FDA Regulatory News
Refine Search
Pharmaceutical
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
Pharmaceutical
The US Food and Drug Administration on Friday granted approval to Oriahnn (an estrogen and progestin combination product consisting of elagolix, estradiol and norethindrone acetate) capsules, co-packaged for oral use, for the management of heavy menstrual bleeding associated with uterine leiomyomas (fibroids) in premenopausal women. 30 May 2020
Biotechnology
Israeli company Protalix BioTherapeutics and family-owned Italian group Chiesi have submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for pegunigalsidase alfa to treat Fabry disease. 28 May 2020
Biotechnology
Azitra, a privately-held dermatology and microbiome specialist, has been awarded Rare Pediatric Disease designation for its candidate ATR-12, for the treatment of Netherton syndrome (NS). 28 May 2020
Biotechnology
The US Food and Drug Administration has approved VESIcare LS (solifenacin succinate) oral suspension for the treatment of neurogenic detrusor overactivity (NDO) in pediatric patients aged two years and older. 28 May 2020
Biotechnology
Belgo-Dutch clinical-stage biotech argenx saw its shares close up nearly 30% at $190.00 on Tuesday, after it released top-line trial results for its myasthenia gravis drug candidate. 27 May 2020
Biotechnology
The US Food and Drug Administration has approved a label extension for Dupixent (dupilumab), to include treatment of children aged six to 11 with moderate-to-severe atopic dermatitis (AD). 27 May 2020
Generics
Shares of London-listed generics drugmaker Hikma Pharmaceuticals rose 1.5% to 2,553 pence on Friday, after the Jordanian company announced that its wholly-owned US subsidiary has received approval from the US Food and Drug Administration for its icosapent ethyl capsules, 1gm, the generic equivalent to Vascepa, a fish-oil-based drug from US firm Amarin. 25 May 2020
Pharmaceutical
UK-based AstraZeneca last week announced an up to $1 billion award from the USA’s BARDA to support the development and production of the investigational COVID-19 vaccine candidate AZD 1222, formerly dubbed ChAdOx1 nCoV-19. Also on the novel coronavirus scene, US biotech Sorrento Therapeutics presented strong findings from a trial of its STI-1499, part of the firm’s COVID-19 program, and US biotech Moderna saw its shares rocket after revealing positive results from a clinical trial of its COVID-19 candidate mRNA-1273. Meantime, HIV specialist ViiV Healthcare presented positive interim analysis of the HIV Prevention Trials Network 083 study. Regulatory news included AstraZeneca and partner Merck & Co gaining another US indication approved for its already blockbuster drug Lynparza, now in mCRPC, a type of prostate cancer. 24 May 2020
Biotechnology
Accelerated FDA approval for Alunbrig in first-line setting for rare and serious form of lung cancer
The US Food and Drug Administration has approved Alunbrig (brigatinib) for adult patients with anaplastic lymphoma kinase-positive (ALK+) metastatic non-small cell lung cancer (NSCLC) as detected by an FDA-approved test. 23 May 2020
Pharmaceutical
In a U-turn by the US regulator, the Food and Drug Administration on Thursday approved Kynmobi (apomorphine HCI) sublingual film (APL-130277) for the acute, intermittent treatment of OFF episodes in patients with Parkinson’s disease (PD). 22 May 2020
Pharmaceutical
The US Food and Drug Administration has approved Ferriprox (deferiprone) twice-a-day tablets for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate, to be marketed by the US subsidiary of family-controlled Italian drugmaker Chiesi Farmaceutici. 22 May 2020
Pharmaceutical
The US Food and Drug Administration has accepted the New Drug Application (NDA) for accelerated review of Zokinvy (lonafarnib) in the treatment of Progeria and Progeroid Laminopathies. 20 May 2020
Pharmaceutical
Leading PARP blocker Lynparza (olaparib) has been approved in the USA for people with HRR gene-mutated metastatic castration-resistant prostate cancer (mCRPC). 20 May 2020
Biotechnology
Roche’s immuno-oncology drug Tecentriq (atezolizumab) continues its charge in lung cancer with another approval in the USA. 19 May 2020
Biotechnology
Mezzion Pharma after-hours on Monday announced that analysis of data from its Phase III FUEL trial showed statistically-significant positive results for an important efficacy endpoint, the myocardial performance index, with the news sending the South Korean biotech firm’s shares up 2.4% to 180,300 won by close of trading today. 19 May 2020
Biotechnology
Daiichi Sankyo and partner AstraZeneca today announced that their Enhertu (fam-trastuzumab deruxtecan-nxki) has been granted Breakthrough Therapy designation (BTD) in the USA for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a HER2 mutation and with disease progression on or after platinum-based therapy. 18 May 2020
Biotechnology
The US Food and Drug Administration approved Qinlock (ripretinib) tablets as the first new drug specifically approved as a fourth-line treatment for advanced gastrointestinal stromal tumor (GIST), a type of tumor that originates in the gastrointestinal tract. 16 May 2020
Biotechnology
On Friday, the US Food and Drug Administration granted accelerated approval to Rubraca (rucaparib) for patients with deleterious BRCA mutation (germline and/or somatic)-associated metastatic castration-resistant prostate cancer (mCRPC) who have been treated with androgen receptor-directed therapy and a taxane-based chemotherapy. 16 May 2020
Pharmaceutical
The US Food and Drug Administration on Friday expanded the indication of already-marketed Pomalyst (pomalidomide) to include treating adult patients with AIDS-related Kaposi sarcoma after failure of highly active antiretroviral therapy and Kaposi sarcoma in adult patients who are HIV-negative. 16 May 2020
Biotechnology
The US Food and Drug Administration has issued a complete response letter (CRL) for the new drug application of avapritinib for the treatment of adults with unresectable or metastatic fourth-line gastrointestinal stromal tumor (GIST). 15 May 2020
Sign up to receive email updates
Join industry leaders for a daily roundup of biotech & pharma news
Today's issue
Company Spotlight
A US biotech company dedicated to creating oral drugs with an emphasis on indications in virology and immunology.
Copyright © The Pharma Letter 2024 | Headless Content Management with Blaze