Our CEO Paula Ragan, Ph.D. recently engaged in a fireside chat with Jeanne Siegel, head of the National Neutropenia Network, Inc. , focused on X4’s efforts to expand treatment options for those with chronic neutropenic conditions. #neutropenia #RareResolve
X4 Pharmaceuticals
Biotechnology Research
Boston, Massachusetts 13,930 followers
Enabling a better future for people with rare immune disorders
About us
We are a fully integrated, commercial-stage biopharmaceutical company driving progress for people living with rare immune disorders. We listen to people whose challenges have been long overlooked, understand their needs, and develop solutions to their most urgent and critical health concerns. Our FDA-approved medication XOLREMDI® (mavorixafor) is commercially available in the U.S. in its first indication. We are evaluating mavorixafor in additional potential indications, and have an ongoing global, pivotal Phase 3 clinical trial for its use in chronic neutropenia. Our corporate headquarters is in Boston, Massachusetts.
- Website
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http://www.x4pharma.com
External link for X4 Pharmaceuticals
- Industry
- Biotechnology Research
- Company size
- 51-200 employees
- Headquarters
- Boston, Massachusetts
- Type
- Public Company
- Founded
- 2014
- Specialties
- Rare disease and primary immunodeficiencies
Locations
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Primary
Get directions
61 N Beacon St
Boston, Massachusetts 02134, US
Employees at X4 Pharmaceuticals
Updates
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Proud to launch our WHIM Syndrome Patient Education Program at this year’s National Neutropenia Network, Inc. Conference highlighting the importance of genetic testing and early diagnosis to support improved health outcomes. #WHIMsyndrome https://whimsyndrome.com/
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ICYMI – our CFO, Adam Mostafa, was recently interviewed by Informa Connect – learn more about his unique journey from Wall Street to biotech: https://lnkd.in/eebM4qSu
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Over the weekend, we presented positive data from our completed Phase 2 trial of mavorixafor for the treatment of chronic #neutropenia (CN) at the European Hematology Association (EHA) Congress. Data highlights include: · Sustainable increases in absolute neutrophil count (ANC) to maintain clinically targeted levels. · Reduced or discontinued use of the current standard of care for primary CN, injectable G-CSF, by physicians administering mavorixafor. These results give us a first glimpse into the potential real-world use of mavorixafor in a market currently served by only one approved therapy. Read more in today’s press release: https://lnkd.in/eNUtH2tV #EHA2025
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Thrilled to share the FDA has granted Fast Track designation to mavorixafor for the treatment of chronic #neutropenia (CN). This designation will allow X4 more frequent communication with the agency and the potential for accelerated approval and/or priority review if certain criteria are met and maintained. Mavorixafor is currently in a Phase 3 clinical trial (4WARD) in certain primary CN conditions. Learn more in today’s release: https://lnkd.in/e8m-XGZM #FDA #raredisease #immunodeficiencies #chronicneutropenia #clinicaltrials #FastTrackdesignation
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We are proud to receive the 2025 Industry Innovation Award presented by the National Organization for Rare Disorders (NORD), as part of its annual Rare Impact Awards! These awards honor innovative companies whose treatments have improved outcomes for people living with rare diseases. At the heart of our #RareResolve is our tenacious focus on driving progress for individuals living with rare immune disorders. Thank you to NORD for this recognition, and congratulations to all the honorees. View the full list of winners here: https://rareimpact.org/ #RareImpactAward #RareDiseases #WHIMsyndrome #chronicneutropenia #immunodeficiencies
Meet the 2025 #NORD Rare Impact Award Honorees! 🎉 This year’s #RareImpactAward recipients are an inspiring group of individuals and organizations driving meaningful change in #RareDisease care, innovation, and support. 🏅 Community Champions • Robert Graham (NY): A pillar of support and advocacy in New York’s rare disease community. • Jaime McHugh (NH): A tireless advocate, policy driver, and two-time Running for Rare Boston Marathon participant. • Mary Nadon Scott (VT): A founding force behind Vermont’s Rare Disease Advisory Council Coalition. 🔬 Medical & Scientific Trailblazers • Dr. Angela Scheuerle (TX): Director at UT Southwestern Medical Center's NORD Rare Disease Center of Excellence. • Dr. Jerry Vockley (PA): Co-Director at UPMC Children's Hospital of Pittsburgh's NORD Rare Disease Center of Excellence and a leader in rare disease research. 🏆 The Abbey S. Meyers Leadership Award • Hermansky-Pudlak Syndrome Network: Led by Donna and Ashley Appell, this organization representing #HermanskyPudlak syndrome patients exemplifies leadership and resilience in advocacy. 💡 Industry Innovation Award Winners • argenx – VYVGART Hytrulo® • Ascendis Pharma – YORVIPATH® • BridgeBio – Attruby™ • Ionis Pharmaceuticals, Inc. – TRYNGOLZA™ • Orchard Therapeutics - U.S. – LENMELDY™ • PTC Therapeutics, Inc. – KEBILIDI™ • X4 Pharmaceuticals – XOLREMDI® Each honoree's journey is a powerful testament to the impact of purpose-driven work in the rare disease community. Discover the extraordinary contributions they’re making at rareimpact.org
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