By Puyaan Singh and Kamal Choudhury (Reuters) -The U.S. Food and Drug Administration has denied approval for PTC Therapeutics (NASDAQ:PTCT)’ oral medication for a rare genetic disorder with limited treatment options, the company said on Tuesday. In its so-called Complete Response Letter, the FDA said the data did not provide substantial efficacy evidence for the drug, vatiquinone, in treating Friedreich’s ataxia and that a separate study was needed before resubmitting the application. At least three analysts said the regulator’s rejection was not surprising and that expectations had been low heading into the decision. PTC (NASDAQ:PTC)’s shares reversed premarket losses to rise more than 8% in morning trading. The FDA approved Sephience - PTC’s oral therapy for a rare metabolic disorder called phenylketonuria - last month, potentially countering revenue declines due to regulatory scrutiny and competition from cheaper rivals for its top-selling muscular disorder therapies, Translarna and Emflaza. "We see today’s CRL as a clearing event since investors can now turn their focus to the Sephience launch," J.P.Morgan analysts said in a client note. The company said it planned to meet with FDA officials to discuss potential next steps. "We are of course disappointed by the FDA’s decision to not approve vatiquinone," CEO Matthew Klein said, adding that the data till date demonstrated that the drug could be safe and effective for children and adults living with the disorder. Friedreich’s ataxia is a rare genetic disorder that causes progressive damage to the nervous system, often leading to difficulty in walking, speech problems and heart complications. The disease tends to develop in children and teenagers and gradually worsens over time. Currently, Biogen (NASDAQ:BIIB)’s Skyclarys remains the only FDA-approved treatment for Friedreich’s ataxia. Don't miss out on the next big opportunity! Stay ahead of the curve with ProPicks AI – 6 model portfolios fueled by AI stock picks with a stellar performance this year... In 2024 alone, ProPicks AI identified 2 stocks that surged over 150%, 4 additional stocks that leaped over 30%, and 3 more that climbed over 25%. That's an impressive track record. With portfolios tailored for Dow stocks, S&P stocks, Tech Stocks, and Mid Cap stocks, you can explore various wealth-building strategies. So if BIIB is on your watchlist, it could be very wise to know whether or not it made the ProPicks AI lists.

Funding for new drugs benefits eligible patients but could risk displacing financial resources from other areas, resulting in population health trade-offs, according to study results published in The…

(Reuters) -Outlook Therapeutics said on Thursday the U.S. health regulator has declined to approve its experimental drug to treat a type of eye condition, which causes blurred vision, sending its shares slumping nearly 70% before the bell. The U.S. Food and Drug Administration, in its so-called "complete response letter", cited a lack of substantial evidence of effectiveness in the company’s application for the drug, ONS-5010, for treating patients with wet age-related macular degeneration. This is a chronic eye disorder that causes blurred vision or a blind spot in the patient’s visual field, and is the leading cause of blindness among the elderly. The FDA’s decision marks another setback in Outlook’s prolonged efforts to enter the market, following a series of previous challenges. The company had withdrawn its application for the eye disease drug in 2022, after the FDA requested additional information. A year later, the regulator declined to approve the drug, citing manufacturing issues. "While we are very disappointed with this outcome, we intend to meet with the FDA to receive additional clarity on their requirements" to potentially approve ONS-5010, said CEO Bob Jahr. The FDA has recommended Outlook to provide additional data on the efficacy to support its application. The company’s resubmitted application was based on the results from a trial that studied the drug compared to Roche’s treatment Lucentis for improving vision in patients. In the key trial conducted by Outlook to address some of the FDA’s previous concerns, ONS-5010 failed to match Roche’s drug at eight weeks. 3rd party Ad. Not an offer or recommendation by Investing.com. See disclosure here or remove ads. Other than Lucentis, the already approved and available treatments for wet age-related macular degeneration include Regeneron’s Eylea and Roche’s Vabysmo. Outlook’s drug, under the brand name Lytenava, is approved in the European Union and the UK for the condition. ProPicks AI evaluates REGN alongside thousands of other companies every month using 100+ financial metrics. Using powerful AI to generate exciting stock ideas, it looks beyond popularity to assess fundamentals, momentum, and valuation. The AI has no bias—it simply identifies which stocks offer the best risk-reward based on current data with notable past winners that include Super Micro Computer (+185%) and AppLovin (+157%). Want to know if REGN is currently featured in any ProPicks AI strategies, or if there are better opportunities in the same space?

Critic of Drug Industry and COVID-19 Measures Appointed to Lead FDA Vaccine Program WASHINGTON (AP) — Dr. Vinay Prasad, a

"The FDA is set to rule on NeuroClear, a new Alzheimer's drug, by July 18. An in-depth look at the clinical trial data, the drug's mechanism, and the stakes."

The drug is part of an expanding cardio-renal-metabolic care portfolio, is approved for CKD associated with type 2 diabetes.

What is drug discovery and what are the phases of clinical research? In this tutorial, we explain the entire process of drug discovery and phases of clinical research in a clear, step-by-step format—from the earliest lab research to real-world testing and market approval. Part 1 covers how new medicines are discovered, the strategic role of pharmaceutical companies, and the innovation behind advanced treatments like targeted cancer therapies and biologics. Part 2 walks you through the phases of clinical research (Phase I to IV)—explaining the purpose of each stage, how drugs are tested in humans, and how safety and efficacy are proven over time. You’ll also understand why new treatments often come with high costs, reflecting years of scientific development, risk, and investment. What You’ll Learn: The full process of drug discovery and development The role of pharmaceutical companies in drug innovation Step-by-step breakdown of clinical trial phases I to IV...

The drug is part of an expanding cardio-renal-metabolic care portfolio, is approved for CKD associated with type 2 diabetes.

The FDA advisory panel has recommended the approval of donanemab, a new Alzheimer's drug by Eli Lilly, after voting unanimously that it effectively slows early-stage Alzheimer's disease and that its b...

<p>The U.S. Food and Drug Administration (FDA) is planning to incorporate artificial intelligence (AI) into its decision-making processes for various health-related fields, according to a recent artic...

High-ranking OpenAI employees have met with the FDA multiple times in recent weeks to discuss AI and a project called cderGPT.

Baylor University begins collaboration with FDA on human abuse potential trial for new drug.

What is drug discovery and what are the phases of clinical research? In this tutorial, we explain the entire process of drug discovery and phases of clinical research in a clear, step-by-step format—from the earliest lab research to real-world testing and market approval. Part 1 covers how new medicines are discovered, the strategic role of pharmaceutical companies, and the innovation behind advanced treatments like targeted cancer therapies and biologics. Part 2 walks you through the phases of clinical research (Phase I to IV)—explaining the purpose of each stage, how drugs are tested in humans, and how safety and efficacy are proven over time. You’ll also understand why new treatments often come with high costs, reflecting years of scientific development, risk, and investment. What You’ll Learn: The full process of drug discovery and development The role of pharmaceutical companies in drug innovation Step-by-step breakdown of clinical trial phases I to IV...