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13 stories matching this filter · ← front page

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STAT1670Endpoints1315Fierce Biotech632BioSpace632BioPharma Dive494raps211onclive165fda recalls108
therapy area
allOncology11784Rare Disease2960Immunology2721Cardiology2404Pulmonology2054Hematology2004Vaccines1729Psychiatry & CNS
  • Pfizer halts Phase 2 trial of PF-06835375 in primary immune thrombocytopenia
    Pfizer announced that it is stopping its Phase 2 study of the experimental program PF-06835375, which was being evaluated in patients with primary immune thrombocytopenia, a rare autoimmune blood d…
    Endpoints · 10d agoNegative readoutNegative↗ source
  • Ambrose Healthcare acquires AM-Pharma for up to €161 million
    Ambrose Healthcare, a British rare‑disease specialist, announced it has completed the acquisition of Dutch biotech AM‑Pharma. The deal values the company at up to 161 million euros (about $181 mill…
    Fierce Biotech · 14d agoDealNeutral↗ source
fda medwatch100
globenewswire health73
fda press56
1141
Ophthalmology906
Endocrinology & Metabolism729
Pain702
Nephrology698
  • 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 · 17d agoPositive readoutNeutral↗ source
  • Caribou Biosciences to halt R&D and cut staff as financing for allogeneic CAR-T dries up
    Caribou Biosciences, co‑founded by Nobel laureate Jennifer Doudna, announced it will stop all clinical‑stage work and lay off staff because of a financing freeze in the allogeneic CAR‑T market. Th…
    BioSpace · 23d agoLayoffsNegative↗ source
  • Vaxcyte's experimental pneumococcal vaccine meets primary endpoints in Phase 3 trial
    Vaxcyte announced that its experimental pneumococcal vaccine, VAX-31, achieved the primary endpoints in a Phase 3 trial. The study enrolled just over 3,500 adults aged 50 and older who were random…
    STAT · 25d agoPositive readoutPositive↗ source
  • Ionis receives FDA approval for Zanvastro, the first treatment for Alexander disease, as Pfizer partners on new ADC program
    The U.S. Food and Drug Administration approved Zanvastro, marking the first approved medicine for the rare and often fatal neurological disorder Alexander disease. Developed by Ionis Pharmaceutical…
    BioPharma Dive · 1mo agoApprovalPositive↗ source
  • Bristol Myers Squibb pauses autoimmune CAR‑T trials after life‑threatening side effects
    Bristol Myers Squibb announced in early June that it is pausing its CAR‑T therapy trials targeting autoimmune diseases after the trials reported life‑threatening side effects. The company informed…
    STAT · 1mo agoRegulatoryNegative↗ source
  • FDA approves Priovant's Lisraya (brepocitinib) for rare skin disease dermatomyositis
    The U.S. Food and Drug Administration has cleared the first oral targeted treatment for dermatomyositis, a rare and disabling skin disorder. The medication, brepocitinib, will be sold under the br…
    BioPharma Dive · 2mo agoApprovalPositive↗ source
  • Medicare GUARD pilot faces biotech push to spare rare disease drugs as Moderna begins first-in-human Ebola vaccine trial
    The Medicare Part D GUARD pilot, designed to lower retail drug prices, is under scrutiny because excluding rare disease medicines would erase most of the projected savings, according to STAT. Biot…
    STAT · 2mo agoRegulatoryNeutral↗ source
  • Merger talks between Bristol Myers Squibb and AstraZeneca fade as Q2 earnings roll out and FDA advisory meetings heat up
    Talks of a possible merger between Bristol Myers Squibb and AstraZeneca have receded after analysts highlighted antitrust hurdles. Both companies, each valued over $130 billion, were rumored to con…
    BioSpace · 2mo agoDealNeutral↗ source
  • Arvinas and Pfizer license newly approved PROTAC breast cancer drug Veppanu to Rigel Pharmaceuticals
    Arvinas and Pfizer have signed a licensing agreement that transfers ownership of their newly approved breast cancer drug, Veppanu, to San Francisco‑based Rigel Pharmaceuticals. The deal gives Arvi…
    BioPharma Dive · 5mo agoDealPositive↗ source
  • Chiesi to acquire KalVista for $1.9 billion, adding hereditary angioedema pill to rare-disease portfolio
    Italian pharmaceutical group Chiesi announced it will purchase KalVista Therapeutics for $1.9 billion. The transaction values KalVista at $27 per share, a 40 percent premium to its last closing pri…
    STAT · 6mo agoDealPositive↗ source
  • Pfizer settles with generics to extend tafamidis patent until 2031, protecting revenue
    Pfizer announced settlements with three generic manufacturers-Dexcel Pharma, Hikma Pharmaceuticals and Cipla-to delay the launch of generic versions of tafamidis, the active ingredient in its rare‑…
    BioPharma Dive · 6mo agoDealPositive↗ source