Latest news with #cysticFibrosis
The Guardian
11 hours ago
- Health
- The Guardian
UK sickle cell patients ‘get worse care than sufferers of similar disorders'
People living with sickle cell disease face substandard care as its treatment significantly lags behind advances relating to other genetic disorders such as cystic fibrosis, a report has found. The study, commissioned by the NHS Race and Health Observatory and carried out by researchers at Imperial College London, analysed various measures of care for sickle cell disease between 2010 and 2024, including clinical trials, approved drugs and reviews of existing studies. The findings indicated that sickle cell care across the UK does not have parity with other genetic disorders, such as cystic fibrosis, with there being only 0.5 specialist nurses per 100 patients for sickle cell, compared with two per 100 for cystic fibrosis. The report also found that there is 2.5 times more research funding for cystic fibrosis than for sickle cell, meaning the former has more treatment options and breakthrough drugs than the latter. Evidence of substandard care for people with sickle cell was also found, with 20% of babies with the condition not being seen by a specialist by three months of age, despite the NHS screening programme guidelines that 90% of babies should be seen by this milestone. Prof Habib Naqvi, the chief executive of the observatory, said sickle cell care 'significantly lags behind' that for other rare genetic conditions. He added: 'These inequalities are stark and, despite being a common genetic disorder, sickle cell has endured years of inadequate attention and investment that has resulted in the experiences we then see play out for people living with the condition. 'We do highlight the stark inequalities that exist for people with sickle cell in comparison with other rare conditions, but we also offer evidence-based solutions for meaningful change.' The report also found that these disparities in care for people with sickle cell disease were not due to a lack of need, given that hospital admissions for sickle cell rose by 42% between 2013 and 2022. These disparities exist despite the fact sickle cell disease is just as prevalent as other rare genetic disorders. The disorder primarily affects people from an African-Caribbean background. In England, about 17,000 people are living with the disease, an inherited blood disorder, with 250 new cases a year. Sickle cell disease changes the shape of blood cells into crescents, hindering blood flow. People with the condition experience severe painful episodes, which can require hospital admission. Approximately 11,000 people in the UK have cystic fibrosis, a condition which causes breathing and digestive problems due to mucus. The median age of death for sickle cell is 47. For cystic fibrosis, it is 48. John James, the chief executive of the Sickle Cell Society, said the landmark report 'makes it impossible for anyone to ignore the existence of deep inequalities faced by people with sickle cell – in healthcare, research funding and wider recognition'. He added that the findings were sobering. 'It is unimaginable that these disparities have, beyond all reason, continued for so long. 'However, by analysing how other long-term conditions are supported and resourced, it is clear to see the tangible difference that investment in healthcare makes.' Prof Bola Owolabi, NHS England's director for the National Healthcare Inequalities Improvement Programme, said: 'It is unacceptable that people with sickle cell disease face worse outcomes than those with other conditions, which is why the NHS England has stepped up with its sickle cell and thalassemia quality improvement project. 'Patient care has also been boosted by introduction of digital care plans so they don't need to repeat their stories to healthcare staff, and we have launched a campaign to boost awareness of key signs and symptoms of the disorder called Can You Tell It's Sickle Cell?'
Yahoo
08-05-2025
- Business
- Yahoo
Why Vertex Pharmaceuticals Stock Is a Screaming Buy on the Dip
Vertex missed Wall Street's Q1 estimates and announced a pause with an early-stage study of its mRNA cystic fibrosis drug. However, the big drugmaker had plenty of good news in its Q1 update, too. This biotech stock should still generate exceptional returns over the next few years. 10 stocks we like better than Vertex Pharmaceuticals › Vertex Pharmaceuticals' (NASDAQ: VRTX) remarkable momentum came to a screeching halt this week. The big biotech stock had been up well over 20% year to date. However, Vertex's shares plunged after the company announced its first-quarter results on Monday. Should investors throw in the towel on Vertex? I don't think so. Instead, the sell-off presents a fantastic opportunity to double up on this big biotech innovator. The main bad news with Vertex's Q1 results was that the company missed Wall Street's revenue and earnings estimates. Vertex reported Q1 revenue of $2.77 billion, up 3% year over year. Analysts were expecting revenue of $2.83 billion. The drugmaker posted adjusted earnings per share of $4.06, below the consensus estimate of $4.29. Vertex also announced that it's temporarily pausing the multiple ascending dose portion of its phase 1/2 study evaluating messenger RNA (mRNA) therapy VX-522 in treating cystic fibrosis (CF). The company said the pause was needed "to assess a tolerability issue." Blame Russia for the weaker-than-expected financial results. Vertex noted that illegal copycat versions of its products in the country are negatively impacting sales. However, the company believes the problem is isolated to Russia. Importantly, Vertex increased the lower end of its full-year revenue guidance range, despite the intellectual property (IP) rights issues in Russia. The company now looks for 2025 revenue of between $11.85 billion and $12 billion. The lower end of its guidance range was previously $11.75 billion. As for the potential tolerability issue with VX-522, Vertex's management wouldn't provide any details because it's an active program. However, it's too soon to assume the worst. Even if there's more bad news, though, I don't think a setback with VX-522 changes the overall investment thesis for Vertex. Anytime a company misses Wall Street's quarterly estimates, it's tempting to focus only on the negatives. But smart investors know that they shouldn't overlook the positives. The reality is that Vertex had a lot of good news in its Q1 update, too. I'm going to start with the company's newest CF therapy, Alyftrek. Vertex CEO Reshma Kewalramani said in the Q1 earnings call that management is "pleased with the early launch dynamics and physician and patient feedback" for the drug. The company expects most U.S. patients who are on its other CF drugs will switch to Alyftrek over time. This cannibalization of existing products should be good for Vertex, though, because Alyftrek's royalty payments are lower than those of the company's other CF therapies. Vertex also picked up a positive Committee for Medicinal Products for Human Use (CHMP) opinion for Alyftrek. As a result, it's looking for European Commission approval for the drug in the second half of 2025. The company also hopes to win approvals in Australia, Canada, and Switzerland. I'm even more excited about the prospects for Vertex's new non-opioid pain drug Journavx. Investors shouldn't be disappointed that there was only "an insignificant amount" of sales from the drug in Q1. Journavx has only been available at retailers since mid-March. The reaction from payers, physicians, and pharmacies has been positive. Vertex expects volume to ramp up in the first half of the year, with revenue accelerating in the second half. Vertex also reported encouraging news about its pipeline programs. Kewelramani said the company should be in a position to file for regulatory approvals of zimislecel in treating severe Type 1 diabetes next year pending positive results from a pivotal study that's underway. She also noted that Vertex could file for U.S. accelerated approval of povetacicept in treating IgA nephropathy in the first half of 2026, assuming a positive interim analysis of data from the phase 3 Ranier trial. CFO Charlie Wagner confirmed Vertex's status as a safe haven from tariffs in the Q1 earnings call. He said: We expect an immaterial cost impact from tariffs based on what we know today due to our low exposure to China and a geographically diverse supply chain. The vast majority of our drug product manufacturing for CF is in the United States and most of our IP is concentrated in the U.S. and the U.K. Sometimes, a revenue and earnings miss is a sign of deeper problems for a company. I don't think that's the case whatsoever with Vertex Pharmaceuticals. This big biotech stock should easily generate exceptional returns over the next few years. Increased adoption of Alyftrek should boost profits, and Journavx is destined to become a huge blockbuster. Vertex's pipeline is poised to produce other big winners. I don't just think Vertex is a stock to buy on the dip -- I think it's a screaming buy. Before you buy stock in Vertex Pharmaceuticals, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and Vertex Pharmaceuticals wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $611,589!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $697,613!* Now, it's worth noting Stock Advisor's total average return is 894% — a market-crushing outperformance compared to 163% for the S&P 500. Don't miss out on the latest top 10 list, available when you join . See the 10 stocks » *Stock Advisor returns as of May 5, 2025 Keith Speights has positions in Vertex Pharmaceuticals. The Motley Fool has positions in and recommends Vertex Pharmaceuticals. The Motley Fool has a disclosure policy. Why Vertex Pharmaceuticals Stock Is a Screaming Buy on the Dip was originally published by The Motley Fool Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
08-05-2025
- Business
- Yahoo
VRTX Stock Down as Q1 Sales of New Drugs Miss Expectations
Vertex Pharmaceuticals Incorporated's VRTX first-quarter results were weak as it missed estimates for both earnings and sales. The company's total revenues of $2.77 billion rose 3% year over year, driven by higher sales of its triple combination cystic fibrosis (CF) therapy Trikafta/Kaftrio and an early contribution from the U.S. launch of Alyftrekt. Trikafta sales rose 9% in the quarter. While sales rose 9% in the United States, in outside U.S. markets, sales decreased 5%, hurt by the availability of an illegal copy of Trikafta in Russia, where Vertex is experiencing a violation of its intellectual property rights. Vertex raised the low end of its total revenue guidance by $100 million from $11.75-$12 billion to $11.85-$12 billion. Though Vertex revenues come mostly from its CF franchise, investor focus on the first-quarter call was on the performance of its newer drugs, which were approved in the past year. Vertex gained approval for two new products, its novel non-opioid pain medicine Journavx (suzetrigine) and its fifth CF medicine, Alyftrek, in the last few months. Vertex and partner CRISPR Therapeutics' CRSP one-shot gene therapy, Casgevy, was approved for two blood disorders, sickle cell disease and transfusion-dependent beta-thalassemia, in multiple regions in late 2023/early 2024. However, sales from its new drugs, Casgevy, Alyftrek and Journavx, fell short of investor expectations, which, coupled with the disappointing first-quarter results, led VRTX stock to decline 10% on Tuesday. Year to date, shares of Vertex have risen 11.8% against the industry's decrease of 2.2%. Image Source: Zacks Investment Research Let's dig deeper to understand how these new products performed in the first quarter and the company's outlook for the same through the rest of the year. Alyftrek (vanza triple), a next-in-class triple combination regimen for treating people with CF aged six years and older, was approved in the United States in December 2024 and in the United Kingdom in April 2025. Vertex's regulatory application for vanza triple is also under review in the EU and some other countries. In April, the European Medical Agency's (EMA's) Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion recommending approval of Alyftrek. Vertex expects Alyftrek's approval in Europe in the second half of 2025. This new once-a-day oral combination medicine has the potential for enhanced patient benefit over Trikafta and to become a new standard-of-care treatment in CF. It can potentially treat CF patients who have discontinued Trikafta or other Vertex CF medicines. It can improve dosing (once daily), lower the royalty burden and extend patent protection. The drug generated sales of $53.9 million in its first quarter of launch. Vertex said it is seeing a steady uptake from all patient groups who are eligible for treatment with Alyftrek, including new patients and patients looking to switch from Trikafta. However, the switch from Trikafta to Alyftrek was slower than expected. Alyftrek's sales fell short of most analysts' expectations. Journavx (suzetrigine), Vertex's non-opioid NaV1.8 pain signal inhibitor, was approved for the treatment of moderate-to-severe acute pain in January 2025. Vertex said Journavx's contribution to total revenues was 'insignificant' in the first quarter as the drug was launched in mid-March. However, many analysts believe that the Journavx launch progress has been slower than expected. Journavx sales are expected to pick up in the second half of the year as the product's uptake accelerates through patient assistance and supply/stocking initiatives in the first half. Vertex and partner CRISPR's one-shot gene therapy, Casgevy, contributed $14.2 million in sales in the first quarter compared with $8 million in the previous quarter. However, Casgevy sales also fell short of expectations of some analysts. The company now has more than 65 activated authorized treatment centers or ATCs in all regions where the therapy is approved. More than 90 patients have initiated cell collection. Vertex is also making rapid progress for the drug's access and reimbursement. Vertex expects Casgevy revenues to ramp up as the year progresses, as more patients are treated in geographies where the drug has secured regulatory approval and reimbursement. Vertex leads the global development and commercialization of Casgevy under the terms of the 2021 agreement with support from CRISPR Therapeutics. Vertex is rapidly advancing its diverse late-stage pipeline with four programs in pivotal development, including povetacicept added from last year's Alpine Immune Sciences acquisition. Vertex believes povetacicept has a 'pipeline in a product' potential. The other three candidates in pivotal development are suzetrigine in diabetic peripheral neuropathy, semelocell in type I diabetes and inaxaplin in APOL1-mediated kidney disease. Three of these phase III programs are on track to complete enrollment this year, setting the stage for several potential regulatory filings next year and potential new drug approvals in a couple of years. However, on the call, Vertex said it is temporarily pausing the multiple ascending dose portion of the phase I/II study of its mRNA therapeutic, VX-522, which it is developing in partnership with Moderna MRNA due to tolerability issues. In partnership with Moderna, Vertex is developing VX-522 for approximately 5,000 people with CF who do not make the CFTR protein and who cannot benefit from its CFTR modulators. A single ascending dose portion of a phase I/II clinical study on VX-522 is complete. Vertex currently carries a Zacks Rank #3 (Hold). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. Vertex Pharmaceuticals Incorporated price-consensus-chart | Vertex Pharmaceuticals Incorporated Quote Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Vertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis Report Moderna, Inc. (MRNA) : Free Stock Analysis Report CRISPR Therapeutics AG (CRSP) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
