Pharmaceutical Market Europe • July/August 2025 • 8-9
NEWS
Pfizer shares phase 3 results for haemophilia therapy Hympavzi
Pfizer has shared positive top-line results from a late-stage study of its haemophilia A and B therapy Hympavzi (marstacimab).
The phase 3 BASIS trial has been evaluating the drug, which already holds approvals to treat haemophilia A and B patients without inhibitors, in adult and adolescent patients with inhibitors, which neutralise factor replacement therapies and make them ineffective.
The study met its primary endpoint, with once-weekly prophylactic treatment with Hympavzi resulting in a statistically significant and clinically relevant reduction in annualised bleeding rate (ABR) of treated bleeds compared to on-demand treatment.
Patients were treated with Hympavzi during a 12-month period versus an on-demand intravenous regimen with bypassing agents, given as part of usual care in a six-month lead-in period.
Hympavzi reduced ABR by 93% over 12 months and also demonstrated superiority across all bleeding-related secondary endpoints, including spontaneous bleeds, joint bleeds, target joint bleeds and total bleeds.
Pfizer said it is planning to discuss the latest results with regulatory authorities, “with the goal of initiating regulatory filings for Hympavzi for the treatment of patients living with haemophilia with inhibitors”.
First NHS patient treated with CSL’s haemophilia B gene therapy
The first patient in the UK has been treated by the NHS with CSL Behring’s haemophilia B gene therapy Hemgenix (etranacogene dezaparvovec).
The patient from the North East of England was treated at Guys & St Thomas’s NHS Foundation Trust in London and may now no longer require regular treatment with intravenous clotting factor IX (FIX).
Affecting more than 2,000 people in the UK, haemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of FIX.
CSL’s Hemgenix addresses the underlying genetic cause of haemophilia B by enabling the body to continuously produce FIX, and is the only one-time gene therapy to be approved in the UK for adults with severe or moderately severe haemophilia B without a history of FIX inhibitors.
Pu-Lin Luo, consultant haematologist at Guy’s and St Thomas’, said: “This is a big step forward in our ability to manage haemophilia B and could change the lives of some of our patients. It is also a testament to the advancement of cell and gene therapies in the UK and these are exciting times.”
Eli Lilly’s once-weekly insulin candidate shows promise for T2D
Eli Lilly has shared promising phase 3 results for its once-weekly insulin candidate efsitora alfa in adults with type 2 diabetes (T2D).
The QWINT-1 trial, which evaluated efsitora alfa in patients using insulin for the first time, showed that Lilly’s candidate reduced A1C by 1.31% compared to 1.27% for insulin glargine at week 52. In the study, efsitora was titrated across four fixed doses at four-week intervals, as needed for blood glucose control.
In QWINT-3 and QWINT-4, which enrolled patients who had previously used daily basal insulin, and those who had previously used daily basal insulin and mealtime insulin, respectively, efsitora was given using traditional insulin dosing, with adjustments based on each patient’s glucose level.
At week 26 in QWINT-3, efsitora reduced A1C by 0.86% compared to 0.75% for insulin degludec. At the same time point in QWINT-4, efsitora reduced A1C by 1.07% compared to 1.07% for insulin glargine.
Lilly said it is planning to submit global regulatory applications for efsitora as a treatment for adults with T2D by the end of this year.
FDA unveils new priority review voucher programme
The US Food and Drug Administration (FDA) has unveiled a new programme that will reduce its review time for certain drug applications from around ten to 12 months to just one to two months.
The Commissioner’s National Priority Voucher programme for companies “supporting US national interests” will convene experts from FDA offices for a team-based review, instead of using the standard system of a drug application being sent to multiple FDA offices.
It will also require sponsors to submit the chemistry, manufacturing and controls portion of the application and the draft labelling at least 60 days before submitting the final application.
FDA commissioner, Marty Makary, said: “Using a common-sense approach, the national priority review programme will allow companies to submit the lion’s share of the drug application before a clinical trial is complete so that we can reduce inefficiencies.”
In the first year of the programme, the FDA will give a limited number of non-transferable vouchers to companies aligned with priorities such as addressing unmet public health needs, delivering more innovative cures and increasing domestic drug manufacturing.
MHRA becomes founding member of global AI network
The Medicines and Healthcare products Regulatory Agency (MHRA) has become a founding member of a global network of health regulators focused on the safe and effective use of artificial intelligence (AI) in healthcare.
The UK is now the first country in the world to join the HealthAI Global Regulatory Network, which will aim to build trust in the technology and accelerate innovation.
Ten ‘pioneer countries’ have been invited to shape the network and will work together to share early warnings on safety, monitor how AI tools perform in practice and build international standards.
A signing ceremony at Westminster in London marked the MHRA’s membership and was attended by science minister Lord Patrick Vallance (pictured left), MHRA chief executive Lawrence Tallon (pictured right) and HealthAI chief executive officer Ricardo Baptista Leite (pictured middle).
Tallon said: “AI has huge promise to speed up diagnoses, cut NHS waiting times and save lives – but only if people can trust that it works and is safe. That’s why we’re proud to be leading the way, shaping how this powerful technology is used safely in healthcare here and around the world.”
UCB unveils late-stage data for fenfluramine in ultra-rare epilepsy
UCB has shared promising results from a late-stage study of fenfluramine in patients with an ultra-rare form of epilepsy.
The phase 3 trial has been evaluating adjunctive fenfluramine in 87 patients aged one to 35 years who have a CDKL5 deficiency disorder (CDD) diagnosis and uncontrolled seizures.
Occurring in approximately one in 40,000 to 60,000 live births, CDD is a developmental epileptic encephalopathy caused by variations in the CDKL5 gene.
The study met its primary endpoint, based on the median percent change in countable motor seizure frequency between baseline and the titration plus maintenance phase compared to placebo.
UCB’s drug was also found to be well tolerated, and its safety profile was consistent with previous studies in its approved indications, Dravet and Lennox-Gastaut syndrome.
A long-term 52-week extension phase of the study is currently ongoing, with the aim of characterising the long-term safety and tolerability of fenfluramine in adult and paediatric CDD patients.
The company said it is planning to submit regulatory applications for fenfluramine in CDD to bring the drug to patients “as soon as possible”.
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