Latest news with #Trikafta
NZ Herald
a day ago
- Business
- NZ Herald
Dan Carter on UNICEF's 'life-changing' work in Kiribati
Minister of Education Erica Stanford addresses the media. She's admitted being a little embarrassed by the snafu. Video / Mark Mitchell The New World Point Chevalier supermarket has opened three weeks early, after the fire at the Victoria Park location. Video / Michael Craig Chelsea Daniels gives Ryan Bride the lowdown on today's episode with business editor at large Liam Dann. Lorna Riley joins Ryan Bridge to discuss New Zealand as a top adventure destination, flight turbulence, and Sagrada Familia's long-awaited completion. Video / Herald NOW Technology journalist Peter Griffin joins Ryan Bridge to discuss Australia's move to ban social media for under-16s. Ryan Bridge speaks with Deborah Pead and Rawdon Christie on Trikafta funding, a child's burger venture, and Cape Palliser road closure proposal. Video / Herald NOW Hawke's Bay reporter Rafaella Melo joins Ryan Bridge to discuss a young entrepreneur's growing home burger business. Chris Penk, Building and Construction Minister on the latest developments around building consent. Video / Herald NOW Wildlife photographer Andy MacDonald on South Wairarapa District Council's plan to limit access to a popular North Island spot. 2Degrees Business- Garth Bray from BusinessDesk joins Ryan Bridge on Herald NOW. Video / Herald NOW Lawyer Minda Thorward on Sarah Shaw's release from a Texas detention centre after being held with her young son. Video / Herald NOW Labour Leader Chris Hipkins joins Ryan Bridge on Herald NOW. A Lower Hutt family relocates to access Trikafta for their 4-year-old daughter, aiming to prevent further irreversible damage from CF. Video / Herald NOW Herald NOW Weather: August 19, 2025.
NZ Herald
2 days ago
- Health
- NZ Herald
Kiwi family move to Australia to get cystic fibrosis drug Trikafta for daughter
Adalyn Delaney was suffering the effects of CF before she was even born, with health staff discovering during a scan that she had a bowel obstruction. The moment she was born, she had to be rushed away to surgery. Adalyn Delaney, 4, moved to Australia with her family, including 1-year-old Emily Delaney, to access Trikafta. Adalyn had to have two surgeries within her first five weeks of life, and another at the age of 1 to get scar tissue removed. She has been hospitalised multiple times, including for pneumonia, a collapsed lung and another bowel obstruction. She has to go on a nebuliser (a mask-like device that turns medicine into a vapour she can inhale) three times a day when she is healthy, and must take medication when she eats to help her body digest food properly. Before going on Trikafta, she went on antibiotics every time she got a cough. Since getting to Queensland in June, Adalyn was quickly prescribed Trikafta and has been on what she calls her 'special pill' now for several weeks. While it is still early days, the medication appears to be working well so far, with Adalyn experiencing a spike in energy and hunger, and a lack of tummy aches. Delaney said it was 'really heartbreaking' to leave behind their support networks, friends and family, but they had to make the decision for Adalyn's health. 'Children don't want to be strapped to nebulisers, they want to be playing and all that.' Delaney said it was disappointing it was 'taking so long' for the drug to be funded for young children in New Zealand. Adalyn Delaney calls Trikafta her "special pill". 'It's definitely a condition that affects things from birth ... the sooner the better that children can go on it for better long-term outcomes.' The family have thought about whether they would move home once Adalyn turns 6, but Delaney said Australia was funding more medications that may have a better impact on Adalyn. 'We want NZ to keep pace with treatments, not just catch up. We need to be where she'll get the best care.' 'This can change the trajectory of their lives' The chief executive of Cystic Fibrosis New Zealand, Lisa Burns, said New Zealand was 'just so far behind' other countries in providing Trikafta to children. Children were suffering irreversible damage to their bodies before the age of 6, she said. To the best of her knowledge, about 55 children in New Zealand would benefit from the drug being funded for them. She noted Pharmac's Pharmacology and Therapeutics Advisory Committee previously said Trikafta could add 27 years to someone's life. While previous estimates have put the average life expectancy in the 30s, updated figures in Australia, where more medications have been funded for longer, are closer to the mid-50s. Alex and Kayla Delaney moved their two daughters, Adalyn, 4, and Emily, 1, to Australia to access the drug for Adalyn. 'When children are normally learning to walk and explore, these kids aren't. They're going through hours of treatment regimes and having to spend time in hospital, and they don't get to have the same opportunities, and this medication could drastically help to improve that in this age group. It's so important,' she said. 'My message to Pharmac is please recognise the value that is being delivered from the patient population ... we want to see equitable, accessible treatment for our children. 'I just don't understand why we wouldn't do it, this can change the trajectory of their lives.' 'It was literally life-changing' Hanna Meates, 33, went on Trikafta a couple of years ago when Pharmac began funding it for people over 6. 'Everybody should be able to have access to something like that if it's going to literally save their life ... like, they can avoid all the lung damage and all of that stuff that's irreversible,' the Lower Hutt woman said. It was an opportunity for them 'to have a normal life right off the bat'. 'I think something like Trikafta is the most incredible medical breakthrough that we've had for cystic fibrosis.' Meates had never expected to have a long life, and upon starting Trikafta, her emotions were 'quite indescribable'. 'For the first time, I actually had a future to look forward to and a future that I could plan for and know that I was going to have that and be healthy for at least most of it. 'It was amazing but also scary in a way as well, because like, you grow up and you're told that ... you're not probably going to have a long life. So you live your life like that. Hanna Meates, 33, started taking Trikafta for her cystic fibrosis, and said the medication was life-changing. 'I didn't plan for anything. I don't have savings or anything. I didn't care about what was going to happen, so I was just living in the moment, and then suddenly you do have a future and then you have to start thinking about savings and what you're going to do for your future.' Meates said her symptoms were significantly reduced on Trikafta. She still experiences more shortness of breath than the average person and can get quite tired, but aside from that and a persistent cough, she otherwise feels healthy. 'You're one way your whole life and you don't really know what you're missing out on, and then suddenly your life just completely changes.' Before Trikafta, she couldn't exercise or hold down a full-time job, or even go for a light walk without running out of breath. Now she can go to the gym and live life relatively normally, and has a full-time job as an adviser in a government department. 'It was literally life-changing,' she said. Pharmac's director of advice and assessment, Dr David Hughes, said when they funded Trikafta for people over 6 in April 2023, Trikafta only had approval for that age group at the time. 'The company obtained Medsafe approval for Trikafta for children aged 2 to 5 years in March 2025, after supplying clinical trial data for this age group,' he said in a statement. A year ago, a respiratory advisory committee recommended Pharmac fund it with a high priority for people aged 2-6 who met certain criteria. 'Our teams are working hard to finish our assessment of this application, which is well under way. Once our assessment is complete, we will then compare this application against applications for other medicines that people would like us to fund on the Options for Investment list. 'We can't say if or when a decision about funding this medicine will be made. It depends on things like the budget we have available, the success of negotiations with suppliers, and how we've prioritised this medicine against others.' In a statement, Vertex, which makes Trikafta, said more than 40 countries, including those with a similar funding system to New Zealand, were funding the drug. 'Vertex is keen to explore innovative funding solutions with Pharmac so that eligible children ages 2-5 years can have sustainable access to Trikafta as soon as possible.' Melissa Nightingale is a Wellington-based reporter who covers crime, justice and news in the capital. She joined the Herald in 2016 and has worked as a journalist for 10 years.
Time of India
28-07-2025
- Health
- Time of India
Wadia doc helps 40 kids with genetic disease get 1cr/yr US drug free for life
Mumbai: Early Monday morning, 14-year-old Bhavin Khare and his mother, Tanuja, set off from Thane's Murbad for Wadia Hospital, Parel, around 100km away, with a sense of anticipation. They had to collect a US drug—estimated to cost over Rs 1 crore for a year's supply—that would help him control the debilitating symptoms of cystic fibrosis, a genetic disease he suffers from. "Bhavin has been struggling all his life. He manages to go to school only 50% of the days due to repeated cough and chest infections," Tanuja said, hoping the new drug would let him lead a "normal life". He is one of five children with cystic fibrosis who received a three-month supply of Trikafta, manufactured by US-based Vertex Pharmaceuticals, at the hospital Monday. Thirty-five others are scheduled to get it in the coming days. You Can Also Check: Mumbai AQI | Weather in Mumbai | Bank Holidays in Mumbai | Public Holidays in Mumbai "We have signed an MoU with the company which, on compassionate grounds, will provide a lifelong supply of Trikafta to 40 patients," said pulmonology department head Dr Parmarth Chandane, who communicated with the US company for almost a year. Due to patent laws, the drug isn't available in India and can only be imported. Affected children in wealthy nations have had access to the drug for almost eight years, and parents' groups in Karnataka, South Africa and Brazil have moved local courts seeking some mechanism like compulsory licensing to ensure their children get access to the drug, too. by Taboola by Taboola Sponsored Links Sponsored Links Promoted Links Promoted Links You May Like Libas Purple Days Sale Libas Undo "The active ingredients for the three-drug combination (Trikafta—elexacaftor/tezacaftor/ivacaftor) that is now being called a miracle, are made in India, but our children don't have access to the finished product," said a doctor who didn't want to be identified. The Wadia Hospital distribution is the beginning of an alternative access for Indian patients. "The drug has shown promise, with certain research reports claiming patients will be able to enjoy a normal lifespan of 80 years as against the 15-20 years one normally associates with the disease," said Dr Chandane, who communicated with the US company for almost a year, seeking access for the 80 patients registered at Wadia Hospital. The company, however, said it would only provide drugs for patients over six years of age, resulting in only 40 qualifying for the free-for-life supply. Each of the affected children has to take three drugs—two in the morning and one in the evening—daily to prevent the buildup of thick, sticky mucus that usually clogs organs such as the lungs, pancreas and intestines, which could further lead to malnutrition, poor growth, frequent respiratory infections, as well as chronic lung disease. It's learnt that Vertex Pharmaceuticals will provide lifelong supply of the drug to patients registered at CMC Vellore in Tamil Nadu, too. "We are also in talks with the company for our patients," said paediatrician Dr Indu Khosla from SRCC Hospital, Haji Ali. "By offering such costly and essential medication free of charge, we're removing financial barriers for families. These children are getting a chance to breathe easier, grow healthy and live longer," said Dr Minnie Bodhanwala, CEO of Bai Jerbai Wadia Hospital for Children.
Newsroom
07-07-2025
- Newsroom
A limit at last on how unreasonable public agencies can be with media
MediaRoom column: Public drug-buying agency Pharmac has conceded to the Chief Ombudsman that its conduct towards the former Today FM station and journalist Rachel Smalley 'fell below the standard expected of a public sector agency' and has apologised. Pharmac refused to give Today FM or owner MediaWorks interviews (and, briefly, any information) after its ex CEO claimed Today had breached an embargo on news it was starting consultation on approving the cystic fibrosis drug Trikafta in December 2022. But neither Smalley nor the station had been provided embargoed material, so could not have breached the embargo, and their attempts to reverse the Pharmac interview ban went unheeded for a week. Multiple attempts to set up interviews with the agency failed. Smalley complained to the Ombudsman, and more than two years on, on his last day in office, Chief Ombudsman Judge Peter Boshier found against Pharmac, ruling it 'acted unreasonably' in its actions against the media outlet. He said Pharmac's responses to Today FM lacked clarity on how to resolve matters and also the duration of its declared ban on interviews. To have banned Today from receiving 'information' could have inadvertently constituted a breach of the Official Information Act, the finding says. 'Any request for information must be considered under the OIA, and depending on the circumstances, a request for an interview may also amount to a request for information under the OIA.' The finding, while historic and specific, does serve as a message to other public agencies, executives and communications staff on what they cannot do to exclude or disadvantage journalists and news organisations without proper reason. Judge Boshier did not have to make any recommendations over his 'acted unreasonably' finding, he said. 'This is because Pharmac has accepted that Today FM did not breach its embargo, that there were alternative ways to resolve its concerns about the reporting. And it should not have imposed the stand-down. Pharmac will also be apologising to the complainant.' The apology to Smalley from its general counsel did not mention departed Pharmac CEO Sarah Fitt, who made the original ban threat and dealt with Today's complaints in the days following. Fitt had been revealed in late 2024 to have privately engaged in internal Pharmac email exchanges deriding Smalley and her show. Fitt finally left the role in May, after seven years, and the apology was sent to Smalley on Fitt's final day in the top job. Pharmac wrote: 'On behalf of Pharmac, I wish to apologise for the way our organisation previously engaged with you, including the events of December 2022. 'Pharmac's handling of Today FM's coverage of the Trikafta story was a regrettable error of judgment. Pharmac has accepted the Chief Ombudsman's finding of unreasonableness, with regards to the imposition of a stand-down period on MediaWorks and the failure to explain Pharmac's position clearly. 'Pharmac also accepts that: a. Today FM did not breach Pharmac's embargo, nor was the radio station subject to it; b. Pharmac should have explained its concerns to Today FM and not have attributed your personal tweet to MediaWorks; c. Normatively, the decision to impose a stand-down period and the manner in which it was imposed fell below the standard expected of a public service agency; and d. Pharmac's actions were exacerbated by subsequent internal communications which were unnecessarily hurtful and personal.' 'I wish to clarify that, despite the poor choice of words, Pharmac did not intend at any time to withhold official information from you or Today FM. What Pharmac failed to do was to clearly explain the reason for that refusal and invite you (or Today FM) to send your questions through another means,' general counsel Saar Cohen-Ronen wrote. 'Pharmac values your health advocacy work. I can only hope that the Ombudsman's report of today, and this letter of apology, will assist with turning a corner in our mutual work. 'We acknowledge that, while being held to account may not always be a pleasant experience, it is an essential feature of a healthy democratic society and a transparent public service which seeks to continuously improve. We encourage you to continue to engage with Pharmac and to keep advocating for a stronger healthcare system, to help make New Zealand a better place for us all.' Today FM closed in March 2023 after a year on air. Rachel Smalley was made a Member of the NZ Order of Merit in the King's Birthday Honours in 2024 for services to broadcasting and health advocacy. Is it a first strike for AI journalism before the Media Council? Illustration: Getty Images An unusual and low-level Media Council case involving the NZ Herald adding a summary above a syndicated RNZ story and introducing an error could well be a milestone in AI's involvement in journalism here. The council found the Herald had breached the principle of accuracy when it summarised the RNZ story on Cook Strait ferry costs wrongly. The judgment also criticised the Herald for redirecting the complainant to RNZ, and leaving its error in place on the story for two months. So far, so bad, but it falls more into the misdemeanour than felony category when it comes to Media Council breaches. The paper told the council the incorrect bullet point (which inflated the Government's Cook Strait ferry-building contract with Hyundai from $550m to $3 billion) was 'written by a NZ Herald digital producer.' Which is where things become mildly interesting. Within the Herald's own newsroom, there is suspicion the summary bullet points on such a story at that time (early March) would have been produced, first, by the editorial First Look AI tool. That programme runs through the text of a story and delivers the digital producer a ready-made set of summary bullet points. It was routinely used at the time and was a standard addition to syndicated stories from other media, such as RNZ. A staffer aware of the system says it's 'likely the false bullet points were produced by the in-house AI model'; another is 'fairly certain' AI was involved in the process. Whether in this case the digital producer either took it upon him or herself to write the points, or re-worked any of the AI suggestions, or published them as offered, might never be known. The Herald's round of editorial job losses through that period included a group of digital producers. Around the same time, the Herald home page curation was largely taken over by AI deciding what stories would appear where and for how long. The paper is sticking by its wording to the Media Council and will not comment further on the staff suspicions. It declined, to MediaRoom, to rule out, or rule in, AI. But if the bullet point error was generated by AI, and that error led to the Media Council upholding a complaint that its accuracy principle was breached, it would be a first. The council has not previously had to rule on AI tools' contributions to NZ journalism. Whether in this case it needed to know if First Look AI had been part of the process before the writings of the digital producer is now moot. Either way, the fact was wrongly highlighted in the summary and the Herald did correct it two months after publication when it finally worked out it was Herald and not RNZ bullet points containing the error. For the record, this is what the Media Council said on the breach: 'The original article conflated the ferry contract with the large increase in cost of the port upgrade work required. We agree with the complainant that the article incorrectly attributed the two elements as both having caused the blowout. 'The NZ Herald has unfortunately picked up and emphasised in some of the bullet points the erroneous report of a ferry cost blowout. It has failed promptly to correct the obvious errors even when they were immediately pointed out. 'The Media Council is not convinced that persisting with such an obvious error is justified by NZ Herald's reliance on the argument that it could make no changes to syndicated articles; and its confusion over authorship of the bullet points. 'The errors spotted by the complainant were added by the NZ Herald and were its responsibility. Once these errors were conclusively established, the NZ Herald had an obligation to correct such errors by promptly publishing a correction so that the NZ Herald's readers understood there had been a mistake.' Why Eerik the happy visitor disappeared Auckland Mayor Wayne Brown didn't like a video ad promoting Auckland and it turns out that was pretty much enough to sink it, despite $740,000 having been spent creating it for possible future uses. The 'Your Happy Auckland Guide' advertising campaign commissioned by development and promotions agency Tātaki Auckland Unlimited included video stories of Eerik from Finland, the happiest country on Earth, extolling the city's virtues and ran briefly last winter. It aimed to improve people's views of Auckland, and particularly the central city. The campaign was partially funded by money from the central city targeted rate. Newsroom published a story in July 2024 quoting Brown labelling it 'deeply embarrassing' and expensive. Although the ad campaign had been deliberately designed to be quirky and grab attention, the mayor was unhappy about so much having been spent 'on a joke'. He claimed the ad campaign ignored Auckland's vital selling points and showed all that was wrong about the system of council-controlled organisations. Eerik and his escapades at Mission Bay, K Rd and around the city apparently reached up to two million potential viewers during the first phase of the campaign. Eerik at Mission Bay. But the mayor's reaction, followed by applications by groups under the Local Government Official Information and Meetings Act for details of the costs, soon doomed future plans for the happy Finn. The whole Eerik affair has had to be documented by Tātaki Unlimited Auckland in a Business Value Proposition case study for the council's revenue, expenditure and value committee. The paper defends the ads and approach, saying to take a 'safer, less creative route would not have achieved cut-through, would be lost in the crowd and wouldn't maximise return on media investment.' Its case study shows that 'considering political criticism via the media, and the uncertainty of CCO reform through much of 2024, the decision was made to stop further activity phases' of the campaign. Tātaki Unlimited Auckland had worked with the council's marketing team and City Centre Programmes through the first quarter of 2024. Although it included details of the Eerik campaign to elected council members including the mayor in May 2024 in a regular newsletter, it had not personally briefed Brown until July (around the time the Newsroom story broke). His reaction publicly, plus the official information requests over the spending, and the CCO reform process, contributed to the campaign ending abruptly. The case study report says: 'In this instance, unrealised value' included 'a second phase of the project being withdrawn from agencies, with talent stood down … and plans with Finland for reciprocal social media and PR activity halted'. Looking forward, a case study section on what could be improved highlights 'political engagement'. 'An early briefing to the mayor's office, lead councillor and/or chair of the CCO oversight committee may mitigate negative political feedback via the media and establish support for marketing activity in advance.' It also says the ongoing reforms of CCOs – for example TAU lost its economic development function on July 1 to a new office within the council itself – could give clarity on the agency's 'remit, with political support, to promote Auckland and work at the top end of the sales funnel (i.e. sentiment)'. The case study says advance audience testing would give 'confidence that the creative solution is the right one (or not)' but would add cost and time to projects. It says the campaign was a success while it lasted, 'lifting sentiment towards Auckland'. 'Those who saw activity rated Auckland more favourably and were more likely to recommend Auckland as a place to visit with all measured perceptions scoring higher. 'Sixty percent took action and 87 percent took away key messages.' But it appears Mayor Brown was an important member of the 13 percent who did not. The 2025 domestic tourism campaign for autumn/winter, fully funded by $400,000 from the city centre targeted rate, is called 'It's on in Auckland' and plays into stereotypes about Aucklanders. The 2025 version of the autumn/winter domestic tourism promotion for Auckland. 'With tongue-in-cheek headlines and vivid imagery, the campaign sets out to surprise and delight,' TAU's press release says. 'Featured creative includes: 'You don't have to be a food snob to eat like one' 'Sure, it can get a little crowded here' (accompanied by a buzzing concert crowd) 'This place is full of exhibitionists' (a nod to a lively art scene) 'Of course there's drama in the big city' (with an image of an aerialist showing pure theatrical flair). For the record, the 2025 iteration was reviewed and approved by the mayor before launch in May via out-of-home (billboard) and social and digital channels. A second wave is proposed but not yet confirmed. In future, the new economic development office at the council, led by former TAU senior executive Pam Ford, will 'lead the destination strategy work in collaboration with Tātaki Auckland Unlimited', TAU says.
Yahoo
02-07-2025
- Business
- Yahoo
Vertex wins European approval for Alyftrek, bolstering cystic fibrosis stronghold
Vertex Pharmaceuticals has won approval in Europe for its next-generation cystic fibrosis therapy Alyftrek (deutivacaftor/tezacaftor/vanzacaftor), shoring up the company's dominance in treating the rare and progressive genetic disease. The approval by the European Commission (EC) follows a positive recommendation by the European Medicines Agency (EMA) in April. Alyftrek will be available for patients aged six years and older with cystic fibrosis who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Alyftrek will provide a revenue safety net as Vertex braces for a loss of patent protection for blockbuster Trikafta (elexacaftor/tezacaftor/ivacaftor). Trikafta, known under the brand name Kaftrio in Europe, is the best-selling cystic fibrosis drug in the world, generating revenue of $11.02bn in 2024. Kaftrio loses patient protection in 2037, at which point biosimilars are set to enter the market. Historically, this erodes the market dominance held by the branded drug. Alyftrek has patent protection until 2039. Alyftrek demonstrated the same efficacy in cystic fibrosis treatment as measured by a lung parameter when pitted against Kaftrio in head-to-head trials. However, the new triple combination therapy was superior in reducing sweat chloride, demonstrating greater improvement in CFTR function, according to Vertex. Along with an edge on certain measures of efficacy, Alyftrek has a dosing advantage. Unlike Kaftrio, which is taken twice daily, Vertex's new product is taken only once per day. Analysis by GlobalData's Pharma Intelligence Centre forecasts Alyftrek will bring in sales of $6.1bn by 2031. Whilst models do not extend to 2037 – the year Kaftrio loses patent protection – data shows that full year revenue will already start falling from 2025, largely due to Alyftrek picking up market share. The pharma company has a market cap of $116bn. Vertex's CEO Reshma Kewalramani said: 'Thousands of people with cystic fibrosis across the EU may now benefit from this new, once-daily medicine, which has demonstrated further improvement in CFTR protein function versus Kaftrio. 'With this approval, we are one step closer to our ultimate goal of restoring normal levels of CFTR function in people living with cystic fibrosis.' The US Food and Drug Administration (FDA) approved Alyftrek in December 2024, providing a window into market dynamics for the drug. Revenue for Alyftrek in its first full quarter of commercial availability in the US was $53.9m, below a William Blair estimate of $81.8m. In a research note following Vertex's Q1 results in May 2025, William Blair analyst Miles Minter said: 'We continue to expect the switch from Trikafta to Alyftrek to be slower than the switch rates seen following historical CFTR modulator launches in Vertex's portfolio, but we expect the company to see a successful switch over the long term and to benefit from midsingle-digit percentage margin improvements due to a favourable royalty structure with the Cystic Fibrosis Foundation.' 'Alyftrek also extends Vertex's CFTR modulator composition-of-matter patent protection from 2037 for Trikafta [Kaftrio] into 2039 for Alyftrek,' Minter added. "Vertex wins European approval for Alyftrek, bolstering cystic fibrosis stronghold" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
