| | | | |  | | By Lauren Gardner and David Lim | | | | | 
The FDA will review a new drug therapy for Barth syndrome, an exceedingly rare genetic disease. | Sarah Silbiger/Getty Images | FDA TO REVIEW BARTH SYNDROME DRUG — Stealth BioTherapeutics said Monday that the FDA had accepted its new drug application after years of back-and-forth with agency officials over a therapy to treat an ultra-rare genetic condition. Elamipretide, if approved, would be available to treat the approximately 150 Americans with Barth syndrome, a disorder that can cause an enlarged and weakened heart, muscle weakness, recurrent infections and delayed growth. Those affected — typically males — face a shortened life expectancy into their 40s, though most early deaths occur by age 5, according to Stealth. Meeting on the docket: The FDA has indicated to the company it plans to hold an advisory committee meeting to discuss the application, a development CEO Reenie McCarthy said she welcomes given how rare Barth is — and the type of data Stealth is asking regulators to consider. “It becomes important to give that explanation and for patients to have a public forum,” she told Prescription Pulse. ‘Not your gold standard’: The company has suggested two ways in which the FDA could approve the drug. The agency could consider full approval based on Stealth’s natural history study, which tracks the progression of a disease without medical intervention. The agency declined to consider this data in 2021, McCarthy said, while adding that more approvals relying on such studies have been handed down since then. Stealth also believes the agency could grant accelerated approval based on a mid-stage clinical trial suggesting long-term improvements in cardiac function, McCarthy said, noting the company has signaled its willingness to conduct postmarketing studies. McCarthy acknowledged the company seeks an FDA endorsement based on “not your gold standard — a placebo-controlled clinical trials approach.” That’s a longstanding issue with rare-disease drug candidates, given the inherently small patient base available for trials. McCarthy said the panel’s input would be helpful as the FDA considers interpreting small datasets for such conditions. But it’s unclear when that will happen — Stealth sought a priority review, which takes six months, but the agency accepted the application under standard review, which takes 10 months. An FDA spokesperson said rare disease drugs, “regardless of population size, go through the same rigorous scientific review process as all other drugs.” IT’S TUESDAY. WELCOME BACK TO PRESCRIPTION PULSE. Your host hopes everyone used proper eyewear to enjoy yesterday’s enthralling eclipse! Send news and tips to Lauren Gardner (lgardner@politico.com or @Gardner_LM) or David Lim (dlim@politico.com or @davidalim).
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Pfizer wants to expand its RSV vaccine to all age groups at risk of the virus. | Mario Tama/Getty Images | PFIZER SIGNALS RSV VAX EXPANSION — Pfizer plans to seek FDA approval to broaden the prescribing label of its respiratory syncytial virus vaccine, Abrysvo, to include all adults at increased risk for the disease, the company said Tuesday, a move that would make the shot the first of its kind. A late-stage clinical trial met its primary immune response and safety endpoints for two RSV strains, said Dr. Iona Munjal, Pfizer’s executive director of clinical vaccine R&D. Adults with underlying conditions like asthma, diabetes and chronic obstructive pulmonary disease are at higher risk of severe disease from the virus, which typically circulates during the winter months. About 10 percent of U.S. adults under 50 have at least one chronic condition predisposing them to this risk, and nearly a quarter of adults ages 50 to 64 do, Munjal said. Pfizer’s RSV vaccine is available for adults 60 years and older and pregnant people — who can pass immunity on to their babies — between 32 and 36 weeks pregnant. GSK’s Arexvy, the first FDA-approved RSV vaccine, is available for adults 60 and older.
| | SLOW HUMIRA COMPETITION RAISES ALARM — Biosimilar competitors to blockbuster AbbVie autoimmune disease drug Humira snagged less than 2 percent of the market in the past year, according to an industry analysis of data from IQVIA, a health analytics company. Craig Burton, the executive director of the Biosimilars Council, argued the lack of uptake of biosimilar competition means some manufacturers will have to decide whether they need to exit the market. Biosimilars, which are cheaper copycats of pricey biologic drugs, were thought to be the wave of the future when they gained approval to launch a year ago. “This was supposed to be the moment for biosimilars,” Burton said. “But that’s only a good thing if it’s a successful moment. If it’s a moment where everyone steps back, then it could be very discouraging for future biosimilars.” Boehringer Ingelheim recently said it would lay off sales staff because of poor sales of its biosimilar Cyltezo, the first interchangeable alternative to Humira, Burton said. “We’re seeing some shifts potentially in formularies, but not as quickly as we need to,” Burton said. “It’s clearly taking much longer than people had hoped, and the question is how many biosimilars are going to be left by the time [pharmacy benefit managers] get serious about this.” The Pharmaceutical Care Management Association, which represents PBMs, has advocated for Congress to eliminate the FDA’s “interchangeable” designation for biosimilars, which it argues confuses providers and patients because the products are already copycat versions of brand names. When the FDA deems a biosimilar “interchangeable” with a branded product, the alternative drug may be substituted at the pharmacy counter without a doctor’s approval, much like generics are swapped for brand-name drugs. The Biden administration proposed eliminating the designation in its fiscal 2025 budget request.
| | | | Easily connect with the right N.Y. State influencers and foster the right relationships to champion your policy priorities. POLITICO Pro. Inside New York. Learn more. | | | | | | | | | CRISPR PATENT CASE HEADS TO COURT — The latest iteration of a long-running legal drama over who was the primary inventor of CRISPR-Cas9 gene editing will play out in a Washington courtroom next month. The U.S. Court of Appeals for the Federal Circuit will hear oral arguments May 7 between the regents of the University of California and the Broad Institute. The two scientists awarded the Nobel Prize for discovering the technology, Jennifer Doudna and Emmanuelle Charpentier, are credited with creating CRISPR. But the Broad Institute — a genomic research partnership between the Massachusetts Institute of Technology and Harvard University — holds the U.S. patents on editing human cells with CRISPR and claims it was the first to apply the innovation to living cells. The case’s outcome shouldn’t affect the availability of Casgevy, the new CRISPR therapy for certain sickle cell disease patients, Jacob Sherkow, a professor at the University of Illinois at Urbana-Champaign College of Law, said. However, the case “continues to cast a pall over whether the next CRISPR drug is going to be able to launch with what people in the industry call ‘freedom to operate,’” he said, referring to a company’s ability to make and sell products without infringing on another entity’s patents.
| | KENTUCKY RESCINDS COLD-DRUG PURCHASE CAP — A measure to eliminate annual purchase limits for pseudoephedrine, a common cold drug, became law Friday in Kentucky. Federal regulation requires products containing the ingredient to be kept behind the counter at retail pharmacies due to the risk of buyers diverting the drugs to produce illicit methamphetamine.
| | | | Donald Trump blasted Sen. Lindsey Graham (R-S.C.) on Monday, arguing the senator’s outspoken conservative stance in favor of strict abortion limits will hurt Republicans in the November election, POLITICO's Anthony Adragna reports. | | | | Follow us on Twitter | | | | Follow us | | | | |
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