-in clinic for pain management. The pain clinic will cater to patients who are eligible for alternative therapies (when traditional treatments have failed to deliver improvements), and will offer specialised medicines, including medicinal cannabis. When it opens, the new facility at City Dock Pharmacy in Wapping will become the UK's first walk-in clinic for pain management, but it will also deliver appointment-related services, the companies said. The partnership is part of the London-based company's wider ambition to extend its reach into physical pharmacies throughout the UK, and its plans to replicate this model across other pharmacies.

The King's condition is "benign" and he will be treated in the hospital next week for a "corrective procedure", Buckingham Palace said a statement released on Wednesday. "In common with thousands of men each year, the King has sought treatment for an enlarged prostate," the Palace said. Because of the upcoming surgery, Charles had to postpone all meetings and events planned for this week. The Palace informed that the King's public engagements will be "postponed for a short period of recuperation." Charles, 75, wanted to share the details of his diagnosis to encourage more men who may be experiencing symptoms to get their conditions checked.

A newly licensed 1mg/ml Omeprazole Powder for Oral Suspension has been developed by Rosemont Pharmaceuticals to meet the special needs of babies from 1 to 12 months old who have gastro-oesophageal reflux disease (GORD). In infants, the symptoms of GORD can be particularly distressing for both the child and parent. Previously, Rosemont Omeprazole Powder for Oral Suspension has been available in 2mg/ml and 4mg/ml strengths. However, the addition of the new 1mg/ml product ensures that the needs of all patient groups with GORD are sufficiently met. The Rosemont range of Omeprazole Powder for Oral Suspension products are the only PPIs (proton pump inhibitors) indicated for use in babies from 1 month to 1 year. Symptoms of GORD in infants are usually resolved by 1 year of age1, so it is key that there is a licensed treatment option available for the 1 to 12 month patient group.

A newly licensed 1mg/ml Omeprazole Powder for Oral Suspension has been developed by Rosemont Pharmaceuticals to meet the special needs of babies from 1 to 12 months old who have gastro-oesophageal reflux disease (GORD). In infants, the symptoms of GORD can be particularly distressing for both the child and parent. Previously, Rosemont Omeprazole Powder for Oral Suspension has been available in 2mg/ml and 4mg/ml strengths. However, the addition of the new 1mg/ml product ensures that the needs of all patient groups with GORD are sufficiently met. The Rosemont range of Omeprazole Powder for Oral Suspension products are the only PPIs (proton pump inhibitors) indicated for use in babies from 1 month to 1 year. Symptoms of GORD in infants are usually resolved by 1 year of age1, so it is key that there is a licensed treatment option available for the 1 to 12 month patient group.

The Medicines and Healthcare products Regulatory Agency (MHRA) on Monday (29 January 2024) approved the medicine Rezzayo (rezafungin) to treat invasive candidiasis, an infection caused by a yeast called Candida. Invasive candidiasis can pose a serious health risk as it can affect the blood, heart, brain, eyes, bones and other parts of the body. Napp Pharmaceuticals Ltd was granted the marketing authorisation for the medicine based on phase 3 clinical trial data. In the randomised, double-blind, controlled study, more than half of the invasive candidiasis patients who were given a weekly dose of rezafungin were cured at day 14. Among 94 patients who were given a daily dose of caspofungin (another antifungal treatment), 57 of them were cured during the same time.

Cancer patients in the UK are being given a new immunotherapy treatment at Imperial College Healthcare NHS Trust as part of a global phase 1/2 clinical trial, which aims to evaluate its safety and potential for treating 'solid tumour' cancers such as melanoma and lung cancer. The experimental therapy, called mRNA-4359, has been designed to train patients' immune systems to recognise and fight cancer cells, according to researchers at Imperial College London. For the first time in the UK, cancer patients received the treatment at the National Institute for Health and Care Research (NIHR) Imperial Clinical Research Facility at Hammersmith Hospital. In this non-randomised trial, mRNA-4359 is administered to patients either alone or in combination with an existing cancer drug called pembrolizumab, a type of immune checkpoint inhibitor. The researchers are hopeful that this new therapeutic approach, if proven to be safe and effective in clinical trials, could lead to a new treatment option for difficult-to-treat cancers.

As the population continues to age, the number of dementia cases in the UK could increase to rise to 1.5 million by 2040, a new study has predicted. The study report from Future Health also cautioned that over half a million people with dementia could remain undiagnosed by 2040, representing an increase of nearly a quarter of a million compared to the current figure, if there is "no progress in improving diagnosis rates." At present, approximately 850,000 individuals in the UK are living with dementia, with an estimated 315,000 cases going undiagnosed. Sponsored by Lilly, the study also highlighted significant variations in diagnosis rates across the four nations. Despite improvements, England's dementia diagnosis rate stands at 64.6 per cent, falling short of the two-thirds target.

In the next 10 years, the incidence of first-time stroke cases in the UK is projected to increase by 60 per cent, costing the government £75bn in healthcare and lost productivity, a charity has suggested. A new manifesto published by the Stroke Association has urged the next UK government to make stroke "the priority", stressing that the next decade is crucial for stroke prevention, treatment and recovery. Currently, stroke is the UK's fourth biggest killer and a leading cause of disability. On a daily basis, 281 individuals experience a stroke. Next year, the cost of stroke in the UK will be £43 billion. The charity predicted that by 2035, there will be 151,000 hospital admissions due to stroke every year, averaging 414 admissions per day, with 42,000 people estimated to die every year. In 2035, the UK is projected to have 2.1 million stroke survivors, imposing a financial burden of £75 billion on the public purse, nearly half the current NHS budget.