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The nomination of Robert F Kennedy Jr — known as RFK Jr — for the role of US Secretary of the Department of Health and Human Services (HHS) President-elect Donald Trump sent shares in vaccine companies sharply downwards. 22 November 2024
Biotechnology
British pharma major AstraZeneca is facing challenges in its bid to secure full approval for its anticoagulant reversal therapy, Andexxa (andexanet alfa), in the USA. 22 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has granted accelerated approval of Ziihera (zanidatamab-hrii) 50mg/mL for injection for intravenous use, say the drug’s developer, Jazz Pharmaceuticals. 21 November 2024
Biotechnology
Astellas Pharma has been hit by bad news from the US Food and Drug Administration, which will not currently approve the firm’s request to supplement the label for Izervay (avacincaptad pegol). 21 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has accepted for review the resubmission of the supplemental Biologics License Application (sBLA) for Dupixent(dupilumab), Regeneron and partner Sanofi revealed late Friday. 18 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has approved Syndax Pharmaceuticals’ Revuforj (revumenib). 18 November 2024
Biotechnology
Oxford, UK-based rare respiratory diseases focused AlveoGene today revealed it has been granted a Rare Pediatric Disease designation (RPDD) by the US Food and Drug Administration (FDA) for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B (SP-B) deficiency. 15 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has granted accelerated approval of PTC Therapeutics’ gene therapy for the treatment of AADC (aromatic L-amino acid decarboxylase) deficiency, making it the first-ever gene therapy approved in the USA that is directly administered to the brain. 14 November 2024
Biotechnology
Shares of US genetic meds company MeiraGTx Holdings were up 7% at $7.12 in early trading after it announced important developments along with its third-quarter 2024 financials, reporting a $0.54 per share loss. 13 November 2024
Pharmaceutical
Intercept Pharmaceuticals, now wholly owned subsidiary of Italy’s Alfasigma, today revealed that the US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) that addresses the supplemental New Drug Application (sNDA) for Ocaliva (obeticholic acid, OCA) seeking full approval for the treatment of indicated patients with primary biliary cholangitis (PBC) – a rare, progressive disease that disproportionally affects women. 12 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has removed the clinical hold on the Investigational New Drug (IND) application for its COVID-19-Influenza Combination (CIC) and stand-alone influenza vaccine candidates, US vaccines developer Novavax revealed yesterday. 12 November 2024
Biotechnology
RAPT Therapeutics has announced the termination of its zelnecirnon program following regulatory concerns around safety. 12 November 2024
Biotechnology
On November 8, the US Food and Drug Administration approved Aucatzyl (obecabtagene autoleucel; obe-cel) from US biopharma Autolus Therapeutics' 11 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program, according to Sarah Wicks and James Valentine at US law firm Hyman, Phelps & McNamara. 11 November 2024
Pharmaceutical
Shanghai-based Dizal has made a submission to the US Food and Drug Administration, seeking approval for sunvozertinib. 8 November 2024
Biotechnology
US healthcare giant Johnson & Johnson today revealed it has submitted applications with the US Food and drug Administration (FDA) and European Medicines Agency (EMA) to expand the approved indications for its Darzalex (daratumumab) subcutaneous (SC) formulation for the treatment of high-risk smouldering multiple myeloma (SMM). 8 November 2024
Biotechnology
Asklepios BioPharmaceutical (AskBio) today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). 7 November 2024
Pharmaceutical
USA-based Acadia Pharmaceuticals has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million upon the closing of the transaction. 6 November 2024
article
The US Food and Drug Administration (FDA) has announced approval of a modification to the Opioid Analgesic Risk Evaluation and Mitigation Strategy (OA REMS) 6 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted medicine for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older, under development by Ionis Pharmaceuticals. 4 November 2024
Biotechnology
Shares of Netherlands-based gene therapy company uniQure were up more than 9% at $32.00 in pre-market US trading, as the company revealed new evidence regarding a safety scare related to its hemophilia B gene therapy. 29 March 2021
Biotechnology
The US Food and Drug Administration on Friday approved Abecma (idecabtagene vicleucel; ide-cel), a first-in-class B-cell maturation antigen (BCMA)-directed genetically-modified autologous T-cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory multiple myeloma. 29 March 2021
Biotechnology
Among last week’s notable news, AstraZeneca released long-waited data from the US Phase III trial of its COVID-1 vaccine, AZD1222, which pave the way for US regulatory approval and restore confidence in Europe, but this was not without further controversy. Roche and Regeneron Pharmaceuticals reported strong Phase III data for their REGN-COV2 antibody cocktail for hospitalized COVID-19 patients. Pfizer and Eli Lilly suffered a setback on their osteoarthritis pain drug tanezumab, with a negative Food and Drug Administration advisory committee vote. Dutch rare diseases firm ProQR Therapeutics announced encouraging early-stage results for its QR-421a in Usher syndrome, a rare blindness disorder. 28 March 2021
Biotechnology
UK pharma major GlaxoSmithKline and Vir Biotechnology have submitted an application to the US Food and Drug Administration requesting Emergency Use Authorization (EUA) for VIR-7831 (GSK4182136), an investigational dual-action SARS-CoV-2 monoclonal antibody for the treatment of adults and adolescents with mild-to-moderate COVID-19 who are at risk for progression to hospitalization or death. 27 March 2021
Biotechnology
Perhaps not surprisingly, given negative briefing documents, the US Food and Drug Administration joint Arthritis Advisory Committee and Drug Safety and Risk Management Advisory Committee on tanezumab voted against the benefits of the drug to treat osteoarthritis pain. 26 March 2021
Biotechnology
Positive top-line results from the primary analysis of AstraZeneca’s USA-based Phase III trial confirm the safety and efficacy of its coronavirus vaccine. 25 March 2021
Generics
Fresenius Kabi Oncology Limited (FKOL) has been sentenced to pay $50 million in fines and forfeiture after pleading guilty to concealing and destroying records prior to a 2013 US Food and Drug Administration plant inspection in India. 24 March 2021
Pharmaceutical
Novo Nordisk late Monday announced that the US Food and Drug Administration has issued a Refusal to File letter covering the label expansion application for once-weekly semaglutide 2.0mg for the treatment of type 2 diabetes, which was filed on 20 January 2021. 23 March 2021
Biotechnology
The US Food and Drug Administration has approved a label update for Merck & Co’s blockbuster checkpoint blocker, targeting advanced esophageal or gastroesophageal (GEJ) cancer. 23 March 2021
Pharmaceutical
Among last week’s notable research news, Eli Lilly presented updated results on its Alzheimer’s disease candidate donanemab which, though touted as promising in a disease area that has been filled with failures, is seen as disappointing by some. Meantime, strong Phase III results saw Sanofi and partner Regeneron Pharmaceuticals halt their trial of Libtayo in cervical cancer. On the deal-making front, rivals in the HIV space Gilead Sciences and Merck & Co signed a pact last Monday to trial a combination of their respective candidates – lenacapavir and islatravir. Regulatory news saw AbbVie suffer a setback last Wednesday, when the US Food and Drug Administration extended the review period for its Rinvoq in the treatment of active psoriatic arthritis. 21 March 2021
Pharmaceutical
The US Food and Drug Administration (FDA) has approved Ponvory (ponesimod), a once-daily oral selective sphingosine-1-phosphate receptor 1 (S1P1) modulator, to treat adults with relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease. 19 March 2021
Biotechnology
The US Food and Drug Administration has approved Arcalyst (rilonacept) injection to treat recurrent pericarditis and reduce the risk of recurrence in adults and children 12 years and older. 19 March 2021
Biotechnology
The US Food and Drug Administration has extended the review period for the supplemental New Drug Application (sNDA) for Rinvoq (upadacitinib) in the treatment of adult patients with active psoriatic arthritis. 17 March 2021
Biotechnology
The regenerative medicine and advanced therapies sector raised a record $19.9 billion in funding in 2020, fueling the broader biotech sector and driving a rapidly advancing pipeline of potentially transformative therapies, according to the Alliance for Regenerative Medicine’s (ARM) 2020 Annual Report released today. 16 March 2021
Pharmaceutical
Stockholm’s Calliditas Therapeutics has completed its US regulatory submission for Nefecon (budesonide), an oral treatment for primary IgA Nephropathy (IgAN). 15 March 2021
Biotechnology
Last week, news related to COVID-19 included US pharma giant Merck & Co and biotech partner Ridgeback Biotherapeutics releasing encouraging mid-stage data on molnupiravir, their antiviral treatment. Producing negative news, CytoDyn reported Phase III data on its COVID-10 candidate Vyrologix (lerontimab) in COVID-19. Elsewhere, US biotech bluebird bio released new analyses relating to LentiGlobin in cell therapy programs that the US Food and Drug Administration put on clinical hold due to a possible link with a case of acute myeloid leukemia (AML). Also of note, after a long wait, AVEO Oncology last Wednesday finally gained FDA approval for its bladder cancer drug Fotivda (tivozanib). 14 March 2021
Biotechnology
After several US setbacks affecting the firm’s lead product, AVEO Oncology late Wednesday finally received US Food and Drug Administration approval for Fotivda (tivozanib) as a treatment of adults with relapsed or refractory advanced renal cell carcinoma who have received two or more prior systemic therapies. 11 March 2021
Biotechnology
Shares of US biotech bluebird bio gained 13% pre-market today and were still up 8.8% at $32.70 by mid-morning after it announced encouraging analyses of its gene therapy program that was linked with a serious adverse event. 10 March 2021
Pharmaceutical
The US Government Accountability Office (GAO) has called on the US Food and Drug Administration (FDA) to address a backlog in inspections that has been worsened by the pandemic. 10 March 2021
Biotechnology
Shares of US gene therapy company Rocket Pharmaceuticals were up more than 9% at $53.14 in early afternoon trading on Tuesday, after the company revealed that the Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L201, its investigational gene therapy for the treatment of leukocyte adhesion deficiency-I (LAD-I). 10 March 2021
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