Second time’s the charm for ALS drug candidate

Presented by Doctors for America: Delivered every Tuesday and Friday by 12 p.m., Prescription Pulse examines the latest pharmaceutical news and policy.

Sep 09, 2022 View in browser   By Katherine Ellen Foley and David Lim Presented by On Tap — Advisers to the FDA endorse the agency approving a third ALS drug candidate. — A restructuring of the federal pandemic response could dismantle the White House Covid team by mid-2023. — The FDA approved a longer-lasting alternative to Botox on Wednesday. It's Friday. Welcome to Prescription Pulse. Have you heard our Pulsecheck podcast? This week, POLITICO's Megan Messerly talks with Krista Mahr about the many unknowns around monkeypox vaccine efficacy, and Professor Robert Blendon discusses the most important top lines from the newest POLITICO-Harvard survey. Listen to the latest episode. Send tips and feedback to David Lim ( dlim@politico.com or @davidalim ), Lauren Gardner ( lgardner@politico.com or @Gardner_LM ) or Katherine Ellen Foley ( kfoley@politico.com or @katherineefoley ).   A message from Doctors for America: TIME IS RUNNING OUT for Congress to put patients first! Before it expires at the end of the month, Congress must act to ensure the final FDA User Fee bill includes critical protections for patients like ensuring clinical trial diversity & reforming the accelerated approval pathway. Learn more about why doctors and patients are advocating for these key provisions to be included in the final reauthorization bill. This chance only happens once every five years!   Eye on the FDA An FDA advisory committee recommended that the agency give the nod to a novel ALS drug. | Andrew Harnik/AP Photo IN A 180 MOVE, EXTERNAL ADVISERS ENDORSE ALS DRUG — The FDA's advisory committee on neurological drugs voted 7 to 2 Wednesday in favor of recommending the agency approve a novel amyotrophic lateral sclerosis drug candidate from Amylyx Pharma, your host reports. Déjà vu: The meeting was the second time the FDA convened an expert panel about the drug candidate. In March, advisers voted against the drug's approval, citing concerns with the evidence generated from a single Phase II study. What changed: The company included two new analyses of its Phase II study and data from a Phase II trial of the drug on Alzheimer's patients, which demonstrated the drug's ability to improve biomarkers associated with neurodegeneration. Some advisers criticized this data rehashing, which they viewed as moving goalposts. "There's a reason that in games and sports … the rules are decided before the game, not after the game, not after the plays are made," said G. Caleb Alexander, a professor of epidemiology and medicine at the Johns Hopkins Bloomberg School of Public Health Center for Drug Safety and Effectiveness, who voted no against a favorable recommendation. But others pointed to the severe unmet need for ALS drugs, which could help make the case for the FDA to use its regulatory flexibility. The two other drugs previously approved for ALS, which both treat the symptoms like Amylyx's candidate does, were brought to market with different evidence than is usually required. Plus, Amylyx's candidate has an ongoing Phase III clinical trial, which advisers saw as a way to assess the drug's future success. If results, expected in a few years, are positive, patients will have access to the drug anyway; if not, Billy Dunn, the head of the agency's Office of Neuroscience in the Center for Drug Evaluation and Research, noted that the FDA could pull the drug from the market. Eyes emoji: "If PHOENIX [the Phase III trial] is not successful, we will do what is right for patients, which includes voluntarily removing the product from the market," said Justin Klee, the co-founder and co-CEO of Amylyx. The FDA is expected to make a final decision on Amylyx's application by Sept. 29. FDA APPROVES BOTOX ALTERNATIVE — The FDA approved Daxxify, a new anti-wrinkle injection from the biotech company Revance, on Wednesday. Like Botox, the 20-year-old drug from AbbVie that now makes up about 70 percent of the facial injection market, Daxxify uses a botulinum toxin to freeze facial muscles. But Revance is banking on Daxxify's longer-lasting effects — up to six months instead of three to four — to grab a share of the multibillion-dollar space. Coronavirus HHS officials say as many as 9,000 deaths and 100,000 hospitalizations could be prevented if Americans get the updated Covid shots from Moderna and Pfizer at the same rate as the annual flu shot. | Joe Raedle/Getty Images   A message from Doctors for America:   COVID WILL STICK AROUND, BUT THE WHITE HOUSE RESPONSE MAY NOT — The Biden administration is planning a restructuring of its approach to major elements of the coronavirus response that could begin the phaseout of the White House Covid team as soon as the middle of next year, Biden officials told POLITICO's Adam Cancryn. "Not a conversation": Officially, there are no plans to wind down the White House Covid team, a White House spokesperson told Adam. In a statement on Thursday , the administration announced that in addition to its booster campaign, it would be purchasing an additional 100 million at-home rapid tests to add to the national stockpile — not enough to replenish supplies, but hopefully enough to meet some potential immediate demands in the coming months. … and speaking of the booster campaign: HHS officials are using estimates that suggest if U.S. residents take the updated Covid shots from Moderna and Pfizer at the same rate as the annual flu shot, the country could prevent as many as 9,000 deaths and 100,000 hospitalizations. But some experts worry that uptake will be low, even with hefty administration backing. Others believe that boosters are better focused on only those most at risk of developing severe infections. "The goal of this vaccine is to prevent serious disease. The goal isn't to prevent mild illness," Paul Offit, the director of the Vaccine Education Center and an attending physician in the infectious diseases division at Children's Hospital of Philadelphia, told POLITICO. Offit, who sits on the FDA's independent vaccine advisory committee (which didn't meet ahead of the latest boosters' authorization), said most at risk are the elderly, those with pre-existing lung conditions and those with compromised immune systems, all of whom would likely be hospitalized for a Covid-19 infection. Industry Intel LABS, AHA, AMA LOBBY CONGRESS FOR TEST REIMBURSEMENT REFORM — Health industry groups wrote to congressional leaders on Thursday, urging passage of the Saving Access to Laboratory Services Act, which would set new limitations on annual increases or decreases in Medicare reimbursement for lab services. Congress has temporarily halted several rounds of cuts mandated by the Protecting Access to Medicare Act during the past several years. MEDICAL DEVICES FDA FLAGS REPORTS OF CANCER IN SCAR TISSUE AROUND BREAST IMPLANTS — The FDA notified the public on Thursday that it is aware of reports of cancers in the scar tissue around breast implants unrelated to breast implant-associated anaplastic large cell lymphoma. "While the FDA believes that occurrences of [squamous-cell carcinoma] or various lymphomas in the capsule around the breast implant may be rare, health care providers and people who have or are considering breast implants should be aware that cases have been reported to the FDA and in the literature," the FDA wrote in its safety communication. The agency said it is aware of fewer than 50 cases and has received 22 medical device reports of the cancers related to the new alert. The agency says it is unknown what the risk factors or incidence rate are. Pharma Moves David Kaslow is joining the the FDA as director of CBER's Office of Vaccines Research and Review. He previously worked at PATH as chief scientific officer and before that, at Merck and NIH.   A message from Doctors for America: Hey Congressional Leaders and staff: Time is running out to drastically improve the lives of patients. This opportunity only presents itself every 5 years: the FDA User Fee Reauthorization opens an avenue for Congress to ensure that new treatments are actually proven to be safe and effective for everyone who needs them. In case you missed it, we (and other leading health advocacy groups) sent a letter asking you to include the life-saving provisions in versions of the FDA User Fee Reauthorization bills. Specifically, we hope you will include the following in the final User Fee package: ● enforceable measures to ensure diverse representation in clinical trials and ● oversight of the accelerated approval drugs to ensure that their clinical benefit is proven in a timely manner. Learn more about why doctors and patients are advocating for key provisions to be included in the final reauthorization bill. Sincerely, DFA   Document Drawer The FDA on Thursday issued final guidance for companies recommending they note use of real-world data and real-world evidence in cover letters for drug and biologics submissions. HHS Secretary Xavier Becerra issued an emergency declaration that allows the FDA to issue emergency use authorizations for monkeypox and orthopoxvirus tests on Wednesday — a move the federal government says will expand testing capacity for the monkeypox virus circulating in the U.S.   Follow us on Twitter David Lim @davidalim Lauren Gardner @Gardner_LM   Follow us

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