Latest news with #Kaftrio
Daily Mirror
4 days ago
- Health
- Daily Mirror
'Worst ever' drug shortage leaves cancer patients unable to eat
Shortages of Creon, which replaces a key enzyme needed to digest food, means patients with pancreatic cancer, cystic fibrosis and chronic pancreatitis are rationing meals Thousands of cancer and cystic fibrosis patients are being hit by a critical shortage of a key drug they need to help them digest food. Pharmacists are warning some patients are only eating once a day to ration supplies of the drug Creon. Patients with chronic pancreatitis are also among around 60,000 people who rely on the drug which replaces a key enzyme in what one pharmacist called the "worst stock shortage" they have ever had to deal with. The National Pharmacy Association surveyed 300 pharmacies and found 96% were having trouble supplying Creon, which is subject to the Government's serious shortage protocols (SSP). Creon is a pancreatic enzyme replacement therapy (Pert) which helps people digest food and absorb its nutrients. A Europe-wide problem in getting hold of stocks is being caused by a shortage of its raw ingredients. Patients like Harriet Corr, 9, from Hebburn, Tyneside, who suffers from Cystic fibrosis are being advised to basically cut back on their calories to reduce the amount of Creon tablets they need. People with CF struggle as it is to keep weight on so this can be dangerous. Speaking last year, her mum Emma Corr told the Express: Emma, of Hebburn, Tyne and Wear, told the Daily Express: "Harriet needs about 10-15 Creon tablets every day and usually it's never a problem getting any. But people increasingly seen unable to source Creon anywhere. It's becoming a full blown crisis. We're now unable to find it either. "We've been scouring our house to see if we've left any pots of Creon lying about - that's how desperate we're getting. Not only does Creon help Harriet absorb the nutrients and fats in her food but it enables her CF drug Kaftrio to work properly too. If we have to reduce the fats in her diet not only will she lose weight but it could mean her Kaftrio does not fully work either." The NPA survey heard examples of patients in distress such as those eating one meal a day to ration their Creon, going without medication or travelling long distances and contacting numerous pharmacies in an attempt to find stock. The NPA said some are travelling more than 30 miles to get hold of a pack of the drug. Some 89% of pharmacies also reported difficulties in supplying Pert alternatives. Olivier Picard, chairman of the NPA, said: "Pharmacies are at the sharp end of medicine shortages, spending hours hunting stock and supporting frustrated and concerned patients. "As this distressing survey shows ongoing supply problems with Creon has had a profound effect on the patients who depend on it to survive and lead a normal life. It simply cannot be right that in the 21st century patients are skipping meals in order to ration their medication.' Alfie Bailey-Bearfield, head of influencing and health improvement at Pancreatic Cancer UK, said: "These deeply worrying findings echo the distress and frustration we are hearing from patients and their loved ones across the UK. "Thousands of people affected by pancreatic cancer rely on taking Pert tablets every time they eat simply to digest their food and absorb nutrients - something most of us take for granted. It's totally unacceptable that they are still taking desperate measures which puts their health, wellbeing and their eligibility for treatment at risk.' The Government has extended the current serious shortage protocols (SSP) in place for Creon until November. This allows pharmacists to supply a reduced quantity of Creon capsules that might be in stock, without having to send a patient back to their GP for a new prescription. The NPA survey also found that 81% of pharmacies felt the current arrangements for managing shortages of Creon were inadequate. It found pharmacists are still having to refer patients back for new prescriptions for an alternative Pert product, stock permitting, or an unlicensed Creon product. Olivier Picard, of the NPA, added: "Although we recognise this situation is complex and not the fault of the Government, it's important they convene a taskforce and a national action plan to tackle this particular shortage given its impact on patients. "Medicine shortages are all too common so highly trained pharmacists should also be permitted to use their professional judgment to supply alternative medicines - where it is safe and appropriate - in the event of the prescribed version being unavailable." Alfie Bailey-Bearfield, of Pancreatic Cancer UK, added: "For over a year, we have been raising concerns with Department of Health and Social Care officials and Pert suppliers, urging them to prioritise solutions that reduce the impact on patients. But despite these conversations, progress has been far too slow and patients continue to pay the price. "It is critical that they take immediate, decisive action and explore every available option to increase supply, including directly purchasing this vital medication from countries with a surplus. This crisis continues to put people's health on the line, and they cannot afford to wait any longer. The situation is unacceptable and it demands immediate action." A Department of Health and Social Care spokesman said: "We know how frustrating and distressing medicine supply issues can be for patients and the clinicians caring for them. "The European-wide supply issues with Creon are caused by a limited availability of raw ingredients and manufacturing capacity constraints. We are working closely with industry and the NHS to mitigate the impact on patients and resolve the issues as quickly as possible."
Yahoo
06-05-2025
- Business
- Yahoo
Why Did Vertex Pharmaceuticals Stock Fall On Tuesday?
On Monday, Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) reported lower-than-expected first-quarter 2025 earnings. Vertex reported first-quarter adjusted earnings per share of $4.06, down from $4.76 a year ago, missing the consensus of $4.32. The cystic fibrosis-focused company reported sales of $2.77 billion, missing the consensus of $2.85 billion. Total revenue increased by 3%, primarily driven by the continued performance of Trikafta/Kaftrio and an early contribution from the U.S. launch of Alyftrek. In the U.S., total revenue increased 9% to $1.66 billion due to continued strong patient demand and higher net realized pricing. Outside the U.S., total revenue decreased 5% to $1.11 billion, as strong patient demand in established and newer markets was offset by the expected revenue decline in Russia, where Vertex is experiencing a violation of its intellectual property rights. Also Read: Vertex Discontinues Development Of Type 1 Diabetes Candidate After Disappointing Data 'Vertex delivered a strong start to 2025 with notable execution across the business as we grow and diversify the revenue base, progress multiple launches and advance the R&D pipeline. We continued to expand our leadership in CF and build global momentum for CASGEVY, and we launched JOURNAVX in moderate-to-severe acute pain,' said Reshma Kewalramani, CEO and President of Vertex. 'With multiple programs in pivotal development including povetacicept, which continues to make rapid progress in achieving its potential as a pipeline-in-a-product, and additional programs in early and mid-stage development, Vertex is poised to continue to deliver value for years to come.' Guidance: Vertex raised the low end of total revenue guidance by $100 million to $11.85 billion-$12 billion from $11.75 billion-$12 billion, compared to the consensus of $11.97 billion. The guidance also includes an immaterial cost impact from tariffs in 2025 based on currently known tariff rates and regulations. Vertex has temporarily paused the multiple ascending dose portion of the Phase 1/2 study of VX-522, a nebulized CFTR mRNA therapy, to assess a tolerability issue. William Blair writes, 'While the pause is unfortunate, we believe that the decision to pause the study rather than discontinue it suggests that, beyond the tolerability issue, management is encouraged by the data generated to date given it is open-label.' Analyst Myles Minter writes, 'While there was a slight miss on the top line leading to shares trading off 3% in the aftermarket, management raised the lower end of total revenue guidance, suggesting Street estimates may not have fully encompassed the previously disclosed impacts of an unauthorized Trikafta copy in Russia. We would be buyers on weakness here as the CF franchise still represents one of the most stable long-term growth franchises with a competitive moat in the biotech sector.'
Yahoo
26-04-2025
- Health
- Yahoo
'Swimming mirrors my cystic fibrosis treatment'
A man who was told he would not live until 30 is to celebrate his birthday doing a lake swimming challenge. Adam Ball, who lives with cystic fibrosis which affects his ability to breathe, will swim the length of 13 lakes in the Lake District over the summer. Mr Ball, who is from South Shields, said swimming did "wonders for his lungs" and the sport had an "uncanny resemblance" to his treatment. "To get to my 30th birthday and feel like I'm in a good position with my cystic fibrosis, I wanted to do something to celebrate that with a purpose," he added. Cystic fibrosis is the most common inherited life-threatening condition in the UK. It affects the ability of cells to transport salt and water, which can cause sticky mucous to build up. It can affect several organs but especially the lungs and the digestive system. Mr Ball was diagnosed with the condition from birth, which he said "always had its challenges" but he has had "amazing support" from his parents and Newcastle RVI. "When I was born, my parents were told life expectancy was 30, so getting to this age potentially wasn't going to be the case." Mr Ball said along with taking 70 pills and other nebulizers, one of treatments had been a positive expiratory pressure mask, which creates pressure to rid mucus from his lungs. "Using the mask alone would take an hour and a half on a daily basis," he said. "But with swimming, I've found it has an uncanny resemblance to the mask. "After I swim, it just feels so nice on your lungs and it's easier to breathe." He said taking a so-called "miracle drug" Kaftrio had "enabled him to get the most out of life" too. Mr Ball will aim to swim across all 13 publicly accessible lakes in the Lake District, covering a total of 71km (44 miles) while raising funds for Cystic Fibrosis Trust. He said he could become the first person with the condition to complete the challenge. "It's almost as if I've been training for this challenge my entire life from having the PEP mask. He said swimming brought him "so much joy". "It's the one thing where you just switch off from everything and it's so therapeutic." Vanessa Newton, from Cystic Fibrosis Trust, said Mr Ball's challenge was "incredible". "Money raised helps us provide emotional, physical, and financial support to everyone with cystic fibrosis and their families, fund lifesaving research, and work towards a future free from the limits of cystic fibrosis." Follow BBC North East on X, Facebook, Nextdoor and Instagram. Swimmer completes 13 lakes in three days I thought I'd die by 30, now I'm rowing the Atlantic Access to cystic fibrosis drugs approved in England Cystic Fibrosis Trust
BBC News
26-04-2025
- Health
- BBC News
South Shields man with cystic fibrosis to swim Lakes challenge
A man who was told he would not live until 30 is to celebrate his birthday doing a lake swimming Ball, who lives with cystic fibrosis which affects his ability to breathe, will swim the length of 13 lakes in the Lake District over the summer. Mr Ball, who is from South Shields, said swimming did "wonders for his lungs" and the sport had an "uncanny resemblance" to his treatment."To get to my 30th birthday and feel like I'm in a good position with my cystic fibrosis, I wanted to do something to celebrate that with a purpose," he added. Cystic fibrosis is the most common inherited life-threatening condition in the affects the ability of cells to transport salt and water, which can cause sticky mucous to build can affect several organs but especially the lungs and the digestive system. Mr Ball was diagnosed with the condition from birth, which he said "always had its challenges" but he has had "amazing support" from his parents and Newcastle RVI."When I was born, my parents were told life expectancy was 30, so getting to this age potentially wasn't going to be the case."Mr Ball said along with taking 70 pills and other nebulizers, one of treatments had been a positive expiratory pressure mask, which creates pressure to rid mucus from his lungs. "Using the mask alone would take an hour and a half on a daily basis," he said."But with swimming, I've found it has an uncanny resemblance to the mask. "After I swim, it just feels so nice on your lungs and it's easier to breathe." He said taking a so-called "miracle drug" Kaftrio had "enabled him to get the most out of life" too. 'So much joy' Mr Ball will aim to swim across all 13 publicly accessible lakes in the Lake District, covering a total of 71km (44 miles) while raising funds for Cystic Fibrosis said he could become the first person with the condition to complete the challenge."It's almost as if I've been training for this challenge my entire life from having the PEP mask. He said swimming brought him "so much joy"."It's the one thing where you just switch off from everything and it's so therapeutic." Vanessa Newton, from Cystic Fibrosis Trust, said Mr Ball's challenge was "incredible"."Money raised helps us provide emotional, physical, and financial support to everyone with cystic fibrosis and their families, fund lifesaving research, and work towards a future free from the limits of cystic fibrosis." Follow BBC North East on X, Facebook, Nextdoor and Instagram.
The Independent
11-04-2025
- Health
- The Independent
Cystic fibrosis patient turns brush with death into Edinburgh Fringe show
A Scottish cystic fibrosis patient who wrote goodbye letters to her family before a revolutionary drug saved her life has turned her ordeal into an Edinburgh Fringe comedy show. Yvonne Hughes, 51, from Clydebank in West Dunbartonshire, has lived with cystic fibrosis (CF) – a condition which causes sticky mucus to build up in the lungs and digestive system – her whole life after her diagnosis at just six weeks old. In 2018 she was told by lung experts she would not survive a transplant after her lung capacity had fallen to 26%. She was placed on oxygen to help her breathe and began making memories with her family, believing she did not have long left to live. She also wrote letters to her family begging them to look after her parents when she was gone. However in 2020, she received a life-changing cystic fibrosis drug called Kaftrio, which transformed her life and drastically improved her health. Since then she has returned to full-time work, is in a new relationship and pursuing a career in stand-up comedy. 'Life's amazing – I've gone from barely surviving to thriving,' Ms Hughes told the PA news agency. 'You have lived your life a certain way, and all of a sudden you can take a deep breath without having a massive cough, and fit.' Before she was offered the Kaftrio drug, Ms Hughes had moved in with her parents as her health had severely declined. 'I was exhausted, I was literally bent over, I couldn't walk, I couldn't really cook anymore,' she said. 'I just didn't have the breath in me anymore to do anything, and even my speech had to slow down. 'I had also already passed the life expectancy as I was hitting into my 40s. 'At the time, the life expectancy for people with cystic fibrosis was only 33 years of age.' Within an hour of taking the drug, which came in tablet form, she started to cough up and eject all of the phlegm that had accumulated in her lungs over the years. 'I went to bed that night, and I had the best sleep I'd had since, I don't know when,' she said. 'I woke up the next day and I had no cough at all.' Since receiving the life-changing treatment, the quality of Ms Hughes' life has improved drastically. 'I can walk up a hill now, I can breathe,' she said. Ms Hughes, who believes she has been given a second chance at life, has decided to take this opportunity to pursue her dream of becoming a stand-up comedian. 'I've tried to get out and do something that I feel was always inside me, but I've never been given the chance to do. I was always too sick to do stuff,' she said. 'I took up comedy to change my life, I'm still learning. I'm going for it this year because I want to be the person that I thought I always was.' Ms Hughes will debut her show, titled Absolutely Riddled, at Gilded Balloon Patter House at the Edinburgh Fringe Festival in August. 'It's based on life before and after taking Kaftrio and how I went from barely surviving to thriving, and I'm in my 50s, when I really shouldn't be here at all,' Ms Hughes said. Her show, which features a song about mucus, makes light of some of the less savoury elements of cystic fibrosis and promises to share an unapologetic and hilarious take on her life.
