Welcome back to your monthly news roundup, where we look back at the biggest stories to hit the pharma headlines. It’s time to reflect on September 2026
There was some welcome respite for the UK’s pharma sector this month, with investment showing signs of a rebound. Foreign direct investment reached £167.2m in the first half of 2026, which is more than double the £88.8m recorded across the whole of 2025.
However, the rebound follows a sluggish period: the ABPI estimates that slower growth since 2020 has left around £3.8bn in potential investment unrealised.
The UK is also up against countries that are actively sharpening their offers. Ireland, for example, raised its R&D tax credit from 30% to 35% this year, while Japan introduced a strategic-technology tax credit offering at least 40% relief.
That makes this month’s warning from nine major pharmaceutical companies – that Europe is losing ground to the US and China – particularly pertinent. The UK may have stopped the slide, but attracting investment is becoming an increasingly competitive global contest.
One area attracting investment is advanced genetic medicine, but the economics of bringing these treatments to market are becoming increasingly difficult to overlook. Ultragenyx’s decision to price Fayuvi (rebisufligene etisparvovec-hopf) at $3.95m has put that debate firmly back in the spotlight.
The one-time gene therapy, approved for Sanfilippo syndrome type A, is now among the most expensive medicines in the US – but it is not an isolated example. Lenmeldy (atidarsagene autotemcel), approved for metachromatic leukodystrophy in 2024, has a list price of $4.25m, while CSL Behring’s haemophilia B therapy Hemgenix (etranacogene dezaparvovec) launched at $3.5m.
The argument from manufacturers is that a one-off treatment should be judged against the lifetime cost of managing a devastating disease. Ultragenyx puts the lifetime cost of caring for a child with Sanfilippo syndrome at around $8m.
But that shifts rather than removes the problem for payers: the multimillion-dollar bill arrives upfront, while the benefits may stretch decades into the future. As more gene therapies reach the market, value-based payment models will become increasingly important – but so too will the question of who should bear the financial risk.
CAR-T therapy has spent the past few years moving beyond blood cancer and into autoimmune disease, but Novartis’ decision to pause eight trials this month is a stark reminder that the technology’s risks have not disappeared.
The Swiss drugmaker paused studies of its experimental therapy after three patients died following severe immune reactions. The affected trials span conditions including lupus, systemic sclerosis, rheumatoid arthritis and multiple sclerosis.
The wider evidence shows why safety remains so important as CAR-T moves into new disease areas. A recent systematic review of 38 studies and 115 patients with autoimmune disease found cytokine release syndrome in 70.4% of patients, most of it low grade, and neurotoxicity in 4.3%. The authors stressed that the evidence base remains nascent.
The setback is particularly significant given the number of companies now testing CAR-T in autoimmune disease, and raises questions about how the field will approach safety as trials move into larger patient populations.
The MHRA’s new health innovation sandbox in Manchester also deserves a mention. The programme will test emerging technologies, including AI-enabled medical devices, in real NHS settings. The scheme’s ambitions extend beyond individual technologies: as the UK looks to strengthen its life sciences sector and accelerate digital adoption across the NHS, giving innovations a route to generate real-world evidence could help tackle two stubborn challenges at once.
Featured image: EMJ
Each article is made available under the terms of the Creative Commons Attribution-Non Commercial 4.0 License.
