The decision, which has been a flashpoint in an ongoing debate about regulatory flexibility, was made because Replimune failed to address the agency's issues with the drug's study results, the FDA said.

Original title: “FDA again spurns Replimune melanoma drug”

New safety information for healthcare professionals Ontozry (cenobamate): new requirements for liver monitoring due to reports of severe liver injuryPRAC agreed on a direct…, Agenda Agenda of the PRAC meeting 7-10 April 2026Draft Reference…, PRAC statistics: April 2026 , PRAC statistics: April 2026

Original title: “Meeting highlights from the Pharmacovigilance Risk Assessment Committee (PRAC) 7-10 April 2026”

GSK discontinued Wellcovorin in 1999, but the FDA in September last year asked the pharma to refile an application, pointing to its potential to treat cerebral folate deficiency with “autistic features.”

Original title: “GSK again pulls application for leucovorin, touted by FDA as potential autism treatment”

Prof Fabian Müller, who led the ... life”. Clinical trials were needed to learn how durable the therapy was and whether it would be effective for other autoimmune diseases, he said. Woman who had pioneering cancer treatment 18 years ago still in remission ... The woman had a rare, life-threatening ... Prof Fabian Müller, who led the team, said the speed and depth of the woman’s response was “remarkable” and the therapy had “significantly improved her quality of life”. Clinical trials were needed to learn how durable the therapy was and whether it would be effective for other autoimmune diseases, he said. Woman who had pioneering cancer treatment 18 years ago still in remission ... The woman had a rare, life-threatening blood disorder, autoimmune haemolytic anaemia (AIHA), whereby rogue immune defences destroy red blood cells. A person receiving CAR T-cell therapy. The treatment has proved gamechanging for certain cancers. Photograph: Lucy North/PA ... Treatment reset wayward immune system of patient with life-threatening conditions, say scientists, in a world first ... A woman who lived with three life-threatening autoimmune diseases for more than a decade has returned to a near-normal life after a cell therapy reset her wayward immune system. The other, known as antiphospholipid syndrome (APS), has an opposing effect and raises the risk of harmful blood clots. All three diseases were due to wayward B-cells which make infection-fighting antibodies. With no options left, the doctors offered a treatment known as CAR (chimeric antigen receptor) T-cell therapy, which has proved gamechanging for certain cancers. “The prolonged response off normal therapy suggests there has been an immune reset,” he said, though how long it would last remained unclear. Parker, who is leading CAR-T trials on lupus and related autoimmune conditions in Manchester, said: “There are many active trials currently recruiting across autoimmune conditions, including lupus, myositis, MS, systemic sclerosis, vasculitis, and some have already reported early results. Case reports don’t prove a treatment works for wider use, hence the need for trials.”

Original title: “Woman with three deadly diseases has ‘remarkable’ recovery after cell therapy | Immunology | The Guardian”

First patient dosed as DOMISOL study grows beyond Australia

Original title: “Kainova expands DT 7012 trial into Europe”

Fourteen months after treatment with engineered immune cells, the recipient has no symptoms and doesn’t need to take medication. Fourteen months after treatment with engineered immune cells, the recipient has no symptoms and doesn’t need to take medication. Nature - Fourteen months after treatment with engineered immune cells, the recipient has no symptoms and doesn’t need to take medication. The flaws in the immune system that make one autoimmune disease possible can also increase the likelihood of a second, says Müller, but the woman was the first person he had seen with three such diseases. That combination “can kill you very rapidly”, says June. “Usually, there’s no cure other than treatment with long-term, high-dose steroids”, which broadly dampen the body’s immune system, boosting the risks of infection. But because steroids and more advanced immunosuppressive medication failed to control her symptoms, says Müller, the woman was driven for 3 hours in an ambulance to his clinic, which has developed a reputation for manufacturing engineered immune cells called chimeric antigen receptor (CAR) T cells for one-off treatments. Fourteen months after treatment with engineered immune cells, the recipient has no symptoms and doesn’t need to take medication. ... Antibodies (three-lobed structures, artist’s impression) that attack a person’s own tissues can lead to autoimmune disease.Credit: Ruslanas Baranauskas/SPL · A woman with an ultra-rare combination of three autoimmune diseases has had no symptoms since receiving a single dose of engineered immune cells, doctors in Germany report today1. The researchers harvested the woman’s T cells and engineered them to attack a protein found only on B cells. Within a month of receiving the CAR-T treatment and two chemotherapy drugs that also kill fast-dividing immune cells, the woman had normal levels of red blood cells.

Original title: “One woman, three autoimmune diseases: CAR-T therapy vanquishes ultra-rare disease trio”

After a Phase 2 flop, Brinsupri exits the race to market for the chronic skin disease hidradenitis suppurativa, but other companies, including Incyte, Novartis and UCB, have recently notched clinical and regulatory victories.

Original title: “Insmed Scraps Skin Plans for Lung Disease Drug as Competitors Make Headway”

Subcutaneous delivery shows IV‑level efficacy in moderate to severe TED

Original title: “Amgen reports positive phase 3 results for subcutaneous TEPEZZA in thyroid eye disease”

Study expands development of the antibody into hepatocellular carcinoma

Original title: “CatalYm begins phase 2b trial of visugromab in second-line liver cancer treatment”

Lunsekimig, a bispecific drug aimed at two popular targets, succeeded in studies in asthma and nasal polyps but missed its mark in an eczema trial.

Original title: “Sanofi immune drug hopeful posts mixed results in mid-stage tests”

An application to the U.S. Food and Drug Administration is expected to be supported by data that showed risto-cel's benefit in 31 patients. As a medical scientist for 18 years, he worked in both academia and industry, where his research focused on the discovery of new vaccines and medicines to treat inflammatory disorders and infectious diseases. Steve is a published author in multiple peer-reviewed scientific journals and a patented inventor. Gene-edited cell therapy U.S. Beam Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to approve ristoglogene autogetemcel, or risto-cel, its single-dose, gene-edited cell therapy candidate for people with sickle cell disease (SCD), as early as year’s end. Disease burden heavy for SCD patients in US, despite promise of gene therapy Base editing is a precise gene-editing technology that can create a change in the DNA’s sequence without cutting through both side rails of the DNA’s twisted ladder, a characteristic structure known as its double helix. Such cuts can damage DNA and create unwanted genetic abnormalities. Interim trial data is described in the study “Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease,” which was published in The New England Journal of Medicine.

Original title: “Beam to seek OK of gene-edited cell therapy risto-cel for sickle cell disease”

Sales of Amgen’s thyroid eye disease drug Tepezza have slowed, dipping 1% to $457 million in the fourth quarter of 2025.

Original title: “Amgen Seeks ‘Return to Growth’ for Tepezza as More Convenient Formulation Aces Phase 3”

The upcoming FDA decision for Replimune’s advanced melanoma drug could be a litmus test for the agency's future regulatory decision-making, analysts say, with implications stretching well beyond one company.

Original title: “Biotech Looks to Replimune RP1 Decision as ‘First Indicator’ of FDA Direction”

Shares of Immunovant, Alto and Oric Pharmaceuticals fell this week on clinical trial updates. Elsewhere, Orca Bio’s cell therapy hit a delay and Korsana entered a reverse merger.

Original title: “Immunovant eye drug flunks Phase 3 studies; Beam sickle cell data published in NEJM”

After Eli Lilly achieved the milestone approval of the weight loss pill Foundayo, Novo Nordisk launched a full-court press to defend oral Wegovy, which has been enjoying a record-breaking launch since January.

Original title: “Approvals in Hand, Lilly and Novo Obesity Pills Will Battle It Out on Hairline Differences”

Foundayo could erase the Wegovy pill’s four-month head start as the two drugmakers clash on a debate of efficacy and convenience.

Original title: “FDA approves Lilly obesity pill, triggering battle with Novo Nordisk”

The FDA approved Eli Lilly’s orforglipron—to be known as Foundayo—on Wednesday, officially igniting what analysts believe will be a fierce rivalry with Novo Nordisk’s oral Wegovy.

Original title: “Eli Lilly's Oral Obesity Pill Approved, Kicking Off Renewed Novo Rivalry”

The U.S. Food and Drug Administration today approved Foundayo (orforglipron) marking the fifth approval under the Commissioner's National Priority Voucher (CNPV) pilot program.

Original title: “FDA Approves First New Molecular Entity Under National Priority Voucher Program ”

Updated dosing offers meaningful clinical benefit

Original title: “FDA approves new high dose Spinraza regimen for SMA”

Company says treatment shows strong efficacy and convenient dosing profile

Original title: “Viridian reports positive results from elegrobart phase 3 trial”