ALS drug rekindles accelerated approval debate

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Mar 21, 2023 View in browser   By Katherine Ellen Foley and David Lim Driving the Day The FDA appears willing to approve Biogen’s ALS drug, tofersen. | Steven Senne/AP Photo ALS DRUG TO RECEIVE EXPERT SCRUTINY — The FDA’s external panel of neurological experts will discuss tofersen, a novel therapy from Biogen for the treatment of amyotrophic lateral sclerosis, on Wednesday. Tofersen is a spinal-cord injection for patients with a form of ALS caused by a mutation that leads the body to produce toxic proteins. The drug candidate stops the body from making those deformed proteins, which lead to progressive muscle weakness and death. Biogen asked the FDA to grant tofersen accelerated approval pathway last July. The pathway lets the agency consider whether a drug affects a surrogate, or different, endpoint — in this case, a protein called neurofilament light chain — instead of concrete evidence of patient benefit. After receiving accelerated approval, drug manufacturers must conduct an additional trial showing their product works. No one debates ALS’ unmet need: The disease is fatal typically within three to five years from symptom onset. The handful of existing treatments for ALS targets patients’ quality of life but not the causes of the disease. Recent scrutiny: The accelerated approval pathway has been the subject of lawmaker and public scrutiny in recent months. Some argue the pathway puts expensive drugs on the market that don’t help patients and the FDA doesn’t do enough to ensure follow-up trials are completed expeditiously. Disease advocates argue the pathway is crucial for those with rare, fatal diseases — a sentiment Peter Marks, head of the FDA’s Center for Biologics Evaluation and Research, echoed to attendees of the annual Muscular Dystrophy Association meeting on Monday. Marks spoke specifically about the pathway’s use for gene therapies that treat conditions affecting fewer than 100 people. ALS affects 5,000 patients annually; the type of ALS that tofersen would treat affects only about 330 people annually. The FDA is expected to make a decision on tofersen by April 25. It doesn’t have to side with the advice of its expert panel, but it usually does. IT'S TUESDAY. WELCOME TO PRESCRIPTION PULSE. It’s Trea Turner’s world and we’re just living in it. Send tweet. Send news and tips to David Lim (dlim@politico.com or @davidalim) or Katherine Ellen Foley (kfoley@politico.com or @katherineefoley). TODAY ON OUR PULSE CHECK PODCAST, host Ben Leonard talks with reporter Rachel Bluth about California’s plan to partner with Civica, a Utah-based generic pharmaceutical company, to start producing California-branded insulin in a bid to make the drug more affordable. The new insulin, which will cost $30 for a 10 mL vial, is awaiting FDA approval. Listen to today’s Pulse Check podcast In Congress Moderna’s CEO will appear before the HELP Committee on Wednesday to discuss the proposed market price of the company’s Covid vaccine. | Carl Court/Getty Images MODERNA IN THE HOT SEAT — No one expects Moderna CEO Stéphane Bancel to get a warm reception from Senate HELP Committee Chair Bernie Sanders (I-Vt.) at Wednesday’s hearing on what his company plans to charge for its Covid-19 vaccine once it is commercialized, but don’t expect a cakewalk from ranking member Bill Cassidy (R-La.) either. Cassidy spokesperson Ty Bofferding said the senator has “serious concerns” that the hearing has been promoted as a “kangaroo court public shaming, rather than a fact-finding mission.” But the lawmaker physician plans to ask “serious questions for Moderna regarding how they came to their proposed price and specifics of their program to offer the vaccine to patients at no cost.” FDA BUDGET REQUEST HEARING TENTATIVELY SET — Senior leadership of the FDA will likely testify before the Senate Appropriations Subcommittee on agriculture, rural development, FDA and related agencies on April 19, according to a tentative schedule. FDA officials can pitch their legislative requests for fiscal 2024 at the hearing. The House Appropriations Subcommittee is set to hold a member day on Thursday at which lawmakers will advocate for their priorities in the coming fiscal year. Separately, Jeff Shuren, the director of the FDA Center for Devices and Radiological Health, is slated to speak to the Alliance for a Stronger FDA on Thursday about his center’s requests. The Senate Committee on Homeland Security and Governmental Affairs holds a hearing Wednesday on what’s driving U.S. drug shortages, what those shortages mean for health and national security and potential reforms. The hearing comes as patients have struggled for months to fill certain prescriptions, such as those for high-profile Adderall and the diabetes drug Ozempic.   DOWNLOAD THE POLITICO MOBILE APP: Stay up to speed with the newly updated POLITICO mobile app, featuring timely political news, insights and analysis from the best journalists in the business. The sleek and navigable design offers a convenient way to access POLITICO's scoops and groundbreaking reporting. Don’t miss out on the app you can rely on for the news you need, reimagined. DOWNLOAD FOR iOS– DOWNLOAD FOR ANDROID.     Eye on the FDA ANOTHER MPOX TEST — On Friday, the FDA granted emergency use authorization to Cue Health’s point-of-care mpox test. Drug Pricing HOSPITALS SLAM PhRMA-TIED GROUP — A wide-ranging group of hospitals punched back on Monday at the newly introduced Alliance to Save America’s 340B Program, describing the unlikely alliance between PhRMA and the National Association of Community Health Centers as an attack on a program intended to boost uninsured and low-income patients’ access to brand-name drugs. “The PhRMA-led group, deceptively branded as an effort to save 340B, proposes to remove many hospitals from the program and significantly narrow its scope of eligible drugs and patients,” the hospital group, which includes the American Hospital Association, America’s Essential Hospitals and the Association of American Medical Colleges, said in a statement. “The proposal would only save drug company profits by reducing their already modest financial contribution to the health care safety net.” But PhRMA spokesperson Nicole Longo argued the status quo is allowing hospitals to profit off the program. “It’s disappointing some 340B hospitals continue to oppose changes that would put the focus of the 340B program back on patients and true safety-net providers,” Longo said. CBO OVERESTIMATED PART D SPENDING — Net spending by Medicare Part D on prescription drugs in 2019 was lower than the Congressional Budget Office estimated in its August 2010 baseline projections, POLITICO’s Ben Leonard reports. The slower growth occurred because of significant generic drug competition and fewer new expensive prescription drugs being introduced than CBO projected, the nonpartisan office wrote Friday to Sen. Sheldon Whitehouse (D-R.I.). Pharma Moves Ashley McEvoy, executive vice president of medtech at Johnson & Johnson, was elected chair of the device trade group AdvaMed board of directors for a two-year term. America's Health Insurance Plans has hired Sean Dickson, formerly of West Health Policy Center, as its senior vice president of pharmaceutical policy and strategy. Document Drawer On Friday, the FDA issued guidance for small businesses outlining how to comply with tobacco product requirements tied to the Tobacco Control Act). The agency also issued final guidance with answers to frequently asked questions about how to demonstrate substantial equivalence of a new tobacco product. And retailers received revised guidance on the FDA Center for Tobacco Products’ policy approach to civil money penalties and no-tobacco-sale orders.   STEP INSIDE THE WEST WING: What's really happening in West Wing offices? Find out who's up, who's down, and who really has the president’s ear in our West Wing Playbook newsletter, the insider's guide to the Biden White House and Cabinet. For buzzy nuggets and details that you won't find anywhere else, subscribe today.       Follow us on Twitter David Lim @davidalim Lauren Gardner @Gardner_LM Katherine Ellen Foley @katherineefoley   Follow us

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