Sarepta falls after gene therapy maker pauses Elevidys shipments in US
The U.S. Food and Drug Administration on Friday asked Sarepta to voluntarily halt shipments of the gene therapy, but the company refused to do so and said it would continue making the treatment available to ambulatory patients.
But Sarepta has now voluntarily and temporarily paused all shipments of the therapy, effective end of Tuesday. The company said the decision would allow it to respond to requests from the FDA and maintain a positive relationship with the agency.
"Though Sarepta claims its pause in shipments was voluntary, we think the company moved preemptively — bracing for an inevitable FDA mandate to pull the drug," said H.C. Wainwright analyst Mitchell Kapoor.
Shares of Sarepta fell 6.9% to $12.40 in early trading on Tuesday.
"Sarepta's decision to comply was a capitulation after recognizing that the fight with the FDA was not winnable and that the agency would have strong-armed Sarepta into removing Elevidys with a formal demand," Kapoor added.
Investor concerns regarding the use of the company's treatments have been heightened after the company on Friday disclosed that another patient who had received its experimental gene therapy died from acute liver failure, marking the third death this year.
The company's shares have plummeted 89% so far this year.
On Monday, Children's Hospital Los Angeles said it had paused usage of Elevidys in all patients with muscular dystrophy.
The FDA's initial request had likely heightened the pressure on physicians to pause treatments, which could also have been a driving factor in Sarepta's decision, William Blair analyst Sami Corwin said.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
The Independent
5 minutes ago
- The Independent
Weight-loss jab Wegovy sees another prescription boost after ban on copycats
Novo Nordisk's weight loss drug, Wegovy, is experiencing a surge in new prescriptions following a US ban on its copycat versions, though analysts caution that more substantial growth is needed to reassure investors. Since the US Food and Drug Administration (FDA) implemented a ban on 'compounded' versions of Wegovy on 22 May, new prescriptions for the drug have risen by approximately 33 per cent. This culminated in 181,200 new prescriptions in the week ending 18 July, according to IQVIA data shared with Reuters by industry analysts. The overall increase in Wegovy prescriptions is also narrowing the market lead held by Eli Lilly and Co's rival drug, Zepbound. While Zepbound prescriptions outpaced Wegovy by nearly 175,000 in the US during the week ending 23 May, this gap had reduced to around 133,000 by 18 July. Early signs of a shift come at a critical juncture for Novo. After Wegovy's initial stunning success, investor confidence was rattled when Zepbound and compounders started to slow the drug's growth. And then in May, the Danish company cut its full-year sales and earnings forecasts and announced the surprise exit of Chief Executive Lars Fruergaard Jorgensen, citing market challenges and a stock price nearly 60% below its 2024 peak. Jorgensen said at the time he expected the FDA compounding ban to lift sales in the second half of this year. Investors are keen to hear whether that is still likely when the company reports quarterly earnings on August 6. That leaves the drugmaker in what Barclays analyst Emily Field called a "show me" phase - no longer buoyed by optimism about a turnaround but under pressure to deliver. She has an "overweight" rating on Novo's shares. "We thought this trajectory change (in new prescriptions) would get the shares moving, but not so far," Berenberg analyst Kerry Holford told Reuters. "I suspect investors are now waiting for the (second quarter) update - will they/won't they trim guidance range? I think they will trim the top." Berenberg has a "hold" rating on Novo. Novo did not respond to a request for comment. Investors and analysts note that the IQVIA prescription data is incomplete, because it does not capture sales of Wegovy through the company's direct-to-consumer platform, NovoCare, launched in March. They estimate the channel is still a small slice of overall Wegovy volume. "We would hope to see strong growth from the NovoCare channel but are cognizant that this may come at initially lower prices" due to discount offers, said Marcus Morris-Eyton, a portfolio manager at Alliance Bernstein, whose fund holds Novo shares. "Sentiment towards Novo Nordisk is currently exceptionally weak, but given low expectations, the low valuation and hopefully accelerating prescription data in (the second half of 2025) we believe the market is underestimating Novo's long term growth potential," he said. Booming sales of Wegovy catapulted Novo to become Europe's most valuable listed company, peaking in June 2024 at about 615 billion euros, after the weekly injection became the first highly-effective obesity treatment approved in the U.S. in 2021. But supply disruptions and gaps in health insurance coverage for Wegovy helped fuel the market for cheaper compounded - or copycat - versions, which are allowed under U.S. law when drugs are in shortage. Investor sentiment on Novo soured after the company lost ground to U.S. rival Eli Lilly, which launched Zepbound in late 2023. Capturing more of the patients who had turned to compounded drugs is key to meeting targets. The FDA determined that Wegovy was no longer in shortage in February, leading to the ban on compounded versions. Novo has rolled out new tactics to bring patients back, including a limited-time discount for one month's supply, and secured better coverage from insurer CVS Health. Analysts at Berenberg and UBS are sceptical these moves will provide enough momentum and expect that Novo will reduce or narrow its full-year guidance ranges, according to research notes. TD Cowen said the outcome was hard to predict due to limited visibility on NovoCare. Bank of America and Guggenheim said they did not expect another guidance cut and Barclays said it was unlikely. ($1 = 0.8513 euros)
Daily Mail
6 minutes ago
- Daily Mail
Trump's 'mysterious condition' resurfaces during Scotland visit
Donald Trump once again had a mysterious tan makeup caked on his hand Sunday during his trip to Scotland. The splotched hand appeared just weeks after The White House revealed that Trump has been diagnosed with 'chronic venous insufficiency.' Earlier this month, Trump, 79, appeared at a White House press gaggle with what looked like makeup covering a patch on the back of his hand, sparking concerns. White House Press Secretary Karoline Leavitt revealed the president recently 'noticed mild swelling in the lower legs' and was evaluated by the White House medical unit. Leavitt said he underwent a 'comprehensive examination' which included 'diagnostic vascular studies.' 'Bilateral, lower extremity ultrasounds were performed and revealed chronic venous insufficiency,' Leavitt stated. She said it's a 'common condition' in individuals over the age of 70 and there was 'no evidence' of deep vein thrombosis or arterial disease. 'The president remains in excellent health,' Leavitt said overall. The president has spent the past few days in Scotland meeting with everyone from UK Prime Minister Keir Starmer to European Commission President Ursula von der Leyen. The makeup covering has been visible in multiple press photos throughout Trump's trip. The makeup appeared to be concealing a raised circular area of skin that Trump's team may have been eager to hide. The splotch has been visible on other occasions throughout the past month, sparking a new round of questions about the cause. Earlier this month, in a surprise statement during a press briefing, Leavitt told reporters the president was checked out by his doctor after noticeable 'swelling.' Leavitt said in response to a question that there was 'no discomfort from the president at all.' She then pointed to his daily activities. 'And you probably all see that on a day-to-day basis, he's working around the clock. As for the treatment, I can defer to the president's physician,' she said. The letter released by White House Physician Sean Barbarella, DO, references 'mild swelling' in Trump's lower legs, and states the diagnosis emerged after a series of vascular studies. Neither Leavitt nor the letter addressed whether Trump would have to modify any of his routines, which include frequent golfing at his private courses on weekends, and his trademark dance moves to 'Y.M.C.A.' when he addresses rally crowds. Leavitt said there was 'no discomfort' for the president at all. Officials have previously mentioned Trump's frequent handshaking – Trump regularly interacts with a large number of people at White House events and when he travels – as the cause of the bruising, but have not previously identified the aspirin issue. The bruising had stayed with Trump from the presidential campaign through his return to the White House. According to the Cleveland Clinic, chronic venous insufficiency occurs when leg veins are damaged, making it more difficult for blood to return to the heart. It 'causes blood to pool in your leg veins, leading to high pressure in those veins.' The condition is fairly common, affecting one in 20 adults, and the risk increases with age. It can lead to achy legs, a feeling of 'pins and needles,' cramping, swelling and itching, as well as 'Leathery-looking skin on your legs.' Up to half the people who have had deep vein thrombosis later develop post-thrombotic syndrome within a year or two. That relates to scar tissue that can develop after a blood clot. Trump was the oldest person to take the Oath of Office when he was sworn in in January at age 78. He has said repeatedly that his predecessor Joe Biden, 82, had no idea what he was doing and this week opened a probe into an alleged 'cover-up' through his use of an autopen for a series of commutations. Trump reposted an item in May calling Biden a 'decrepit [expletive],' days after Biden got a prostate cancer diagnosis. Trump has made a point of demonstrating his own physical and mental vigor – taking question after question from reporters inside the Oval Office, sometimes speaking for up to an hour.
BreakingNews.ie
6 minutes ago
- BreakingNews.ie
Tariff of 15% ‘challenging' but avoids a trade war
A tariff rate of 15 per cent is 'challenging' but avoids a rate of 30 per cent – which would have 'closed the market' in the US, The Minister for Enterprise has said. Peter Burke said that the EU-US deal avoids both a trade war and EU counter-measures, which would have had an effect on the north-south economy. Advertisement He said 'the devil is in the detail' of the trade agreement finalised on Sunday by Donald Trump and European Commission president Ursula von der Leyen in Scotland. 'We had a lot of modelling carried out on the various different options, and some were very perverse, that would have closed the market if you had over a 30 per cent tariff with a stacking mechanism,' Mr Burke told RTÉ Radio. 'The key thing is that there will be a number of carve outs. Obviously, aviation has been cited as zero-for-zero, but also in relation to agrifoods and potentially spirits.' The bloc is set to face 15 per cent tariffs on most of its goods including cars, semiconductors and pharmaceuticals entering the US and 'zero for zero' tariffs on a number of products including aircraft, some agricultural goods and certain chemicals – as well as EU purchases of US energy worth 750 billion dollars (€643 billion) over three years. Advertisement Mr Burke said it was his understanding that the 15 per cent tariff on the pharmaceutical sector would be a maximum rate. He added: 'I think the president of the Commission has been very clear that 15 per cent will be a ceiling.' It is still unclear from the deal, agreed five days before Mr Trump's threat of a 30 per cent tariff would have come into effect, will mean Ireland will need to invest in US energy, he added. 'This all has to be worked out yet, as you can appreciate, I'm only hearing this for the first time last night, and we have nothing on paper.' Advertisement Ireland's premier Micheal Martin and deputy premier Simon Harris welcomed the agreement struck on Sunday, saying that while Ireland 'regrets' the baseline tariff of 15 per cent, it welcomed the certainty for businesses. Mr Harris said further detail was needed around how tariffs would affect sectors including pharmaceuticals. Ireland remains vulnerable to a slow down in trade with the US economy, due to exports of products such as alcohol, dairy and beef. The Government has also expressed concern at how tariffs could affect pharma multinationals based in Ireland, which employs about 45,000 people in Ireland, as Mr Trump had signalled he intended to target that industry. Advertisement In addition, 65 per cent of all aircraft are leased through Ireland globally. Last week, Minister for Finance, Paschal Dohonoe said the Government would spend €9.4 billion euro on its budget in October, based on a zero-tariff scenario for next year. He and Minister for Public Expenditure, Jack Chambers said these estimates would need to be revised if there was a shock to the Irish economy. Mr Burke said it was not naive to base the government's economic scenario on a zero-for-zero trade agreement with the US. Advertisement 'No it wasn't because we didn't know what we were to be faced with,' he said on Monday. 'We do need to find out what happens in other areas, because this is very complex. 'It depends what happens with China, that's a very significant market that a deal hasn't happened yet. Ireland Hospital overcrowding: Over 450 patients on trolle... Read More 'It really impacts what happens with our exporters here in Ireland as well, because so much product is in danger of being redirected into EU market. 'We also don't know what separate carve outs are going to emerge for the different sectors that are so vulnerable from an Irish perspective. 'Until we get flesh on the bones and all those areas over the coming weeks, we'll be in a better position then to really put forward what budgetary parameters (we) will end up with.'
