ALETH / LIFESCI-BRIEF / 2026-04-24 / FDA APPROVES FIRST DEAFNESS GENE THERAPYThe Aleth Briefs link to the sources behind the stories and show how the week unfolded.
The week in five lines:
Regeneron’s Otarmeni became the first FDA-approved gene therapy for deafness.
Lilly will pay up to $7bn for Kelonia’s in-body CAR-T for multiple myeloma.
UK Biobank participant data was advertised for sale on Alibaba platforms in China.
Trump ordered faster FDA reviews for qualifying psychedelic drugs.
Roche’s fenebrutinib more than halved MS relapses, but deaths were imbalanced.
Browse by day:
Weekend catch-up: UCB-Neurona · psychedelics order · 10x Atera
Monday: Lilly-Kelonia · AZ COPD · Novo sickle cell · Arcus
Tuesday: Idvynso · Welireg · Ultomiris · Enspryng · fenebrutinib · Danaher
Wednesday: Amneal-Kashiv
Thursday: Otarmeni · Biobank · RAPID · AbbVie · Sanofi · bioMérieux
Weekend catch-up
UCB agreed to buy Neurona Therapeutics for up to $1.15bn, $650m of it upfront.
Neurona’s NRTX-1001 is a single-dose neural cell therapy, injected into the brain, in phase 1/2 trials for drug-resistant mesial temporal lobe epilepsy. UCB announced the deal on Friday evening and calls it a move into regenerative medicine and advanced therapies. It expects to close by the end of Q2 2026.
UCB to acquire Neurona Therapeutics advancing its leadership in epilepsy · UCB
US President ordered the FDA to speed up psychedelic drug reviews.
On Saturday, Trump signed an executive order telling the FDA to give Commissioner’s National Priority Vouchers to psychedelic drugs that hold Breakthrough Therapy designation and meet the program’s criteria. The FDA and the Drug Enforcement Administration must also open a route to psychedelics, including ibogaine, under the Right to Try Act. ARPA-H will put at least $50m into state programs.
Accelerating medical treatments for serious mental illness · The White House
10x Genomics launched Atera, an in situ spatial platform for whole-transcriptome analysis at single-cell sensitivity.
10x unveiled it over the weekend, ahead of AACR. Pre-orders are open, and shipping should begin in H2 2026.
10x Genomics introduces Atera · 10x Genomics
Monday 20 April
Eli Lilly agreed to buy Kelonia Therapeutics for up to $7bn.
$3.25bn is upfront, the rest tied to milestones. Kelonia’s lead drug, KLN-1010, is a one-time infusion that makes BCMA-targeted CAR-T cells inside the patient’s body. It is in phase 1 in relapsed or refractory multiple myeloma, and it does away with the patient-specific manufacturing conventional CAR-T needs. The deal should close in H2 2026.
Lilly to acquire Kelonia Therapeutics to advance in vivo CAR-T cell therapies · Lilly
AstraZeneca’s anti-IL-33 antibody tozorakimab cut moderate-to-severe COPD flare-ups in a third positive phase 3 trial.
In MIRANDA, dosing every two weeks reduced the annualised exacerbation rate in former smokers, the primary population, and across all patients. AstraZeneca will submit the data to regulators.
Tozorakimab met primary endpoint in phase III MIRANDA trial · AstraZeneca
Novo Nordisk’s oral etavopivat met both primary endpoints in the phase 3 HIBISCUS trial in sickle cell disease.
The once-daily pill cut the annualised rate of vaso-occlusive crises by 27% against placebo. At week 24, 48.7% of patients achieved a haemoglobin increase of >1 g/dL, against 7.2% on placebo. Novo plans its first filing in H2 2026.
Etavopivat meets co-primary endpoints in phase 3 HIBISCUS trial · Novo Nordisk
Arcus Biosciences stopped its phase 3 STAR-121 trial of the anti-TIGIT antibody domvanalimab for futility.
STAR-121 tested domvanalimab with zimberelimab and chemotherapy against Keytruda and chemotherapy in first-line metastatic non-small cell lung cancer. Gilead declined to extend its options on Arcus’s early pipeline, so its option period ends in July. It keeps existing time-limited options on programs including AB801 and AB598.
Arcus Biosciences Form 8-K, 20 April 2026 · SEC
Tuesday 21 April
The FDA approved Merck’s Idvynso (doravirine/islatravir), a once-daily two-drug pill for adults with HIV whose virus is already suppressed.
It replaces a stable regimen in patients with no history of virological treatment failure and no known resistance to doravirine. Merck says it is the first complete two-drug regimen without an integrase inhibitor or tenofovir to match a three-drug regimen, Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide), head to head in phase 3.
FDA approves Merck’s once-daily Idvynso (doravirine/islatravir) · Business Wire
FDA approval letter, NDA 216964 · FDA
Adding Welireg (belzutifan) to Keytruda and Lenvima failed in first-line advanced kidney cancer in Merck and Eisai’s phase 3 LITESPARK-012 trial.
At an interim analysis, neither the triplet nor a Keytruda (pembrolizumab)-quavonlimab coformulation given with Lenvima (lenvatinib) beat Keytruda plus Lenvima on progression-free or overall survival.
Merck and Eisai provide update on phase 3 LITESPARK-012 trial · Eisai
AstraZeneca’s Ultomiris (ravulizumab) cut proteinuria at week 34 in an interim analysis of the phase 3 I CAN trial in IgA nephropathy.
It will seek accelerated approval. The kidney-function endpoint reads out at week 106.
Ultomiris proteinuria reduction in IgA nephropathy in I CAN trial · AstraZeneca
Roche’s Enspryng (satralizumab) cut the risk of relapse by 68% against placebo in the phase 3 METEOROID trial in MOGAD.
MOGAD is a rare autoimmune disease that attacks the optic nerves, brain and spinal cord, and no drug is approved for it. At 48 weeks, 87% of patients on Enspryng were relapse-free, against 67% on placebo. Roche presented the data at the American Academy of Neurology (AAN) meeting.
Genentech’s Enspryng reduces risk of relapses by 68% in MOGAD · Genentech
Roche’s fenebrutinib cut relapses by more than half against Aubagio in two phase 3 MS trials.
The BTK inhibitor cut annualised relapse rates by 51.1% and 58.5% against Aubagio (teriflunomide) in FENhance 1 and 2, in relapsing multiple sclerosis. The data, presented at AAN, also showed seven deaths on fenebrutinib against one on Aubagio during the reporting period, from causes including infections, complications of type 1 diabetes, bleeding and suicide. One more death was observed after that period. Roche will file the relapsing and primary progressive data together.
Fenebrutinib reduced relapses versus standard of care in RMS · Genentech
Danaher’s Q1 revenue rose 3.5% YoY to $6.0bn, with core revenue up 0.5%.
Bioprocessing and Life Sciences largely offset a light respiratory season at Cepheid. Danaher nudged its 2026 adjusted EPS guidance up to $8.35-8.55.
Danaher reports first quarter 2026 results · Danaher
Wednesday 22 April
Amneal agreed to buy biosimilars developer Kashiv BioSciences for $750m at closing plus further payments.
It pays $375m in cash and $375m in shares at closing, plus up to $350m in regulatory milestones, potential royalties tied to commercial milestones and funding of operations through closing. Amneal expects to have >12 biosimilars on the market by 2030. The deal needs Amneal shareholder approval and should close in H2 2026.
Amneal agrees to acquire Kashiv BioSciences to create biosimilar leader · Amneal
Thursday 23 April
The FDA approved Regeneron’s Otarmeni, the first gene therapy for a form of inherited deafness.
Otarmeni (lunsotogene parvec) delivers a working copy of the OTOF gene to the inner ear’s hair cells in patients with severe-to-profound hearing loss caused by two faulty copies. It is the first approved dual-AAV gene therapy. The FDA granted accelerated approval 61 days after filing, using a Commissioner’s National Priority Voucher. Eligible patients must have preserved outer hair cell function and no prior cochlear implant in the ear to be treated.
Under Regeneron’s new White House pricing deal, US patients will get it at no cost.
FDA approves first-ever gene therapy for treatment of genetic hearing loss · FDA
Regeneron deal on most-favored-nation pricing · The White House
UK Biobank participant data was advertised for sale on Alibaba platforms in China.
Minister Ian Murray told the Commons on Thursday that three listings had been found, and that at least one appeared to hold data on all 500,000 volunteers. The data carried no names, addresses or contact details, and the listings have been removed.
UK Biobank revoked access for the research institutions identified as the source and, at the government’s request, has paused further access to its data.
Minister of State statement to the House of Commons: 23 April 2026 · GOV.UK
A message to our participants: UK Biobank data security update · UK Biobank
CMS and the FDA announced RAPID, a planned pathway to speed Medicare coverage of Breakthrough Devices.
CMS will propose a national coverage determination on the day an eligible device wins FDA authorisation, which could bring coverage as soon as two months after authorisation rather than a year or more. CMS will publish a proposed procedural notice for 60 days of comment, and the pathway is expected to take effect when the final notice is published. The existing Transitional Coverage for Emerging Technologies pathway is paused for new candidates.
CMS and FDA announce RAPID coverage pathway · CMS
The FDA rejected AbbVie’s botulinum toxin TrenibotE over manufacturing.
AbbVie said the FDA wants more information on its manufacturing processes but raised no safety or efficacy concerns and asked for no new studies.
AbbVie provides update on TrenibotE biologics license application · AbbVie
Sanofi’s Q1 sales grew 13.6% at constant exchange rates, as Dupixent (dupilumab) sales rose 30.8%.
Belén Garijo takes over as chief executive next month. All five of its regulatory approvals in the quarter were in immunology.
Q1 2026: double-digit sales and business EPS growth · Sanofi
Roche’s Q1 sales rose 6% at constant exchange rates.
Diagnostics sales grew 3% at constant rates and fell 7% in francs, with healthcare pricing reforms in China still a drag. Roche confirmed its full-year outlook.
Roche reports sales growth of +6% at constant exchange rates in Q1 2026 · Roche
bioMérieux cut its 2026 guidance after Q1 organic sales fell 3.9% against a strong respiratory season a year earlier.
It now expects organic sales growth of 3-5%, down from 5-7%, and organic growth in CEBIT, its operating profit measure, of 0-10%, down from at least 10%.
First-quarter 2026 business review · bioMérieux
Aleth · Open Access Research