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INSIGHTS+

EMA Marketing Authorization of New Drugs in April 2026    

Shots:  The European Medicines Agency advanced multiple approvals and late-stage recommendations in April 2026 across infectious diseases, neurology, immunology, endocrinology, and rare disorders, involving companies including Merck, Moderna, Novartis, Sanofi, and Mitsubishi Tanabe Pharma  CHMP positive opinions included Redemplo for familial chylomicronemia syndrome, Itvisma for spinal muscular atrophy, and Cenrifki for non-relapsing SPMS  The EC also approved therapies including Enflonsia for RSV prevention in infants, mCOMBRIAX for influenza/COVID-19 prevention, Palsonify for acromegaly, Rhapsido for…
3 days ago

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NEWS

X4 Pharmaceuticals’ Xolremdi Receives the EC’s Approval for WHIM Syndrome 

Shots:  The EC has approved Xolremdi (Mavorixafor), under exceptional circumstances, for the treatment of WHIM syndrome  Approval was supported by the global pivotal P-III (4WHIM) trial, PBO-controlled 52-wk study evaluating mavorixafor’s efficacy & safety in pts ≥12 yrs with WHIM syndrome  Xolremdi (PO, QD), a CXCR4 antagonist, is used in pts ≥12 yrs with WHIM syndrome to increase circulating mature neutrophils & lymphocytes  Ref: X4…
April 27, 2026

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INSIGHTS+

EMA Marketing Authorization of New Drugs in February 2026    

Shots:  The European Commission approved multiple innovative therapies across vaccines, oncology, cardiology, immunology, and rare diseases, including products from Moderna, Hansoh Pharmaceutical, Cytokinetics, ImmunityBio, and GSK  The Committee for Medicinal Products for Human Use also issued positive opinions for late-stage assets from Ipsen, X4 Pharmaceuticals, Novartis, and Moderna, alongside additional recommendations for therapies targeting chronic spontaneous urticaria, WHIM syndrome, pediatric…
March 17, 2026

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NEWS

X4 Pharmaceuticals’ Xolremdi (Mavorixafor) Receives the CHMP Positive Opinion for WHIM Syndrome

Shots: The CHMP has recommended approval of Xolremdi, under exceptional circumstances, for the treatment of WHIM syndrome, with EC’s decision expected in Q2’26 Opinion was backed by the global P-III (4WHIM) trial assessing Xolremdi vs PBO in 31 pts (≥12yrs.) with WHIM syndrome, which showed improved absolute lymphocyte counts, absolute neutrophil counts, & fewer infections,…
March 2, 2026

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PharmaShots Interview X4 Pharmaceuticals’ Paula Ragan Shares Insights on Mavorixafor for the Treatment of Multiple Immunodeficiencies
VIEWPOINTS

PharmaShots Interview: X4 Pharmaceuticals’ Paula Ragan Shares Insights on Mavorixafor for the Treatment of Multiple Immunodeficiencies

In an interview with PharmaShots, Paula Ragan, Ph.D., CEO, and President at X4 Pharmaceuticals shared her views on the data of Mavorixafor which showed increased immune cell levels regardless of patients having a CXCR4 mutation to treat multiple immunodeficiencies, presented at ASH 2021 Shots: The ongoing long-term extension arm of the P-II trial evaluates Mavorixafor (CXCR4 antagonist) in patients with WHIM syndrome. The therapy continues…
May 3, 2022

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Viewpoints_Paula Ragan
VIEWPOINTS

Pharmashots Interview: Paula Ragan Shares Insights on the Data of Mavorixafor in Combination with Ibrutinib Presented at 2021 EHA Annual Congress

In an interview with PharmaShots, Paula Ragan, Ph.D., President, and Chief Executive Officer at X4 Pharmaceuticals shared her views on preliminary efficacy and safety data of Mavorixafor + Ibrutinib in an ongoing P-Ib study to treat waldenström's macroglobulinemia Shots: The ongoing P-Ib clinical trial evaluates the safety & efficacy of mavorixafor + ibrutinib in patients with WM with both MYD88 &…
September 3, 2021

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