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Mashup Score: 1New Model For Real World Evidence? Leqembi Registry Data Will Be Bolstered By Medicare Claims - 10 month(s) ago
Senior Centers for Medicare and Medicaid Services official Lee Fleisher described the game plan behind the CMS-facilitated patient registry for the new Alzheimer’s disease treatment, but former CMS official Sean Tunis is still concerned the approach won’t meet data expectations in the planned two-year window.
Source: Pink SheetCategories: Latest Headlines, Rare DiseaseTweet
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Mashup Score: 1After Leqembi Approval, US FDA In No Rush To Declare Amyloid A Validated Surrogate Endpoint - 10 month(s) ago
Ability to use beta amyloid plaque reduction to support a traditional approval In Alzheimer’s, or for use with other drug classes, needs to be evaluated case-by-case based on the available data, agency says. In the HIV/AIDS space, it took FDA decades to declare a surrogate endpoint validated.
Source: Pink SheetCategories: Latest Headlines, Rare DiseaseTweet
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Mashup Score: 0Amylyx To Appeal Against EMA Rejection Of ALS Drug Albrioza - 11 month(s) ago
Amylyx is standing by the clinical trial data it used to support its EU marketing application for its amyotrophic lateral sclerosis treatment, saying that the same data formed the basis of approvals for Albrioza in the US and Canada.
Source: Pink SheetCategories: Latest Headlines, Rare DiseaseTweet
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Mashup Score: 0Product Quality ‘Wild Card’? Sarepta’s Gene Therapy Manufacturing Process Change Means More Empty Capsids - 12 month(s) ago
SRP-9001 intended for commercialization is not analytically comparable to the product used in earlier clinical studies; FDA CMC reviewer said agency does not mandate the extent of empty or full capsids but, instead, looks at the totality of data to assess safety and efficacy.
Source: Pink SheetCategories: Latest Headlines, Rare DiseaseTweet
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Mashup Score: 0For Lilly’s Donanemab, US FDA Commissioner Is Already Thinking About Post-Market Questions - 1 year(s) ago
From positive topline data to Robert Califf weighing in on the right patient population to treat with the Alzheimer’s candidate in less than a day, the drug rejected by the agency for accelerated approval earlier this year looks to make a quick comeback.
Source: Pink SheetCategories: Latest Headlines, Rare DiseaseTweet
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Mashup Score: 1Pharma Spent Record Amount on Lobbying in 2022 - 1 year(s) ago
Our infographic breaks down the biggest contributors, recipients and legislative targets.
Source: Pink SheetCategories: Latest Headlines, Rare DiseaseTweet
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Mashup Score: 1
A November 2022 refuse-to-file letter cited clinical and statistical issues for the stem cell therapy, which failed its Phase III clinical efficacy endpoint. However, BrainStorm has consistently asserted that NurOwn demonstrated benefit in patients with less advanced disease at baseline, and it is encouraged by regulatory flexibility FDA recently has shown for the neurodegenerative…
Source: Pink SheetCategories: Latest Headlines, Rare DiseaseTweet
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Mashup Score: 1Accelerated Approval: Potential Sources Of Confirmatory Evidence Weighed At Tofersen Review - 1 year(s) ago
‘Clear signal’ in the ATLAS study of presymptomatic, SOD1 mutation carriers could provide evidence needed to confirm tofersen’s benefit in symptomatic ALS patients, FDA’s Teresa Buracchio says, adding that getting confirmatory evidence from the VALOR open-label extension ‘could be a little more tricky.’
Source: Pink SheetCategories: Latest Headlines, Rare DiseaseTweet
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Mashup Score: 2BrainStorm Hoping For Advisory Committee After FDA Refuses To File BLA For Its ALS Cell Therapy - 1 year(s) ago
BrainStorm, which has a history of antagonizing FDA with direct appeals to patients for NurOwn, tells investors that public elements of an adcomm could be ‘persuasive,’ but first step will be formal meeting request to try to overcome the RTF. Firm sees Relyvrio as template for success.
Source: Pink SheetCategories: Latest Headlines, Rare DiseaseTweet
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Mashup Score: 1
Center for Drug Evaluation and Research plans to issue a request for information this year to engage stakeholders on issues related to AI and machine learning. It received 175 AI/ML submissions in 2022, up from 14 in 2020.
Source: Pink SheetCategories: Latest Headlines, Rare DiseaseTweet
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