May 2026 • PharmaTimes Magazine • 9
// CONDITIONS //
FDA approves new high-dose Spinraza regimen for SMA
Biogen has received approval from the US Food and Drug Administration for a new high-dose regimen of Spinraza for the treatment of spinal muscular atrophy (SMA).
The company said the regimen, which includes 50mg and 28mg doses, was designed to deliver a higher concentration of the drug during both loading and maintenance phases.
The approval is based on data from the phase 2/3 Devote study, which evaluated the efficacy and safety of the high-dose regimen in both treatment-naïve patients and those previously treated with Spinraza.
The company said the new regimen will be available in the United States in the coming weeks and is already approved in the European Union, Switzerland and Japan.
Richard Finkel, director of the Center for Experimental Neurotherapeutics at St Jude Children’s Research Hospital, said: “Optimising the dose of nusinersen builds on a therapy that we already know can change lives.
“The high-dose regimen demonstrated meaningful clinical benefit while maintaining a well-characterised safety profile.” He added: “I believe high-dose Spinraza will play an important role in the future of SMA care.”
The Devote study showed that treatment-naïve infants receiving the high dose experienced statistically significant improvements in motor function compared with a prespecified matched sham group. Biogen said the safety profile was generally consistent with the known safety of the low-dose regimen.
Cure Parkinson’s convenes expert panel to assess funding call
Cure Parkinson’s has announced the formation of a new evaluation panel to review applications submitted to its recent £2 million funding call for projects investigating combination therapies for Parkinson’s. The panel will meet in April, with final funding decisions expected in May.
The charity said proposals include both preclinical and clinical studies testing rationally designed treatment combinations. The panel will assess whether the projects are scientifically robust, aligned with Cure Parkinson’s mission and capable of generating results that matter to the Parkinson’s community.
To ensure rigorous evaluation, the group brings together expertise in drug discovery, statistics, pharmacology and clinical trials.
Chaired by Professor Fiona Ducotterd, Chief Scientific Officer of the Alzheimer’s Research UK UCL Drug Discovery Institute, the panel includes academics, industry specialists, clinicians and people with Parkinson’s. Members are Ian Reynolds, Kevin McFarthing, Kalpana Merchant, Dave Weiner, Peter Debiaso, Caroline Williams-Gray, James Wason, Sue Learned, Jérémie Nsengimana, Camille Carroll and Michele Hu.
Combination therapies use two or more active treatments that may target different pathways or enhance each other’s effects. They are already used to manage Parkinson’s symptoms, such as co-careldopa, which combines levodopa and carbidopa. Cure Parkinson’s said it hopes to encourage further research into whether such approaches could also modify the course of the disease.
Johnson & Johnson has announced a landmark regulatory decision that will allow people living with multiple myeloma to administer the subcutaneous formulation of daratumumab themselves, or have it given by a caregiver, from the fifth dose.
The Committee for Medicinal Products for Human Use has approved a Type II variation to the medicine’s labelling, making it the first oncology injectable in Europe cleared for self-administration.
Viridian Therapeutics has announced encouraging topline results from its REVEAL-1 phase 3 clinical trial evaluating elegrobart in patients with active thyroid eye disease.
The study tested two subcutaneous dosing schedules – every four weeks and every eight weeks – against placebo. According to the company, both regimens achieved clinically meaningful improvements in proptosis, with responder rates of 54% and 63% respectively, compared with 18 percent for placebo at week 24.
Positive high-level results from the phase 3 OBERON and TITANIA trials indicate that AstraZeneca’s tozorakimab achieved statistically significant and clinically meaningful reductions in the annualised rate of moderate-to-severe COPD exacerbations compared with placebo.
The findings applied both to the primary population of former smokers and to the overall population, which included former and current smokers, with patients spanning all blood eosinophil counts and all stages of lung function severity.
CatalYm has dosed the first patient in its global GDFATHER-HCC-01 study, a phase 2b trial evaluating visugromab in combination with chemoimmunotherapy for people with unresectable or metastatic hepatocellular carcinoma who have progressed after first-line anti-PD-(L)1 therapy.
The study will test visugromab alongside nivolumab and the tyrosine kinase inhibitor lenvatinib. It begins with an open-label safety run-in to confirm the recommended dose for expansion.
Recent NHS setbacks include rising waiting lists, renewed strike action by junior doctors and warnings from hospital leaders that services are becoming increasingly unsafe. Financial pressures have deepened, with several Trusts reporting severe budget gaps and delays to planned upgrades.
Emergency departments continue to miss key targets, and staff shortages are worsening morale. Critics say the system is struggling to recover, leaving patients facing longer delays and reduced access to care.
Responding to the news that the BMA Resident Doctors Committee has rejected the government's offer and called for six days of strike action, NHS Confederation’s Rory Deighton, said:
"These strikes not only cause appointments to be cancelled and patients to have to wait longer for tests, treatment and surgery, but also cost up to £300m each time – money that would be better spent on patient care. They also undermine the capacity NHS leaders and their teams have to modernise and transform services.”
PharmaTimes
PharmaTimes is the UK’s leading pharmaceutical magazine, tracking the trends and issues that affect the industry here in the UK, in Europe and across the world. We deliver independent, authoritative and trustworthy content through impartial, balanced and informed opinion and analysis.