Life science news 14 September 2026: two US approvals for rare diseases, a landmark antibody-drug conjugate readout, and two UK moves on pharmacy access and AI oversight.
Scholar Rock’s Isembyld won FDA approval, described by the agency as the first SMA therapy to target muscle loss directly. Novo Nordisk had a mixed week, halting two heart trials while reporting strong paediatric obesity data. Hundreds of participating pharmacies in England will treat five more common conditions from this autumn.
Scholar Rock’s Isembyld won FDA approval on 11 September 2026. The therapy treats spinal muscular atrophy in adults and children aged two and older. Patients must already be receiving an SMN2-targeted treatment such as risdiplam or nusinersen. Isembyld, known chemically as apitegromab-mstn, is a myostatin inhibitor. It is given by monthly infusion. It works by targeting muscle loss directly, alongside existing SMN-boosting drugs rather than replacing them. Scholar Rock describes it as the first and only muscle-targeted treatment approved for SMA in the United States. It addresses a part of the disease that older therapies do not reach.
The pivotal SAPPHIRE trial enrolled 188 patients aged two to twenty-one who could not walk or move independently. The primary analysis covered 156 of them, aged two to twelve. Those given the 10 mg/kg dose were more than twice as likely as placebo patients to show a clinically meaningful motor function improvement. The rate was 34.2% against 13.5% on placebo. The approval was supported by earlier studies in the programme, including the TOPAZ and ONYX trials. The FDA also recorded an increased risk of fractures, including serious fractures, in treated patients. It says the drug may cause fetal harm and may affect reproductive function. The Muscular Dystrophy Association’s chief research officer, Angela Lek, called the approval “what progress looks like”. She cited the charity’s long investment in the underlying science.
Pharming’s Joenja gained FDA approval on 11 September 2026 for children aged four to eleven weighing at least 27 kg. The oral drug treats activated phosphoinositide 3-kinase delta syndrome, a rare inherited immune disorder known as APDS. Joenja, or leniolisib, is a selective PI3K delta inhibitor. It is taken twice daily at 40 mg or 50 mg doses depending on the patient. It was already approved for adults and adolescents in the US. Pharming says the extension makes it the first FDA-approved APDS treatment for this younger age group. That closes a gap in paediatric care for the rare disorder.
A 12-week open-label, single-arm Phase III study in children aged four to eleven showed reduced lymphadenopathy and increased naïve B cell counts. Both are markers of the underlying immune dysfunction seen in APDS. All treatment-emergent adverse events recorded were mild to moderate. No drug-related serious adverse events were reported during the study period. Pharming’s chief commercial officer, Leverne Marsh, called it “an important achievement” in expanding access to Joenja for people living with APDS. Dosing for children weighing between 13 kg and 27 kg remains under separate regulatory review, Pharming said. A decision on that group is expected at a later date.
Novartis said on 8 September 2026 that its Phase III HARBOR study had missed its main goal. The trial tested del-desiran, also known as delpacibart etedesiran, in myotonic dystrophy type 1. It is an antibody oligonucleotide conjugate. The molecule pairs a muscle-targeting antibody with an siRNA aimed at toxic DMPK messenger RNA. This is a novel approach in this rare muscle-wasting disease. The study measured video hand opening time against placebo over the treatment period. It did not reach statistical significance on that primary endpoint, Novartis confirmed, marking a second Novartis trial setback within days.
Novartis said safety findings were generally consistent with earlier data from the programme. It pointed to signs of clinical activity on secondary and exploratory measures, though it gave no comparator figures for these. Shreeram Aradhye, the company’s president of development and chief medical officer, said “setbacks are part of scientific progress” in a complex disease like DM1. He was speaking as Novartis confirmed the result. The company is now reviewing the full HARBOR dataset in detail. It will discuss next steps for the del-desiran programme with health authorities in the coming months.
Novo Nordisk informed trial investigators on 4 September 2026 that it was halting two Phase III heart trials of ziltivekimab. The decision was first reported on 7 September. The HERMES and ATHENA studies were stopped after a data monitoring committee found them unlikely to succeed. Ziltivekimab is an anti-IL-6 antibody designed to lower inflammation as a route to better cardiovascular outcomes in heart failure patients. The decision follows the drug’s earlier failure in the ZEUS trial, reported previously. No named company statement accompanied the disclosure. The halt surfaced through trade media rather than a formal company release. Life Science Daily News could not locate a Novo Nordisk press notice confirming it directly.
In ZEUS, ziltivekimab lowered inflammatory markers but did not cut the risk of major complications, including death and heart attack. Independent reporting by STAT News, Endpoints News and Clinical Trials Arena corroborated both the halt and the futility rationale behind it. The result adds to a run of setbacks for anti-inflammatory approaches to cardiovascular disease. Three of the four Phase III cardiovascular studies of ziltivekimab are now negative or discontinued. ARTEMIS is testing the drug as an acute treatment after a heart attack. It will continue, with a readout expected in the first half of 2027. That still leaves Novo Nordisk’s cardiovascular pipeline outside diabetes and obesity looking noticeably thinner heading into next year. Life Science Daily News covered the halt of the two IL-6 heart failure trials in full last week.
In further life science news 14 September 2026, the week also brought positive paediatric obesity data, a major antibody-drug conjugate readout, and two UK regulatory moves.
A separate Novo Nordisk trial brought better news the same week. On 7 September 2026, the company reported first results from STEP Young. The Phase III study tested semaglutide in 165 children aged six to under twelve with obesity. Most participants, over 85%, had class II or III severe obesity at baseline. The trial met its primary endpoint of percentage change in body mass index at week 68. On a confirmatory secondary endpoint, 40.4% on semaglutide plus lifestyle changes were no longer classified as having obesity after 68 weeks. None of the placebo group reached that mark. That figure rests on the trial product estimand, which estimates the effect if all children had adhered to treatment.
Novo Nordisk said safety and tolerability were consistent with adult and adolescent semaglutide trials already on file with regulators. No new safety concerns were identified, including none affecting growth or pubertal development over the study period. Martin Holst Lange, the company’s chief scientific officer, said Novo Nordisk was “encouraged by the positive STEP Young results”. He pointed to semaglutide’s potential to help children living with obesity who need medical treatment. Both trial arms received structured diet and physical activity support throughout the 68-week study. The announcement was topline only, with no mean change in body mass index, treatment difference or confidence intervals released. Detailed results are due at ObesityWeek 2026 in November.
Roche’s partner MediLink reported positive interim Phase III data on 13 September 2026 for the antibody-drug conjugate Tam-Peli. The randomised, open-label TAISHAN-302 trial enrolled 451 patients in China with relapsed small-cell lung cancer after platinum-based chemotherapy. Tam-Peli, known as tambotatug pelitecan, targets B7-H3. It pairs the antibody with a topoisomerase 1 inhibitor payload on MediLink’s TMALIN platform. Roche holds development and commercial rights to the drug outside mainland China, Hong Kong and Macau under an existing licensing agreement. It plans to build on the Chinese data with trials in its own licensed territories.
Median overall survival reached 13.3 months on Tam-Peli against 9.4 months on topotecan. That is a hazard ratio of 0.46, a 54% reduction in the risk of death. Progression-free survival ran to 7.4 months against 2.8 months, and the objective response rate was 59.1% against 9.7%. Roche’s chief medical officer, Levi Garraway, said the result “reinforces our confidence in its potential to improve outcomes” for people with cancer. He was commenting on the TAISHAN-302 data specifically. Roche now plans to start global Phase III trials of Tam-Peli across its licensed territories. This is a second positive Phase III readout, after TAISHAN-301 in nasopharyngeal carcinoma.
NHS England said on 10 September 2026 that hundreds of participating pharmacies will treat five more common conditions from this autumn. The Pharmacy First scheme will add low-frequency episodic migraine in adults and seasonal allergic rhinitis in children. It will also cover acute otitis externa, mild to moderate acne, and mild skin and soft tissue infections and scabies. The service starts with the small number of pharmacies that qualify as prescribing pharmacies in 2026/27. Patients using those pharmacies will not need a GP appointment first. NHS England says evidence suggests 2 to 3 million extra consultations a year once the expansion is fully rolled out. That would sit alongside the 14 million already delivered under the wider scheme.
Dr Amanda Doyle is NHS England’s national director for primary care and community services. She said the change would free up GP capacity “in the colder months”. Health and Social Care Secretary Yvette Cooper said community pharmacies were “vital to shifting care into the community”. Rob Music, chief executive of The Migraine Trust, welcomed the move. He said patients with migraine “can struggle to access timely care”, pointing to research the charity had carried out. Community Pharmacy England has warned separately that no dedicated funding is attached to the prescribing service. It says the initiative has been set up to fail as things stand. Around 10,000 community pharmacies operate across England. Some 14 million Pharmacy First consultations have taken place since the scheme launched in February 2024.
An independent commission led by NHS doctors set out a blueprint on 10 September 2026. The MHRA established the National Commission into the Regulation of AI in Healthcare in September 2025. It is an independent, non-statutory advisory body. Professor Alastair Denniston, an NHS consultant, chaired the group and launched the report alongside Professor Henrietta Hughes. It recommends staged authorisations for new AI models, likened to L-plates for learner drivers. New models would run under close supervision and tight guardrails before full approval. It also wants continuous real-world monitoring of AI-enabled medical devices throughout their working lives. That would replace a one-off check at the point of approval, catching problems that only emerge in routine use.
Other recommendations include public access to safety information on specific AI devices and any adverse incidents linked to them. The commission also wants clearer rules on telling patients when AI is used in their care. It called for stronger enforcement powers for the MHRA too. Professor Denniston said people are open to AI improving their care, but “only if it is safe, overseen by humans” and used with their knowledge. Government and the MHRA will formally respond to the recommendations in due course, the announcement said.
That’s your round-up of life science news 14 September 2026, back next Monday with the latest from pharma, biotech and healthcare at www.lifesciencedaily.news.
Catch up on the 7 September news roundup.
