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.
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