FDA Regulatory News
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Biotechnology
UK pharma major GSK has gained US regulatory approval for a new prefilled syringe presentation of its shingles vaccine, Shingrix (zoster vaccine), a move aimed at streamlining administration and boosting immunization coverage. 17 July 2025
Biotechnology
UK pharma major GSK is seeking US approval for Blenrep (belantamab mafodotin) in two multiple myeloma indications. 16 July 2025
Biotechnology
The Division of Hepatology and Nutrition of the US Food and Drug Administration (FDA) has lifted the partial clinical hold on the completed PORTOLA Phase IIa clinical trial evaluating zetomipzomib, a first-in-class selective immunoproteasome inhibitor, in patients with autoimmune hepatitis (AIH), California, USA-based drug developer Kezar Life Sciences announced yesterday. 16 July 2025
Biosimilars
The US Food and Drug Administration approved, the first rapid-acting interchangeable biosimilar product, Kirsty (insulin aspart-xjhz, as a 3 mL single-patient-use prefilled pen and 10 mL multiple-dose vial. Kirsty is biosimilar to and interchangeable with Novolog (insulin aspart), from Denmark’s Novo Nordisk. 16 July 2025
Pharmaceutical
Germany’s Bayer has announced that the US Food and Drug Administration (FDA) has approved Kerendia (finerenone) to treat patients with heart failure (HF) with left ventricular ejection fraction (LVEF) ≥40%, following the agency’s Priority Review of its supplemental New Drug Application (sNDA). 14 July 2025
Generics
Even as the US Food and Drug Administration (FDA) has unveiled a strategic expansion of its oversight mechanisms for foreign manufacturing facilities supplying the American market, aiming to bolster product safety and ensure consistent quality for US consumers, the intensified oversight presents significant long-term benefits for the Indian pharmaceutical sector as a whole, reports The Pharma Letter’s India correspondent. 14 July 2025
Pharmaceutical
The US Food and Drug Administration (FDA) last week authorized KalVista Pharmaceutical’s Ekterly for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. US President Donald Trump put out another warning on imposing tariffs on pharmaceutical products, albeit they may get a grace period of 10 months. US biopharma Rhythm Pharmaceuticals released encouraging Phase II results for its bivamelagon in acquired hypothalamic obesity. Also of note, AbbVie entered an up to $1.9 billion licensing deal with Ichnos Glenmark on autoimmune diseases. 13 July 2025
Biotechnology
US biotech Capricor Therapeutics today revealed receipt of a Complete Response Letter (CRL) from the US Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for deramiocel, the company’s lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). 11 July 2025
Pharmaceutical
Artificial Intelligence (AI) is reshaping pharmacovigilance, promising faster detection of safety signals hidden within mountains of adverse event (AE) data. However, as the US Food and Drug Administration (FDA) sharpens its focus on transparency, credibility, and real-world monitoring, the days of relying on opaque algorithms are numbered. In this article, we explore why sponsors and safety teams must rethink their approach to AE prediction to ensure the very tools designed to protect patients don’t become risks themselves. 11 July 2025
Biotechnology
The US Food and Drug Administration (FDA) has approved US mRNA specialist Moderna supplemental Biologics License Application (sBLA) for Spikevax, the company's COVID-19 vaccine, in children six months through 11 years of age who are at increased risk for COVID-19 disease. 11 July 2025
Pharmaceutical
The US Food and Drug Administration (FDA) has taken the radical move of publishing more than 200 decision letters, known as complete response letters (CRLs). 10 July 2025
Pharmaceutical
The US Food and Drug Administration (FDA) has accepted for review Merck & Co’s New Drug Application (NDA) for doravirine/islatravir (DOR/ISL), an investigational, once-daily, oral, two-drug regimen for adults with HIV-1 infection that is virologically suppressed on antiretroviral therapy. 10 July 2025
Pharmaceutical
The US Food and Drug Administration (FDA) has accepted the supplemental new drug application (sNDA) for Inqovi (decitabine and cedazuridine) plus venetoclax as a treatment for adults with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive induction chemotherapy. 10 July 2025
Biotechnology
The US Food and Drug Administration (FDA) has approved a label update with a new recommended titration dosing schedule for Kisunla (donanemab-azbt), Eli Lilly's once-monthly amyloid-targeting therapy for early symptomatic Alzheimer's disease (AD), including patients with mild cognitive impairment (MCI) and the mild dementia stage of AD, with confirmed amyloid pathology. 9 July 2025
Pharmaceutical
The US Food and Drug Administration (FDA) and the National Institutes of Health (NIH) have hosted a hybrid workshop on reducing animal testing. 9 July 2025
Pharmaceutical
USA and UK-based KalVista Pharmaceuticals today announced that the US Food and Drug Administration (FDA) has approved Ekterly (sebetralstat), a novel plasma kallikrein inhibitor, for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. 7 July 2025
Biotechnology
US pharma major AbbVie announced it plan to acquire Capstan Therapeutics, along with its autoimmune disease candidate CPTX2309, for up to $2.1 billion. Organon posted disappointing mid-stage trial results for its investigational endometriosis pain drug, OG-6219. Also of note last week, the US Food and drug Administration (FDA) granted accelerated approval for Regeneron’s multiple myeloma drug Lynozyfic. 6 July 2025
Biotechnology
The US Food and Drug Administration has granted accelerated approval to sunvozertinib (trade name Zegfrovy) from Dizal (Jiangsu) Pharmaceutical for adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC). 3 July 2025
Biotechnology
The US Food and Drug Administration (FDA) has provided an update on the risk of myocarditis after receiving an mRNA COVID-19 vaccine, noting the risk is higher in young men. 3 July 2025
Biotechnology
The US Food and Drug Administration (FDA) has granted accelerated approval for Regeneron Pharmaceuticals’ Lynozyfic (linvoseltamab-gcpt) to treat adult patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti CD38 monoclonal antibody. 3 July 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
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
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
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
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
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
Biotechnology
Shares of Avidity Biosciences were up almost 5% at $38.00 pre-market today, as it announced the accelerated approval regulatory pathway in the USA is open for delpacibart braxlosiran (del-brax) in the treatment of facioscapulohumeral muscular dystrophy (FSHD). 9 June 2025
Pharmaceutical
George Medicines, a late-stage UK-based biopharmaceutical company focused on addressing unmet needs in cardiometabolic disease, revealed a regulatory approval that it says could reshape hypertension treatment in the USA. 9 June 2025
Biotechnology
Regulatory news last week included US biotech Moderna receiving Food and Drug Administration (FDA) approval for its new COVID-19 Vaccine mNEXSPIKE. On the M&A front, French pharma major Sanofi announced it is punting more than $9 billion to acquire Blueprint Medicines and its Ayvakit, BLU-808 and immunology pipeline. USA-based Cullinan Therapeutics has entered into a license deal with China's Genrix Bio for the latter’s velinotamig for multiple myeloma. Also of note, RegeNXBio released what lookedlike good results for its investigational Duchenne muscular dystrophy drug RGX-202. 8 June 2025
Biotechnology
German genetic medicine company ViGeneron has announced a rebrand to become VeonGen Therapeutics. 5 June 2025
Biotechnology
US rare disease meds developer Sarepta Therapeutics revealed that its rAAVrh74 viral vector used in the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy type 2E/R4, has been granted platform technology designation by the US Food and Drug Administration. 5 June 2025
Pharmaceutical
The American medicines regulator has committed to reviewing the safety of the abortion pill mifepristone, amid renewed political scrutiny and pressure from conservative lawmakers. 4 June 2025
Pharmaceutical
The American medicines regulator has approved Bayer’s Nubeqa (darolutamide) for broader use in patients with metastatic castration-sensitive prostate cancer (mCSPC), marking a step forward for the drugmakeFood and Drug Administraton,r’s prostate cancer franchise. 4 June 2025
Biotechnology
Research news last week included Rocket Pharmaceuticals suffering a setback with its Phase II trial of RP-A501, which resulted in a patient death and a US Food and Drug Administration (FDA) hold on the study. Germany’s InflaRx announced it would discontinue development of vilobelimab in a dermatology condition due to futility. Regeneron and Sanofi presented mixed results for itepekimab, leading them to consider the future of the chronic obstructive pulmonary (COPD) therapy. Also of note, Japan’s Otsuka Pharma revealed that the US FDA has accepted its biologics license application (BLA) for sibeprenlimab for review as an immunoglobulin A nephropathy IgAN) treatment. 1 June 2025
Biotechnology
The US Food and Drug Administration (FDA) has approved mNEXSPIKE (mRNA-1283), a new vaccine against COVID-19, US mRNA specialist Moderna announced on Saturday, amidst emerging coronavirus variants. 31 May 2025
Pharmaceutical
Swiss eye care specialist Alcon yesterday revealed the US Food and Drug Administration (FDA) has approved Tryptyr (acoltremon ophthalmic solution) 0.003%, formerly known as AR-15512.The new pushed the firm’s shares up 2.5% to 72.32 francs this morning. 30 May 2025
Pharmaceutical
The US Food and Drug Administration (FDA) has identified a potential path forward for elamipretide for the treatment of Barth syndrome, Stealth BioTherapeutics revealed today. 29 May 2025
Biotechnology
Texas, USA-based clinical-stage biopharma Savara on Tuesday announced that the company received a refuse to file (RTF) letter from the US Food and Drug Administration (FDA) for the biologics license application (BLA) of Molbreevi (molgramostim) as a therapy to treat patients with autoimmune pulmonary alveolar proteinosis (PAP), with the news sending its shares plunging 32% to $1.94. 28 May 2025
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