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Biotechnology
Last week, a US Food and Drug Administration (FDA) advisory committee (AdCom) voted against approval of Danish drugmaker Lundbeck and Japan-based Otsuka’s Rexulti for post-traumatic stress disorder. France’s Sanofi announced it is expanding its respiratory vaccines pipeline with an up to $1.6 billion acquisition of UK biotech Vicebio. French biotech Abivax released new Phase III data on its investigational ulcerative colitis candidate obefazimod that saw its shares nearly quadruple. Also of note was the latest setback for Sarepta’s Duchenne muscular dystrophy drug Elevidys, when the European Medicines human health committee, the CHMP, declined partner Roche’s accelerated approval of the drug. 27 July 2025
Pharmaceutical
The US Food and Drug Administration (FDA) has notified Bayer that it has extended the review period for the New Drug Application (NDA) for elinzanetant, the first neurokinin 1 and neurokinin 3 receptor antagonist for the treatment of moderate to severe vasomotor symptoms (VMS; also known as hot flashes) due to menopause. 26 July 2025
Biotechnology
As if there had not been enough negative news regarding Elevidys (delandistrogene moxeparvovec) this month, there was more today from the European regulator. 25 July 2025
Pharmaceutical
The European Medicines’ (EMA) human medicines committee (CHMP) has recommended seven novel medicines for approval at its July 2025 meeting. 25 July 2025
Biotechnology
French drugmaker Ipsen announced that the European Commission (EC) has approved Cabometyx (cabozantinib) for adult patients with unresectable or metastatic, well differentiated pancreatic (pNET) and extra-pancreatic (epNET) neuroendocrine tumors who have progressed following at least one prior systemic therapy other than somatostatin analogues. 25 July 2025
Pharmaceutical
The Trump administration has asked the US Supreme Court to allow the National Institutes of Health (NIH) to resume canceling hundreds of research grants supporting work on health equity, racial disparities, and other diversity-related public health initiatives. 25 July 2025
Biotechnology
In an import contrast to events in the USA, UK pharma major GSK late Thursday announced the approval of Blenrep (belantamab mafodotin) by the European Commission. 25 July 2025
Biotechnology
Following the positive opinion by the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) on June 19, 2025, the European Commission has approved French pharma major Sanofi's (isatuximab) in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for the induction treatment of adult patients with newly diagnosed multiple myeloma (NDMM) who are eligible for autologous stem cell transplant. 25 July 2025
Generics
Hospitals are rationing cancer treatments. Pharmacies are scrambling to fill routine antibiotic prescriptions. And across the USA, patients are facing growing delays, substitutions, or outright denials of essential medicines, not because the drugs don’t exist, but because the supply chains behind them have buckled. 24 July 2025
Biotechnology
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has today approved US pharma major AbbVie’s Elahere (mirvetuximab soravtansine) to treat adults with ovarian cancer, fallopian tube cancer, and primary peritoneal cancer. 24 July 2025
Pharmaceutical
Privately-held Danish dermatology specialist LEO Pharma today revealed that the US Food and Drug Administration (FDA) has approved Anzupgo (delgocitinib) cream (20 mg/g) for the topical treatment of moderate-to-severe chronic hand eczema (CHE) in adults who have had an inadequate response to, or for whom topical corticosteroids are not advisable. 24 July 2025
Biotechnology
Swiss pharma giant Roche has paused shipments of the gene therapy Elevidys (delandistrogene moxeparvovec) for new orders in certain countries outside the USA. 24 July 2025
Biotechnology
US biotech Avidity Biosciences’ stock rose 4% on Wednesday after the company announced that the US Food and Drug Administration (FDA) had granted Breakthrough Therapy designation to delpacibart zotadirsen (del-zota, formerly known as AOC 1044), the company’s investigational therapy for the treatment of people with Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44). 24 July 2025
Biotechnology
The US Food and Drug Administration (FDA) has extended the review period for the Biologics License Application (BLA) for GSK’s Blenrep (belantamab mafodotin-blmf) combinations for the treatment of patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy. 24 July 2025
Biotechnology
US healthcare giant Johnson & Johnson (NYSE: JNJ) has announced that the European Commission (EC) has approved a new indication and an indication extension for Darzalex (daratumumab) and Imbruvica (ibrutinib), respectively. 23 July 2025
Pharmaceutical
Privately-held Spanish CNS specialist Minoryx Therapeutics and Germany’s Neuraxpharm Group today announced that the marketing authorization application (MAA) for Minoryx’ lead candidate Nezglyal (leriglitazone) has been submitted to the European Medicines Agency (EMA) for the treatment of pediatric and adult male patients with cerebral adrenoleukodystrophy (cALD). 23 July 2025
Biotechnology
The European Commission has approved Swiss pharma giant Roche’s Itovebi (inavolisib), in combination with palbociclib (Ibrance) and fulvestrant, for the treatment of adult patients with PIK3CA-mutated, oestrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer, following recurrence on or within 12 months of completing adjuvant endocrine treatment. 23 July 2025
Biotechnology
Patients in the UK will now be able to access advanced, personalized treatments prepared at the hospital or even in mobile units near their homes, thanks to a world-first regulatory shift. New legislation introduced by the country’s drug regulator allows hospitals and care centers to produce small or individual batches of cutting-edge therapies at the point of care. 23 July 2025
Biotechnology
French biotech ImCheck Therapeutics has announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its lead program, ICT01, a humanized anti-butyrophilin 3A (BTN3A) monoclonal antibody designed to selectively activate γ9δ2 T cells, in acute myeloid leukemia (AML). 22 July 2025
Biotechnology
Shares in Replimune Group, a US biotech developing novel oncolytic immunotherapies, went in freefall Tuesday, as the company announced a major setback. 22 July 2025
Biotechnology
The US Food and Drug Administration has approved US biotech Incyte’s Monjuvi (tafasitamab-cxix) with lenalidomide and rituximab for adults with relapsed or refractory follicular lymphoma (FL). 19 June 2025
Biotechnology
The biopharmaceutical industry is grappling with a complex mid-year outlook in 2025, as accelerating innovation collides with mounting policy and market uncertainty. From US drug pricing reforms to global tariff tensions, sentiment has taken a hit - reflected in a 16% drop in industry optimism. 19 June 2025
Biotechnology
EY today released its 35th annual Biotech Beyond Borders Report. This finds that the biopharma industry is currently at an inflection point – grappling with a unique environment of macroeconomic uncertainty and constrained access to capital, including high interest rates, inflation, shifting regulatory policies and tariff turmoil. 18 June 2025
Pharmaceutical
The US Food and Drug Administration has announced the Commissioner’s National Priority Voucher (CNPV) program aimed at enhancing the health interests of Americans. 18 June 2025
Biotechnology
US biotech Dyne Therapeutics has revised its regulatory approach for DYNE-101, a treatment in development for myotonic dystrophy type 1 (DM1), after discussions with the American medicines regulator. 18 June 2025
Pharmaceutical
A controversial proposal to change the Inflation Reduction Act’s pricing framework for rare disease drugs has been dropped from the Senate’s reconciliation package, marking a win for patient advocacy groups that lobbied against it. The ORPHAN Cures Act, which sought to shield some rare disease drugs from Medicare price talks, was notably absent from the text released this week by the Senate Finance Committee. 18 June 2025
Generics
Medicines for Europe, a manufacturers of generic and biosimilar trade group, today presented five trade policy recommendations to strengthen global access to medicines and enhance the competitiveness of the off-patent pharmaceutical sector in a seminar with the European Commission and Member States representatives. 17 June 2025
Biotechnology
Australian biotech CSL has announced that the US Food and Drug Administration (FDA) has approved Andembry (garadacimab-gxii), the only treatment targeting factor XIIa for prophylactic use to prevent attacks of hereditary angioedema (HAE) in patients aged 12 years and older. 17 June 2025
Pharmaceutical
French drug developer Averoa today announced that the European Commission (EC) has granted marketing authorization for Xoanacyl (ferric citrate coordination complex). 16 June 2025
Pharmaceutical
China’s National Medical Products Administration (NMPA) held a meeting last week to conduct the 2024 annual review of the quality management system (QMS) in vaccine regulation and pharmaceutical manufacturing inspection. 16 June 2025
Biotechnology
Notable research news last week included US biopharma Avidity Biosciences announcing a positive update on its muscular dystrophy candidate delpacibart braxlosiran (del-brax), and Dutch firm NewAmsterdam released new Phase III on its investigational Alzheimer’s drug obicetrapib. US Health Secretary Robert F Kennedy Jr announced he has dismissed an independent panel of vaccine experts in order to restore trust in vaccines. Also of note, Merck & Co gained US food and Drug Administration (FDA) for its Enflonsia for the prevention of respiratory syncytial virus (RSV). 15 June 2025
Pharmaceutical
The US Food and Drug Administration (FDA) has notified KalVista Pharmaceutical that it will not meet the Prescription Drug User Fee Act (PDUFA) goal date for the New Drug Application (NDA) for sebetralstat, the company’s investigational oral on-demand treatment for hereditary angioedema (HAE). 14 June 2025
Biotechnology
The Food and Drug Administration approved US pharma giant Merck & Co’s mega-blockbuster cancer drug Keytruda (pembrolizumab) for a new indication. 13 June 2025
Biotechnology
US mRNA specialist Moderna today announced that the US Food and Drug Administration (FDA) has approved mRESVIA (mRNA-1345), the company's respiratory syncytial virus (RSV) vaccine, for the prevention of lower respiratory tract disease (LRTD) caused by RSV in individuals 18-59 years of age who are at increased risk for disease. 13 June 2025
Pharmaceutical
The American medicines regulator has approved Zusduri (mitomycin), a hydrogel-based treatment developed by US biotech UroGen Pharma, for adults with recurrent low-grade, intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). 13 June 2025
Pharmaceutical
South Korean drugmaker JW Pharmaceutical has received regulatory approval in China for an injectable antibiotic made with its own synthesized active ingredient, marking a strategic expansion into the Asian market. 12 June 2025
Biotechnology
The US Food and Drug Administration (FDA) yesterday approved Nuvation Bio’s Ibtrozi (taletrectinib), a kinase inhibitor, for adults with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC). 12 June 2025
Pharmaceutical
US biotech Novavax has reported that its experimental combination COVID-19 and influenza vaccine generated immune responses on par with approved shots in a descriptive Phase III trial involving older adults. The company said the results would help shape a future registrational study. 12 June 2025
Pharmaceutical
The UK’s medicines regulator has released a suite of new guidelines to steer companies through its upcoming decentralized manufacturing framework, set to come into effect on July 23, 2025. 11 June 2025
Pharmaceutical
Gilead Sciences has paused five clinical trials of its investigational HIV drugs GS-1720 and GS-4182 after the American medicines regulator raised safety concerns. The once-weekly oral regimen was being studied as an alternative to the company’s blockbuster daily pill, Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide). 11 June 2025
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