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#罕见病

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Oct 8Thu
  1. biopharmadive.com40

    Halozyme prevails in patent fight with Merck; Enliven readies anticipated leukemia trial

    A Netherlands court blocked sales of Merck's subcutaneous Keytruda Qlex in eight European countries, siding with Halozyme in a patent dispute over its under-the-skin drug delivery technology. Separately, Enliven Therapeutics reached agreement with the FDA on a late-stage trial of relcobitinib in chronic myeloid leukemia, enrolling 450 people to test superiority at spurring a major molecular response. Halozyme is also challenging Merck in a U.S. court after a win in Germany.

    Why it matters: Patent ruling blocks subcutaneous Keytruda across Europe and FDA-aligned CML trial design sharpens competition for Novartis and Merck.

Oct 7Wed
  1. biocentury.com41

    RougeTx: Reattaching pericytes to stabilize vessels in HHT

    RougeTx, a Naarden, Netherlands-based spinout of Leiden University Medical Center, launched with a $58 million series A co-led by BioGeneration Ventures, Angelini Ventures and Kurma Partners to advance RTX-001 into the clinic. The oral small molecule, designed for once-daily dosing, aims to stabilize blood vessels by reattaching pericytes rather than suppressing angiogenesis in hereditary hemorrhagic telangiectasia. The company will now move RTX-001 toward clinical testing in HHT.

    Why it matters: Series A funds a first-in-class oral approach to a rare vascular disease with no such option named.

Oct 6Tue
  1. biospace.com67

    Shionogi makes $2B rare disease play with IntraBio buy

    Shionogi will acquire IntraBio for $2 billion upfront, gaining Aqneursa, the first and only approved treatment for ataxia-telangiectasia and the only therapy for Niemann-Pick disease type C, an ultrarare genetic disorder with around 1,000 potential US patients. Aqneursa generated $68 million in net sales last year in NPC, and Jefferies estimates $1 billion in global peak sales. The deal extends a rare disease build-out that includes Radicava, acquired from Tanabe Pharma for $2.5 billion, and IntraBio continues studying Aqneursa in GM2 gangliosidoses and other disorders.

  2. biocentury.com72

    Building again in rare diseases, Shionogi pays $2B for IntraBio

    Shionogi will acquire privately held IntraBio for $2 billion to expand its rare disease portfolio, gaining rights to Aqneursa levacetylleucine (IB1001), a modified N-acetyl-L-leucine amino acid therapy targeting neurological dysfunction and degeneration. The drug is approved for two indications, including one first-in-disease approval. The deal awaits completion.

    Why it matters: $2 billion rare-disease acquisition adds an approved first-in-disease neurological therapy to Shionogi's portfolio.

  3. biopharmadive.com65

    Shionogi pays $2B to acquire a rare disease startup

    Shionogi agreed on Oct. 5, 2026 to acquire Texas-based IntraBio for $2 billion in cash, gaining Aqneursa, a marketed treatment for Niemann-Pick disease type C and Ataxia-Telangiectasia. IntraBio, which will become a wholly owned subsidiary of Shionogi's U.S. division, reported $68 million in sales in 2025. Shionogi said it will review the deal's effect on its outlook for the fiscal year ending next March.

Oct 5Mon
  1. biopharmadive.com40

    Optogenetics researchers win a Nobel Prize; Roche bets on Alector brain drug

    The Nobel Prize in Physiology or Medicine went to Peter Hegemann, Georg Nagel and Karl Deisseroth for optogenetics, a light-based method for studying neurons. Alector licensed its preclinical Parkinson's therapy AL050 to Roche's Genentech for $100 million in guaranteed cash and up to $1.17 billion in milestones plus royalties. Separately, the FDA approved Lilly's Jaypirca as a first-line treatment for CLL or SLL patients without a 17p deletion.

    Why it matters: Nobel recognition for optogenetics and a sizeable Roche-Alector brain drug deal matter to neuroscience-focused readers.

  2. biospace.com62

    CSL strikes up to $1.6B deal with Alentis for autoimmune drug with broad potential

    CSL will pay Alentis Therapeutics $355 million upfront, with up to $1.2 billion more, to co-develop and co-promote the claudin-1-targeting antibody lixudebart. The drug is in the Phase 2 RENAL trial for AAV-RPGN, a rare autoimmune kidney disease, and is also planned for mid-stage development in FSGS and PSC, with CSL funding these studies and any approved product profits split 55/45 in CSL's favour. An FDA orphan drug designation for IPF, granted in May 2024, sits outside the deal.

Aug 21Fri
Aug 19Wed
  1. biocentury.com42

    Aiming for larger rare disease market, BioMarin buys Alesta program

    BioMarin has acquired Alesta Therapeutics' program for hypophosphatasia, adding an oral therapy to its skeletal disease portfolio through an acquisition-and-spinout deal. Alesta's asset could serve what may become BioMarin's largest rare disease market to date. The deal follows BioMarin's $4.8 billion acquisition of Amicus Therapeutics under CEO Alexander Hardy and CBO James Sabry, who plan continued dealmaking.

Aug 18Tue
  1. fiercepharma.com58

    CSL shares up 17% as immunoglobulin boost points to signs of recovery

    CSL shares rose more than 17% after the Australian plasma and vaccine specialist reported fiscal 2026 sales of $15.8 billion and projected mid-single-digit growth for its CSL Behring unit in FY27. Immunoglobulin demand increased 7% year over year, and the HAE drug Andembry brought in $240 million. Vifor Pharma sales are projected to decline 25% in FY27 after $7.1 billion in impairments.

  2. fiercepharma.com54

    Sanofi to lay off 229 Blueprint Medicines staffers, close Cambridge offices following acquisition

    Sanofi is laying off 229 Blueprint Medicines employees in Massachusetts and closing two legacy Blueprint sites in Cambridge, including the former global headquarters at 45 Sidney St. The cuts, detailed in a WARN notice, follow Sanofi's $9.1 billion acquisition of Blueprint, which brought the rare immunology drug Ayvakit and KIT inhibitor candidates. Layoffs will occur between October 2026 and June 2027, and remaining Blueprint staff will move to Sanofi's Cambridge Crossing campus.

Aug 17Mon
CBACBA Frontier Insights (CFI)Chinese Biopharmaceutical Association (CBA)www.cba-usa.org