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136 stories matching this filter · ← front page

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  • Merck KGaA to acquire Silence Therapeutics for $1.3 billion, expanding siRNA portfolio
    Merck KGaA, the German science and technology conglomerate, announced an agreement to purchase UK‑based Silence Therapeutics. The deal is valued at about $1.3 billion and gives Merck access to Sil…
    Endpoints · 23h agoDealPositive↗ source
  • Disc Medicine's rare sun-sensitivity drug meets all endpoints in Phase 3 trial
    Disc Medicine announced that its experimental therapy for a rare condition that makes sun exposure painful has completed a Phase 3 study. The trial reported success on every primary and secondary…
    Endpoints · 1d agoPositive readoutPositive↗ source
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102
fda medwatch89
globenewswire health73
fda press51
1609
Psychiatry & CNS1100
Ophthalmology841
Endocrinology & Metabolism684
Pain650
Nephrology633
  • Oxford biotech OMass Therapeutics secures $310 million to advance genetic adrenal disorder program
    Oxford-based OMass Therapeutics announced a $310 million private financing round, one of the largest recent fundraises for a UK biotech. The capital will fund the development of therapies targetin…
    Endpoints · 5d agoDealNeutral↗ source
  • FDA launches rare disease framework initiative
    A new initiative from the US Food and Drug Administration (FDA) aims to develop “an optimized, fit-for-purpose” framework to facilitate the development and marketing of treatments for rare diseases…
    raps · 5d ago↗ source
  • FDA proposes new framework prioritizing consistency over flexibility for rare disease drug reviews
    The FDA announced a shift in its approach to evaluating rare disease therapies, emphasizing a more uniform assessment process. Agency officials explained that the flexibility shown over the summer…
    Endpoints · 5d agoRegulatoryNeutral↗ source
  • FDA launches Rare Disease Framework Initiative to reshape drug development for small patient populations
    The FDA announced a new Rare Disease Framework Initiative aimed at overhauling how drugs and biologics for rare conditions are developed. The initiative will produce a tailored framework that refl…
    fda press · 5d agoRegulatoryPositive↗ source
  • Ultragenyx sells second priority review voucher as Biomea shares tumble after oral GLP-1 data
    Ultragenyx Pharmaceutical, a commercial-stage rare disease company, completed its second sale of a priority review voucher this month. The voucher sale reflects the company's strategy of monetizin…
    Endpoints · 5d agoDealNeutral↗ source
  • Beam Therapeutics reports early Phase 1/2 data for base editing therapy in GSD Ia, partners with Pfizer and Enodia
    Beam Therapeutics disclosed early results from a Phase 1/2 study evaluating a base editing approach aimed at correcting the genetic defect that causes glycogen storage disease type Ia, a rare metab…
    Endpoints · 6d agoPositive readoutNeutral↗ source
  • Syndax halts development of Niktimvo after Phase 2 failure in IPF trial
    Syndax announced it is stopping work on its experimental drug Niktimvo for idiopathic pulmonary fibrosis (IPF) after the compound did not meet its primary endpoints in a placebo-controlled Phase 2…
    Endpoints · 7d agoNegative readoutNegative↗ source
  • Mirum's Livmarli cuts itching in ultra-rare liver disease trial, showing significant benefit
    Mirum Pharmaceuticals reported that its ileal bile acid transporter inhibitor Livmarli achieved a statistically significant reduction in cholestatic pruritus compared with placebo in a late‑stage s…
    Endpoints · 7d agoPositive readoutPositive↗ source
  • AstraZeneca attributes Phase 3 failure of bone disease enzyme therapy to trial design flaws
    AstraZeneca disclosed that its Phase 3 study of an enzyme replacement therapy for a rare genetic bone disorder did not meet its primary objectives. The company said the shortfall was largely due t…
    Endpoints · 8d agoNegative readoutNegative↗ source
  • Gardeners Basics Recalls Waltham 29 Broccoli Sprouting Seeds Due to Possible Salmonella Contamination
    October 8, 2026, Gardeners Basics of Spanish Fork, Utah, is recalling one-pound bags of broccoli sprouting seeds because they may be contaminated with Salmonella, an organism which can cause seriou…
    fda recalls · 11d ago↗ source
  • Nuvig Therapeutics halts Phase 2 trial of NVG-2089 in chronic inflammatory demyelinating polyneuropathy
    Nuvig Therapeutics announced it is terminating one of its two ongoing clinical programs, a Phase 2 study of its candidate NVG-2089. The trial was designed to evaluate NVG-2089 in patients with chr…
    Endpoints · 12d agoCRLNegative↗ source
  • Eden Brothers recalls Calabrese broccoli seeds over possible Salmonella contamination
    Eden Brothers LLC announced a recall of its Calabrese broccoli seeds after being informed by its supplier that the product may be contaminated with Salmonella. The recall includes 1‑ounce and 1‑po…
    fda recalls · 12d agoCRLNegative↗ source
  • Shionogi agrees $2 billion upfront purchase of IntraBio, adding oral rare-disease drug Aqneursa to its portfolio
    Shionogi announced it will acquire Austin-based biotech IntraBio for $2 billion paid upfront. The deal brings IntraBio’s oral therapy Aqneursa into Shionogi’s rare‑disease pipeline, expanding its…
    Endpoints · 14d agoDealPositive↗ source
  • Report highlights 20 women reshaping biopharma across oncology and rare disease fields
    The 2026 special report spotlights twenty women who are driving change in biopharma, covering areas such as oncology and rare diseases. The series, now in its eighth year, has recognized over 160…
    Endpoints · 14d agoPositive↗ source
  • EAK Distribution recalls Home Grown broccoli seed packets over possible Salmonella contamination
    EAK Distribution LLC announced on October 2, 2026 that it is recalling its 8‑oz Home Grown Green Sprouting Calabrese Broccoli Seeds due to a potential Salmonella contamination. The company warned…
    fda recalls · 17d agoRegulatoryNegative↗ source
  • Gias Foods recalls Bettergoods Lemon Alfredo Fettuccine over possible Listeria contamination
    Gias Foods of New York announced a recall of all lot codes of its Bettergoods Authentic Italian Lemon Alfredo Fettuccine after routine testing indicated possible contamination with Listeria monocyt…
    fda recalls · 19d agoRegulatoryNegative↗ source
  • FDA approves Egetis’ Emcitate for rare genetic disease as Novo secures Chinese weight-loss pill in $2.6 billion deal
    The U.S. Food and Drug Administration has granted approval to Egetis' Emcitate, a therapy targeting a rare genetic disorder. This marks a regulatory milestone for the company and patients awaiting…
    raps · 21d agoDealPositive↗ source
  • FDA approves Egetis Therapeutics' Emcitate for rare X linked disease shortening life expectancy to 35 years
    Egetis Therapeutics announced that the U.S. Food and Drug Administration has granted approval for its drug Emcitate. The therapy is intended for patients with a rare X linked genetic disorder that…
    Endpoints · 21d agoApprovalPositive↗ source
  • FDA approves Emcitate for peripheral thyrotoxicosis in patients with MCT8 deficiency
    The U.S. Food and Drug Administration announced approval of Emcitate (tiratricol) tablets formulated for oral suspension to treat peripheral thyrotoxicosis in patients with MCT8 deficiency, also ca…
    fda press · 21d agoApprovalPositive↗ source
  • Nonprofits launch ultra-rare immune disease gene therapy Waskyra at $3.25 million price
    Two nonprofit organizations have received FDA approval for a gene therapy that targets an ultra-rare immune disorder. The therapy, called Waskyra, is now being launched with a list price of $3.25…
    Endpoints · 21d agoApprovalNeutral↗ source
  • FDA advises early engagement and error prevention for BLA submissions, says CBER expert
    A CBER specialist shared practical guidance for companies preparing biologics license applications. The expert stressed that early interaction with the FDA’s Office of Therapeutic Products can str…
    raps · 22d agoRegulatoryNeutral↗ source
  • FDA approves Mirum's Atebrioz for ultra-rare disease that hardens muscle and connective tissue
    The U.S. Food and Drug Administration has granted approval for Mirum Pharmaceuticals' new therapy for an ultra-rare genetic disorder that causes muscle and connective tissue to turn into bone. The…
    Endpoints · 24d agoApprovalPositive↗ source
  • Experts say expanding beyond rare disease markets can make ultra-rare therapies financially viable
    The piece highlights that investment in ultra‑rare disease treatments remains limited, and experts argue the answer is not to focus solely on the rare condition itself. They propose that drugmaker…
    Endpoints · 24d agoPositive↗ source