Biotech, in brief

The FDA, the US regulator for medicines and medical devices, approved the Autus Size-Adjustable Valve on 1 October. [1][2]

It replaces the pulmonary valve in children born with a faulty one. This valve lets blood leave the right side of the heart for the lungs, and stops it flowing back. [1]

About 4,200 babies a year are born in the US with a pulmonary valve that is too narrow or never formed properly, the FDA says. [1]

Until now, no replacement valve could be widened after surgery. As the child grows it becomes too small, and a bigger one has meant another open-heart operation. [1][2]

The Autus valve can go in as small as about 13 millimetres, the right size for a toddler. [1]

Later, doctors widen it with a small balloon threaded in through a blood vessel, up to 22 millimetres, about the size of an adult's valve. [1][2]

Its flaps are made of a man-made material instead of animal tissue, which hardens and wears out faster in children. No valve with flaps like these had been approved before. [1]

The approval rests on a study of 62 children at 12 US hospitals. Every operation succeeded, with no deaths, strokes or blood clots that needed treatment. [1]

Three valve frames cracked and two flaps moved less than they should, but none of these children had symptoms. [1]

Two children began to outgrow their valves, and both had them widened without surgery. The FDA says experience with widening is still limited. [1]

Edwards Lifesciences, a California maker of heart devices, holds the approval, and a company called Autus Valve Technologies developed the valve. [1][2] Edwards says the children will be followed for 10 years. [2]

Why it matters. The next test is time: whether stretching the valve with a balloon keeps it working as children keep growing, which the FDA says longer studies are still checking. [1]

Sanofi and Regeneron, the two drugmakers behind Dupixent, said on Thursday that they will expand their partnership. [3][4] Sanofi will pay Regeneron $1 billion up front, and up to $7 billion more if the new drugs pass set tests. [4] Dupixent, a medicine for eczema and asthma, sold $10.9 billion in the first half of this year. [3] Key patents protecting it run out over the next five years. [3] The deal covers four longer-lasting antibodies aimed at the same targets, and only one has reached human testing. [3]

Why it matters. When the patents end, other companies can make close copies of Dupixent. [3] An analyst at RBC Capital Markets, an investment bank, said the new drugs are still very early, with no substantial data yet. [3]

The US health department launched new programmes on Wednesday to speed up the testing of new medicines. [5] The main one, SURPASS, is a five-year programme run by ARPA-H, the department's research funding agency. [6] It asks teams to design testing that runs as one continuous process, instead of separate phases with paperwork between them. [5][6] A new drug now takes about 10 years and $1 billion to $2 billion to develop, and more than 90% fail, the department says. [5]

Why it matters. China passed the US in its share of early-stage trials in 2021. [5] In Australia, trials can start six to 12 months sooner and cost about half as much, a think-tank report says. [5] The announcement did not say how much money the programme has. [6]

STAT, a US health news site, sent a reporter to a two-day biotech summit in Shanghai's Science City district. [7][8] Drugs designed in China now make up about a third of all drugs being developed worldwide, and draw more than half of all licensing and partnership deals. [7] Zhui Chen, a co-founder of Abbisko, a Shanghai company now worth $6 billion, said it ran a trial for Germany's Merck KGaA in 11 months. [8] Merck's managers then asked whether Abbisko could train their staff. [8]

Why it matters. China's ruling Communist Party expects its drugmakers to have at least five blockbusters by 2030, STAT reports. [7] A blockbuster is a drug that sells more than $1 billion a year. That is the competition the new US trial programmes are meant to answer. [5]

Daraxonrasib, a pill approved in the US in August, roughly doubled how long people with advanced pancreatic cancer typically lived. [9] But it is not a cure, and it can suddenly stop working after about seven months. [9] Ben Sasse, a former US senator from Nebraska, said in late September that his stage 4 cancer had stopped responding to it. [9] On 25 September, at a cancer meeting in San Diego, researchers presented early clues from tumour samples taken from 66 patients. [9]

Why it matters. Nearly every pancreatic cancer patient on the drug eventually becomes resistant to it, said Andrew Aguirre, a cancer doctor at the Dana-Farber Cancer Institute in Boston. [9] The researchers hope that learning how the cancer escapes will help them keep patients alive longer. [9]

AstraZeneca has asked the FDA to approve two pills taken together, Orpathys and Tagrisso, for a kind of lung cancer, its Chinese partner HUTCHMED said on 29 September. [11] Tagrisso blocks EGFR, a faulty growth signal that drives some lung cancers. [11] One of the commonest ways those cancers start growing again is by turning up a second signal, MET, which Orpathys blocks. [11] In a final-stage trial called SAFFRON, the pair held the cancer back and kept patients alive longer than chemotherapy. [11]

Why it matters. The combination is already approved in China, on the strength of an earlier Chinese trial. [11] If the FDA agrees, patients whose cancer grew despite Tagrisso could take two pills instead of chemotherapy. [11]

A modified cold virus called VCN-01 helped chemotherapy against pancreatic cancer that had spread, in a trial published in Nature Medicine on 30 September. [10] The virus infects cancer cells and makes an enzyme that breaks down part of the dense tissue around pancreatic tumours. [10] In 101 patients, those given the virus with chemotherapy typically lived 10.6 months, against 8.6 months on chemotherapy alone. [10] At 18 months, 31.1% of the virus group were alive, against 8.5% of the others. [10]

Why it matters. The survival gain passed the trial's main statistical test in one count of 96 patients, but not when all 101 were counted. [10] Serious side effects hit 22.6% of those given the virus, and the authors say a larger, blinded trial is needed. [10]

Medicare Advantage plans are private plans that older Americans can choose instead of the US government's own Medicare. The insurers that run them are raising what members pay out of their own pockets in 2027. [16] They are also cutting money for dental work and other extras, and dropping plans with wide choices of doctors and hospitals, STAT found in new federal data. [16] Big insurers such as UnitedHealth Group and Humana are doing this to raise profits, after investors complained that profits had come in lower than promised. [16]

Why it matters. Members who do not compare plans during the yearly sign-up period, which opens on 15 October, may find their doctor is no longer covered. [16] The cuts come even though the insurers won a payment increase from the Trump administration in April. [16]

At least 32 children have died since May in remote forest villages in Balaghat, a district of India's Madhya Pradesh state, officials say. [22] Most were Baiga, one of India's 75 tribal groups officially counted as particularly vulnerable. [22] Officials link about eight to 10 deaths to measles and about 10 to malaria, which may have been made worse by hunger and slow treatment. [22] The nearest state hospital is about 70km away, along dirt tracks that turn to mud in the monsoon. [22]

Why it matters. Records showed 90% to 95% of children vaccinated in some areas, but health workers found children with no records at all. [22] Officials then offered the measles vaccine to every child under 15, and say the outbreak has now slowed. [22]

About 1 in 13 adults aged 18 to 29 already had plaque, a fatty build-up, in at least one artery, in a scan study of 16,808 people in Denmark and Spain. [24] None had known heart or artery disease. [24] This build-up drives most heart disease, and it can grow for decades without any symptoms. [24] By ages 60 to 70, about 9 in 10 had some. [24] The study, called REACT, was published in the New England Journal of Medicine and paid for by the Novo Nordisk Foundation. [24]

Why it matters. The usual risk scores, built from age, blood pressure and cholesterol, found only a small share of the people who already had plaque. [24] Having plaque does not mean a heart attack will follow, and the researchers want quick ultrasound scans to find it early. [24]

Giving immunotherapy before surgery for advanced melanoma, a skin cancer, left most patients alive five years later, a pooled study of 1,038 patients from 26 hospitals found. [25] Immunotherapy drugs help the immune system attack a cancer. Five years on, 83.3% of those given one immunotherapy drug and 87.5% of those given two were alive. [25] Among patients whose tumours had mostly died by the time of surgery, about 98% were alive. [25] Patients given BRAF and MEK pills instead did worse, at 69.8%. [25]

Why it matters. Immunotherapy before surgery is already standard care for this stage of melanoma, and this is a five-year count of how it has done. [25] Patients whose tumours responded early gained nothing from more of the drug after surgery. [25]

The US Centers for Disease Control and Prevention plans to cut its yearly National Health Interview Survey for adults from 482 questions to 150, STAT reported on 1 October. [15] The survey has collected Americans' health data since 1957. [15] The redesign, proposed in August, drops questions about hearing aids, tiredness, thinking, and wheelchairs and scooters. [15] The US health department said the changes were driven by the cost of collecting data, but would not say why the disability questions went. [15]

Why it matters. More than a quarter of Americans have a disability, and advocates say the cuts will mean they are counted even less. [15] A paper co-written by CDC statisticians found the survey may already miss 75% of people with intellectual and developmental disabilities. [15]

Medicaid is the US health plan for people on low incomes. Nebraska's new work rule for Medicaid is already costing people their cover, an analysis by KFF found. [7] Fourteen percent of new applicants were turned down for not meeting the work rule. [7] Seven percent of people trying to renew lost their cover for the same reason. [7] From January, Nebraskans will have to send extra documents to prove they qualify. [7]

Why it matters. The state is also expected to narrow who counts as too frail to work, which could push the numbers higher. [7] STAT has reported that the rules are hard for residents to understand. [7]

A US federal judge ruled on 30 September that part of an investor lawsuit against UnitedHealth Group, a Minnesota-based health insurance company, can go ahead. [17] CalPERS, the pension fund for California's public employees, says UnitedHealth inflated its 2024 earnings by $3.3 billion to hide that its Medicare Advantage plans were doing worse. [17] Judge Jeffrey Bryan threw out most of the fund's other claims as too vague, or as ordinary sales talk. [17]

Why it matters. One of the lawsuit's central claims is that UnitedHealth made its Medicare Advantage patients look sicker on paper to get more money from the US government. [17] That is the same insurance business now cutting extras for 2027. [16]

Two more weight-loss injections reported results this week at the European diabetes meeting in Milan. [12] Boehringer Ingelheim, a German drugmaker, said people with obesity and type 2 diabetes who stayed on its shot survodutide lost 13% of their weight, against 3% on a dummy shot. [12] AbbVie's ABBV-295, which works on amylin, a digestive hormone,, took off up to 10% in 12 weeks in a small early trial. [12] Given once a month it took off 8%, and a third of people felt sick, against a fifth on the dummy. [12]

Why it matters. Both companies licensed these drugs from smaller firms: Boehringer from Zealand Pharma, and AbbVie from Gubra for $350 million. [12] These latecomers hope to compete with Wegovy and Zepbound partly by needing fewer injections, such as one a month. [12]

Invivyd, a small biotech company, reported on 29 September that its antibody shot VYD2311 caused fewer side effects than Pfizer's Covid vaccine Comirnaty, in a final-stage trial called LIBERTY. [13] The shot is a lab-made antibody, meant to protect against Covid infection the way a vaccine does. [13] Six days after the shot, 57% of people given VYD2311 reported headaches, chills, tiredness or a sore arm, against 91% given Comirnaty. [13] It could also be given safely alongside Comirnaty. [13]

Why it matters. Invivyd wants to offer an alternative to vaccines, and LIBERTY compared the two head to head. [13] The side-effect result does not show how well the antibody prevents infection.

Pierre Fabre Laboratories, a drugmaker, has asked the FDA a third time to approve Ebvallo, a cell treatment the US regulator has turned down twice. [14] Ebvallo treats a deadly complication of infection with the Epstein-Barr virus. [14] It has been available in Europe since late 2022. [14] The FDA rejected it in 2025 over manufacturing problems, and again in January. [14] The new application uses updated results from a trial with no comparison group, plus records from patients treated in Europe. [14]

Why it matters. Atara Biotherapeutics, which first developed Ebvallo, said after the January refusal that the FDA had gone back on an earlier agreement. [14] So patients in the US are still waiting for a treatment that patients in Europe have had for nearly four years. [14]

The FDA has approved Atebrioz, a drug from Mirum Pharmaceuticals, for FOP, an ultra-rare bone disease, Mirum said in late September. [14] In FOP, bone slowly forms in muscle and other soft tissue. Incyte, another drugmaker, had already finished the final tests when it licensed the drug to Mirum in May for $16 million up front. [14] It is approved for adults and children aged 12 and over. [14]

Why it matters. Atebrioz will compete with FOP medicines from Regeneron and Ipsen. [14] Mirum paid a small sum for a drug whose final tests were done, and had an approval within months. [14]

Several big FDA decisions are due before the end of 2026, BioPharma Dive reports. [34] Roche's breast cancer pill giredestrant faces two, by 30 November and 18 December. [34] After surgery, giredestrant with hormone therapy cut the risk of the cancer returning, or of death, by 30%. [34] Gilead's cell treatment anito-cel, for the cancer multiple myeloma, is due by 23 December. [34] In testing, 96% of patients whose cancer had come back after at least three earlier treatments responded. [34]

Why it matters. Until a new commissioner is confirmed, the FDA is run by its acting head, Kyle Diamantas. [34] Heidi Overton, the nominee, may not get a US Senate vote before the US Senate breaks on 5 October, an analyst at RBC Capital Markets wrote. [34]

A year-long trial looked for how semaglutide, a weekly diabetes injection, protects the kidneys of people with type 2 diabetes and kidney disease. [26] Its 106 patients got a weekly shot of the drug or a dummy, and some had kidney scans and biopsies before and after. [26] The trial's main measures, of oxygen, blood flow and swelling in the kidney, did not change. [26] Other measures suggested the drug eased resistance to blood flow into the kidney and slowed scarring. [26]

Why it matters. Semaglutide was already known to protect the kidneys in diabetes, and this trial, published in Nature Medicine on 1 October, looked for the reason. [26] Because the main tests came back flat, the explanation rests on secondary results from smaller groups. [26]

Researchers at Boston Children's Hospital and the Broad Institute found a new genetic switch that holds back fetal haemoglobin. [27] Haemoglobin is the molecule in red blood cells that carries oxygen. Within the first year of life, the body mostly switches from the fetal kind to the adult kind. [27] Studying the DNA of more than 28,000 people, they found that a gene called BACH2 keeps it switched off. [27] Blocking BACH2 in lab cells raised fetal haemoglobin, the team reported in Nature. [27]

Why it matters. Casgevy, an approved gene-editing treatment for sickle cell disease, turns fetal haemoglobin back on through a different switch. [27] A second, separate switch could lead to a simpler treatment, though for now it is a laboratory finding. [27]

The World Health Organization said last week that it expects reversible contraception for men within five to 10 years, the Guardian reported on 1 October. [21] Most of these methods use hormones to switch off sperm production, which must be kept up every day. [21] A US-funded worldwide trial is testing a hormone gel rubbed into the shoulders daily, and early results are surprisingly effective, said Richard Anderson of Edinburgh University. [21] His group of 40 couples had no pregnancies. [21]

Why it matters. About 30% of couples already rely on a method used by the man, yet men's options have barely changed since the condom. [21] The basic science has been known for decades, the Guardian says; turning it into a product is what has been slow. [21]

The journal Science published a special issue on women's health on 1 October. [20] Its editors, all women, named political attacks on women's health care and cuts to research funding as reasons for it. [20] One review says women have lasting pain without a clear physical cause about twice as often as men, and that female pain is poorly understood. [20] Another argues that the XX and XY chromosomes should be considered in every precision-medicine plan. [20]

Why it matters. Women remain poorly represented in clinical trials, STAT notes. [20] Much of the attention on women's health still centres on having babies. [20]

A five-year, 2.3 million pound UK trial will test whether a 12-week pain programme helps women with endometriosis whose pain surgery and medicine have not controlled. [23] Endometriosis is a disease in which cells like the womb lining grow in other parts of the body and cause long-term pain. [23] The programme, led by the University of Aberdeen and NHS Grampian, combines talking therapy, specialist physiotherapy and medicine. [23] Women will get it with their usual care, or usual care alone, and the results will be compared. [23]

Why it matters. There is no cure for endometriosis, and diagnosis can take years. [23] The trial will also measure whether the programme is worth what it costs. [23]

Nicole Saphier, President Trump's third choice for US surgeon general, passed the US Senate health committee on 30 September in a party-line vote. [18] The surgeon general is the US government's top public health educator. [18] Saphier is a radiologist who has appeared on Fox News. [18] Trump's first two picks, Casey Means and Janette Nesheiwat, could not win enough support. [18]

Why it matters. At her hearing Saphier said she had seen no reputable evidence that vaccines cause autism, and called the measles vaccine the greatest tool against measles. [18] The full US Senate has not set a date for its vote. [18]