FDA advisers to discuss ALS drug at Wednesday meeting

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Mar 29, 2022 View in browser   By David Lim and Katherine Ellen Foley With Alice Miranda Ollstein On Tap — All eyes are on FDA's neurological drug advisory committee, which will meet Wednesday to discuss a drug intended to treat ALS. — Biden's budget request calls for $8.4 billion in total funding for FDA, which includes budget authority, user fee revenue and a pandemic preparedness fund. — Democrats are setting up a vote on a bill that aims to cut insulin costs insulin for patients. It's Tuesday. Welcome back to Prescription Pulse. 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). POLITICO will host its inaugural health care summit on Thursday, March 31, as the U.S. enters its third year of the Covid-19 pandemic. Through a series of interviews and panel discussions, summit participants — including providers, policymakers, regulators and patient representatives — will discuss the policies, political dynamics and tech trends shaping the sector. For details, or to attend in person or watch online, click here.   SUBSCRIBE TO NATIONAL SECURITY DAILY : Keep up with the latest critical developments from Ukraine and across Europe in our daily newsletter, National Security Daily. The Russian invasion of Ukraine could disrupt the established world order and result in a refugee crisis, increased cyberattacks, rising energy costs and additional disruption to global supply chains. Go inside the top national security and foreign-policymaking shops for insight on the global threats faced by the U.S. and its allies and what actions world leaders are taking to address them. Subscribe today.     Eye on FDA FDA ADVISERS TO MEET TO DISCUSS ALS DRUG — A highly anticipated meeting of FDA's advisory committee on neurological drugs is convening Wednesday to discuss a drug that aims to treat amyotrophic lateral sclerosis, or ALS, from Amylyx, Katherine reports. Background: Amylyx has completed Phase II clinical testing of its drug, for now called AMX0035. The drug slowed disease progression, though data showing the drug helped patients live longer wasn't statistically significant. But a longer-term continuation of the study showed the drug extended patients' lives by about six months. Last year, Amylyx announced FDA requested additional testing on the drug. However, in September 2021, it said it would submit the drug for FDA approval, following a meeting it had with the agency in July. The company completed an application in November, and FDA accepted it a month later. Doubting the data: Committee members will vote on whether the drug is effective at treating ALS. In presentation materials released Monday, FDA said the findings from Amylyx's Phase II study "do not translate to robust support for a drug effect in patients with ALS." The agency's presentation cites issues with the clinical trial design, including questions over how Amylyx counted deaths during the trial, trouble randomizing patients and potential accidental unblinding because of the drug's bitter taste. A repeat of Aduhelm? FDA's neurological drug advisory committee hasn't met since it discussed the controversial Alzheimer's drug Aduhelm, which it voted against approving. Both Aduhelm and AMX0035 are intended for fatal neurological diseases that have limited treatment options. FDA told both drugs' manufacturers they needed more data before approval. And both drugs have strong patient advocates behind them, who have pressed both FDA and Congress to give patients access to the drugs. "Did the FDA schedule an advisory committee meeting to placate patient advocates and other political pressure in the hope that the advisory committee would vote against it? Or is the FDA really considering approving it?" said Diana Zuckerman, the president of the National Center for Health Research, a nonprofit organization. She noted that a negative vote from the independent panel could give cover to the agency if it ultimately does not approve the drug. Disease advocates hope the agency will be flexible. "I'm not sure they're framing the overall question correctly," said Neil Thakur, the chief mission officer of the ALS Association. "The question really should be something like, 'Do we know enough about the effectiveness of this drug to make it a treatment option for people living with ALS today?'" What's next: FDA doesn't have to take its advisory committee's recommendations. It is expected to make a decision on the drug by June 29. BUDGET DAY — President Joe Biden's fiscal year 2023 budget request for FDA includes an increase of $356 million in budget authority appropriations over the agency's fiscal year 2022 appropriated amount. The administration is also asking for $1.6 billion in mandatory funding to be provided to FDA to prepare for future pandemics. In total, the budget request contains an ask of $8.4 billion, which is comprised of approximately $3.7 billion in budget authority, $3 billion in user fees and the $1.6 billion in pandemic preparedness funding. Under the budget proposal, FDA wants to spend $40 million on building upkeep and rent, $24 million on recruitment and training of new FDA inspectors and $68 million on data modernization efforts. "Additional funding brings new ways to leverage opportunities to protect and advance the health of every American with reliable and science-based information," FDA Commissioner Robert Califf said in a statement. FDA is floating several legislative proposals it argues will help the agency better protect public health: Reforming generic exclusivity: FDA wants to incentivize drugmakers to quickly market first generics after winning approval instead of delaying commercialization for years. The agency proposes amending the 180-day exclusivity provisions to allow approval of subsequent applications unless a first applicant begins marketing its drug. Tweaking accelerated approval: FDA is asking for new authority to place additional requirements on drugmakers that submit a drug to its accelerated approval program. The agency wants to compel manufacturers to demonstrate an ability to complete any proposed post-approval study in a timely manner and ensure it is adequately designed to verify clinical benefit. FDA also wants stronger withdrawal authority for the program. Requiring importers to pay destruction costs: When imported FDA-regulated products are refused and pose a significant public health concern, the agency wants the authority to compel the importer to pay destruction costs upfront to reduce legal burdens and cut down re-importation risk. Requiring device makers to address cybersecurity: FDA wants new authority to require medical device manufacturers to show, as part of a premarket submission, that their device was designed with cybersecurity considerations. The agency also wants companies to provide a software bill of materials with their devices and ensure that products can be quickly updated and patched. Coronavirus FDA LIMITS SOTROVIMAB USE — Health care providers in Connecticut, Maine, Massachusetts, New Hampshire, Rhode Island, Vermont, New Jersey, New York, Puerto Rico, and the Virgin Islands should no longer administer sotrovimab to Covid-19 patients because the current drug dosage is unlikely to be effective against the increasingly prevalent Omicron BA.2 subvariant, the FDA announced Friday. GlaxoSmithKline and Vir Biotechnology are preparing to submit data on a higher dose of the monoclonal antibody to global regulatory authorities, the companies said Friday. FDA SIGNS $2.1M CONTRACT TO STOP FRAUDULENT COVID DEVICES — FDA awarded a $2.1 million contract to Advanced Network Consulting on March 15 to help design a Covid-19 product authentication program for its Center for Devices and Radiological Health. "The contract is to develop analytical and modeling capabilities to detect and prevent counterfeit and fraudulent devices from entering the U.S. market," FDA spokesperson Stephanie Caccomo said. In Congress HOUSE RULES TO TEE UP INSULIN VOTE — The House Rules Committee is expected to vote Wednesday on a rule governing debate over Democratic legislation that aims to cut the insulin costs for patients, a Democratic aide told Alice. The bill could see a floor vote as soon as Thursday. "At the end of the day, I hope that we bring forward a reconciliation bill with additional reforms this year," Rep. Angie Craig (D-Minn.) told Alice. "I know we need to do Medicare negotiation. But we can't wait any longer to act on insulin. It's long overdue. We can't let perfect be the enemy of the good." Drug Pricing CMS ACTUARY: RETAIL RX DRUG SPENDING WILL CLIMB — Spending on retail prescription drugs is projected to climb at an average rate of 5 percent between 2021 and 2030, according to the CMS Office of the Actuary's new National Health Expenditure report. Coming Up in Pharma Wednesday: BioNTech holds its quarterly earnings call at 8 a.m. EDT. Also on Wednesday: The House Energy and Commerce Health Subcommittee holds a hearing at 9 a.m. EDT on reauthorization of FDA's medical device user fee program. Pharma Moves The American Clinical Laboratory Association has named Susan Van Meter as its new president. She previously worked at AdvaMedDx as executive director. Quick Hits POLITICO's Alex Thompson explores the influence of former Google CEO Eric Schmidt's foundation on the White House Office of Science and Technology Policy. Walmart is ceasing cigarettes sales in some of its stores, The Wall Street Journal's Sarah Nassauer and Jennifer Maloney report. Inside AgencyIQ Here are some highlights from our colleagues over at AgencyIQ, the regulatory insight platform for FDA. FDA rejects drug based on over-reliance of Chinese data — On March 24th, Eli Lilly announced that it had received a Complete Response Letter (CRL) from FDA, an action indicating that the application for licensure is not able to be approved based on currently available information for its investigational cancer drug sintilimab. The CRL was sent less than 6 weeks after FDA's Oncologic Drugs Advisory Committee met to hear Eli Lilly's case for the approval of sintilimab — and FDA's critique that the company's application for approval relied on a pivotal clinical trial conducted exclusively in China and was therefore poorly representative of the populations in which it would be used in the U.S. According to Lilly's press release, the CRL recommends "an additional clinical study, specifically a multiregional clinical trial comparing standard of care therapy" against sintilimab. The CRL comes just as FDA has also been requiring some other oncology companies to conduct post-approval studies to better assess representative diversity. Document Drawer FDA is hiring a chief medical officer. The application period closes April 18.   DON'T MISS POLITICO'S INAUGURAL HEALTH CARE SUMMIT ON 3/31: Join POLITICO for a discussion with health care providers, policymakers, federal regulators, patient representatives, and industry leaders to better understand the latest policy and industry solutions in place as we enter year three of the pandemic. Panelists will discuss the latest proposals to overcome long-standing health care challenges in the U.S., such as expanding access to care, affordability, and prescription drug prices. REGISTER HERE.       Follow us on Twitter David Lim @davidalim Lauren Gardner @Gardner_LM   Follow us

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