Latest news with #rareDiseases
Forbes
21-07-2025
- Health
- Forbes
The Big Beautiful Bill Fixes One Drug Problem—But Highlights An Even Bigger One
"It's a good thing that the Orphan Cures Act was added to the One Big Beautiful Bill Act," writes ... More health policy expert Sally Pipes. "But it would be better had the legislation not been necessary in the first place." Buried within the One Big Beautiful Bill Act, which President Donald Trump signed into law July 4, is a provision that could improve or even save the lives of the 30 million Americans suffering from rare diseases. That provision is the Orphan Cures Act, which exempts certain drugs that treat rare diseases from the scheme of price controls Democrats established in Medicare as part of the 2022 Inflation Reduction Act. The first round of price controls takes effect on ten medicines dispensed through the Part D drug benefit in January 2026. This exemption is common sense. Why should federal policy discourage companies from investing in drugs that treat rare diseases—defined as those that afflict fewer than 200,000 Americans per year—by threatening them with price controls? But that's exactly what the IRA did. Drugs that were approved for treating just one rare disease were carved out from the law's price controls. But if a manufacturer secured approval for a second indication for a given rare-disease drug, then it lost that exemption. That's obviously not in patients' interests. Shouldn't public policy encourage pharmaceutical companies to investigate whether their drugs have multiple applications? Thousands of rare diseases lack effective therapies of any kind. The IRA, wittingly or not, aimed to keep it that way. It's a good thing that the Orphan Cures Act was added to the One Big Beautiful Bill Act. But it would be better had the legislation not been necessary in the first place. The IRA's price controls stifle pharmaceutical innovation. The Incubate Coalition's Life Sciences Investment Tracker shows that drug companies have discontinued more than 50 research programs and canceled 26 drug projects since the passage of the IRA. And there's evidence that companies have been curtailing their research into orphan drugs, in particular. According to the National Pharmaceutical Council, 'the percentage of drugs with a first orphan designation that later received a second designation decreased by 48.0%' after the Inflation Reduction Act passed. That's especially shocking considering that, over the four years prior to the passage of the bill, the number of orphan drugs that received a second indication had been steadily increasing. Orphan drug research may be an especially risky proposition. But developing more mainstream drugs is hardly a blue-chip process. It costs $2.6 billion and can take more than a decade for companies to bring a single new drug to market. Roughly one in ten drug candidates successfully makes it from phase one clinical trials to the market. Drug development would almost certainly be a money-losing industry more often than not, were it not for the fact that pharmaceutical companies in America have a period of exclusive, market-driven sales during which they can earn a return on their investment. The money they earn from the drugs that do get approved allows them to recoup their losses from failed drugs and earn enough money to invest in future lines of research. Price controls upend that economic calculus. If drug makers know that the government will swoop in and set arbitrarily low prices on the products they manage to bring to market, they'll be discouraged from investing the billions it takes to do so. The IRA's price controls could lead to the development of 135 fewer drugs by 2039, according to one study. That disruption in innovation could cost the United States $18 trillion in 'health losses' in the same window, according to research from University of Chicago economist Tomas Philipson. The inclusion of the Orphan Cures Act in the One Big Beautiful Bill may ward off some of those potential losses. But making a single exemption to the IRA is not enough. Congress must roll back price controls on prescription drugs altogether.
Yahoo
07-07-2025
- Business
- Yahoo
BioCryst Pharmaceuticals Sells European ORLADEYO Business for Up to $264M
BioCryst Pharmaceutical Inc. (NASDAQ:BCRX) is one of the best high short interest stocks with huge upside potential. On June 27, BioCryst Pharmaceuticals announced a definitive agreement to sell its European ORLADEYO (berotralstat) business to Neopharmed Gentili for a total consideration of up to $264 million. The amount includes an upfront payment of $250 million for the European assets and rights related to ORLADEYO, along with up to an additional $14 million in future milestones contingent on sales in Central and Eastern Europe. The closing of this transaction is anticipated by early October this year. BioCryst plans to utilize the proceeds from this sale to retire all remaining term debt, amounting to $249 million from Pharmakon. A scientist in a lab coat observing a line of medicine pills in a container. This is expected to eliminate ~$70 million in future interest payments over the remaining life of the loan. Consequently, BioCryst projects to end 2027 with ~$700 million in cash and no term debt, representing an increase of $400 million compared to its previous net cash guidance for 2027. The acquisition by Neopharmed Gentili also involves taking over BioCryst's European organization, which is expected to result in at least $50 million in annual operating expense savings for BioCryst. BioCryst Pharmaceutical Inc. (NASDAQ:BCRX) is a biotechnology company that develops oral small-molecule and injectable protein therapeutics to treat rare diseases. Neopharmed Gentili is a pharmaceutical company founded in Italy. While we acknowledge the potential of BCRX as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey.
The Guardian
06-07-2025
- Health
- The Guardian
Patients with ultra-rare diseases worry new FDA rules will leave them without treatment
US drug regulators have increasingly signaled a focus on faster approvals and rare diseases, but patients with ultra-rare ailments fear they are falling through the cracks, especially given challenges to conducting clinical trials. One drug, elamipretide, garnered a narrow recommendation from independent advisers for the US Food and Drug Administration (FDA), but the agency rejected the drug's application in May and recommended another potential pathway for approval. Patients and advocates worry about new rules on who may receive the medication during this process, and whether the drug will reach approval before the pharmaceutical company runs out of funding for it. It underscores the challenges of making progress on rare and ultra-rare diseases while also making sure treatments are safe and effective. Hope Filchak is a sassy four-and-a-half-year-old who loves swimming in the lakes and pools near her home in Gainesville, Georgia. She's also deaf and blind, with some functional vision in one eye and hearing with an aid in one ear. Hope was born with an extremely rare mitochondrial condition called MLS syndrome, of which there were only 64 documented cases in the US as of 2018. MLS syndrome, for Hope, causes a potentially life-threatening heart condition called cardiomyopathy, which can make her heart pump blood less efficiently. In February 2024, she started sleeping about 17 hours a day, and her speech began regressing. An echocardiogram revealed that Hope's heart function had dropped about 14 percentage points, into potentially hazardous territory. She then started taking elamipretide, an investigational drug for mitochondrial conditions. 'Pretty soon, honestly, she had a lot more energy,' her mother, Caroline Filchak, said. Most importantly, her heart stabilized. Hope's aunt, Anna Bower, said her niece's 'quality of life dramatically improved' and soon after, she was 'running, dancing, and playing' like any other child her age. First developed in 2004, elamipretide has a long history. Advocates for patients with Barth syndrome – another mitochondrial condition with about 150 known patients – asked Stealth BioTherapeutics to pick up the drug in 2014 and shepherd it through the regulatory process. Stealth submitted its first application to the FDA in 2019, and then it went through four different review divisions at the agency. In an October 2024 meeting of the FDA's cardiovascular and renal drugs advisory committee, patients and physicians spoke about the positive effects of the drug, and the advisers eventually voted 10-6 to recommend it. 'Patients and families saw the [advisory committee's] endorsement as an encouraging sign because the FDA almost always follows its recommendation,' Bower said in June. 'But last month, it didn't.' The FDA rejected the application in May. Internal FDA reviewers noted that the drug had not met its endpoint in phase 2 trials of 12 study participants. 'We don't feel like they looked at a totality of evidence where the patient's voice was heard in the decision,' Caroline Filchak said, who added that it's been difficult to measure the effectiveness of the drug because of how rare the disease is. The FDA did offer a new pathway to approval, Stealth said in a press release. That process takes at least eight months, though it can also take years. Stealth laid off 30% of its staff after the rejection. Advocates such as Filchak are worried the company will not be able to continue pursuing approval. 'If [the FDA] drag their feet like they have throughout this entire process, Stealth is not going to be able to continue operations,' she said. Under the new pathway, the medication is not available for infants. Stealth has said that 35 patients around the world are receiving the medication, and two-thirds of them are very sick infants. In a congressional hearing in late June, the Republican representative Earl L 'Buddy' Carter of Georgia asked Robert F Kennedy Jr, the secretary of the US Department of Health and Human Services, about treatments for rare mitochondrial conditions. Carter mentioned two young constituents with these conditions, including Hope Filchak. The children 'need your help in accessing life-saving medications', Carter said, promising to follow up with Kennedy after the hearing. For now, Hope has a three-month supply of the drug. 'For children like Hope, there are no other options,' Bower said. There are no FDA-approved medications like elamipretide, and there are no similar drugs in late-stage development. Caroline Filchak said that this administration 'does have a stated commitment to accelerating therapies for rare diseases. And it seems like this recent decision by the FDA doesn't align with that commitment.' Marty Makary, the FDA's commissioner, recently announced plans to accelerate approval for select drugs and companies. He has also floated the use of machine learning, often called AI, to review applications quickly. But there are already four ways for the FDA to expedite the review of new medications, and the approval speed is not the sticking point for drugs such as these, Filchak said. Elamipretide is an example of the difficulty of developing drugs for ultra-rare conditions – and for approving them based on clinical evidence, said Holly Fernandez Lynch, bioethicist at the University of Pennsylvania Perelman School of Medicine. 'It's not the poster child of FDA efficiency,' Fernandez Lynch said, noting the long time span and the four different review divisions at FDA. 'But it's also not the poster child of 'Oh my God, we have a drug that works amazingly well, and FDA is standing in the way, and why won't they just use their regulatory flexibility?'' The drug hasn't been approved yet because it hasn't met a pre-specified endpoint, Fernandez Lynch added: 'If the evidence doesn't support approval, if the systematic evidence collection doesn't show benefit, then FDA really can't approve it.' The biotech company is now resubmitting data on knee strength improvement as part of its new application. 'Of course, these patients have a need. Of course, they have an altered tolerance for risk and altered tolerance for uncertainty,' said Fernandez Lynch. 'That's the really devastating part of all of this. And it's really heartbreaking, but it does not mean the FDA should grant approval to a product that hasn't been demonstrated effective, because we really don't know that it works.' Approving a medication without this evidence could lead to issues developing other drugs for the same conditions, Fernandez Lynch said. 'People say, 'Well, what's the big deal? These patients have nothing. Just let them try it.' I get that. If I was that mom, I would do the same thing, right? But the FDA has to make judgments for the population,' she said. For Caroline Filchak, who works for a petroleum delivery company, she plans to continue advocating for her daughter and other affected children – and has even gotten the whole family involved. 'You don't, when you think about having a kid, think that you're going to be doing this, but you do what you've got to do for your kids,' she said, noting that she and her husband, Ben, took their seven-year-old son, Thomas, to the October meeting. 'We call him our baby advocate. Ever since that meeting, every night when he would say his prayers, he would pray that the FDA says 'yes'.'
Wall Street Journal
03-07-2025
- Business
- Wall Street Journal
Orphan Drugs Are Neglected No More
A bill as large as the GOP reconciliation budget takes time to pore through, and we can now report a hidden gem: It fixes the Inflation Reduction Act's disincentive to develop treatments for rare diseases. The IRA was a woeful bill in countless ways, but the worst is its Medicare drug price controls. The early damage has been less investment in so-called orphan drugs for rare diseases. Because the pool of patients is small, manufacturers have to set higher prices to recoup their investment. This makes them an attractive target for Medicare bureaucrats. The IRA price controls exempted orphan drugs, but only if they are approved for a single indication. They lose their exemption if they are used to treat more than one rare disease, though many diseases share an underlying pathology such as a gene mutation. Of the 280 orphan drugs approved between 2003 and 2022, 63 were approved later for another indication. The law has discouraged companies from studying orphan drugs for multiple rare diseases. Some face a Hobson's choice. If an experimental medicine works for two rare diseases, companies might have to jettison one use lest their drug become subject to price controls and return on its investment collapse.
Yahoo
02-07-2025
- Business
- Yahoo
ANI Pharmaceuticals to Participate at Leerink Partners Therapeutics Forum: I&I and Metabolism on July 8th
PRINCETON, N.J., July 02, 2025 (GLOBE NEWSWIRE) -- ANI Pharmaceuticals, Inc. ('ANI' or the 'Company') (Nasdaq: ANIP) today announced that Nikhil Lalwani, President and Chief Executive Officer, will host 1x1 meetings at the Leerink Partners Therapeutics Forum: I&I and Metabolism on Tuesday, July 8, 2025, in Boston, MA. About ANI Pharmaceuticals, Inc. ANI Pharmaceuticals, Inc. (Nasdaq: ANIP) is a diversified biopharmaceutical company committed to its mission of 'Serving Patients, Improving Lives" by developing, manufacturing, and commercializing innovative and high-quality therapeutics. The Company is focused on delivering sustainable growth through its Rare Disease business, which markets novel products in the areas of ophthalmology, rheumatology, nephrology, neurology, and pulmonology; its Generics business, which leverages R&D expertise, operational excellence, and U.S.-based manufacturing; and its Brands business. For more information, visit Investor Relations: Lisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793 E: lwilson@ Source: ANI Pharmaceuticals, 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
