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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
The approval of a Food and Drug Administration (FDA) emergency use authorization (EUA) for AstraZeneca’s COVID-19 vaccine AZD1222, now dubbed Vaxzevria, will boost confidence and use of the vaccine globally. 16 April 2021
Biotechnology
Following swiftly on approval for metastatic triple-negative breast cancer (mTNBC), the US Food and Drug Administration has also granted accelerated approval of Trodelvy (sacituzumab govitecan-hziy) for use in adult patients with locally advanced or metastatic urothelial cancer (UC) who have previously received a platinum-containing chemotherapy and either a programmed death receptor-1 (PD-1) or a programmed death-ligand 1 (PD-L1) inhibitor. 14 April 2021
Pharmaceutical
Johnson & Johnson’s jab is the latest COVID-19 vaccine to be linked to rare cases of blood clots, prompting US health agencies to recommend pausing its use pending a review. 13 April 2021
Biotechnology
In COVID-19 related news last week, Japan’s Takeda and Australia’s CSL Ltd reported that the project they are leading, dubbed CoVig-19 Plasma Alliance, had failed to meet Phase III goals, and is therefore concluded. AstraZeneca’s COVID-19 vaccine, now called Vaxzevria, came under the spotlight last week regarding possible links to blood clots. On the regulatory front, Acadia Pharmaceuticals had a setback, as the US Food and Drug Administration issued a complete response letter (CRL) relating to its supplementary Biologics License (sBLA) for Nuplazid as a treatment for dementia-related psychosis. FibroGen revealed that some false data had been presented on its drug candidate roxadustat for the treatment of chronic kidney disease-associated anemia. Also, Provention Bio revealed problems with its regulatory filing for diabetes candidate teplizumab. 11 April 2021
Biotechnology
The US Food and Drug Administration has accepted for Priority Review the Biologics License Application (BLA) seeking accelerated approval for tisotumab vedotin, that was submitted to the agency by Danish biotech firm Genmab and US firm Seagen. 10 April 2021
Biotechnology
Stock in Provention Bio fell 35% ahead of trading on Friday, after the firm reported a regulatory setback related to its novel diabetes med teplizumab. 9 April 2021
Pharmaceutical
In the USA, a partial clinical hold has been placed on a Phase Ib trial of RVU120, being undertaken by Krakow-based Ryvu Therapeutics. 9 April 2021
Biotechnology
In the USA, the National Institutes of Health (NIH) is to initiate a Phase II trial to investigate the causes of certain allergic reactions to mRNA-based vaccines. 8 April 2021
Generics
Indian drugmaker Zydus Cadila, part of Cadila Healthcare, has received final approval from the US Food and Drug Administration to market ibrutinib capsules, 70mg and 140mg, a generic of blockbuster cancer drug Imbruvica. 8 April 2021
Biotechnology
The US Food and Drug Administration has granted full approval to Trodelvy (sacituzumab govitecan-hziy) for adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (mTNBC) . 8 April 2021
Pharmaceutical
USA-based FibroGen’s shares plunged almost 30% to $24.09 in pre-market trading today, on a disclosure of false heart safety data from previous presentations for its regulatory filing with the Food and Drug Administration on its anemia treatment in chronic kidney disease (CKD) patients. 7 April 2021
Pharmaceutical
Shares of US drugmaker Acadia Pharmaceuticals plunged more than 17% to $21.18 by close of trading Monday, after it revealed receipt of a Complete Response Letter (CRL) from the US Food and Drug Administration regarding its supplemental New Drug Application (sNDA) for Nuplazid (pimavanserin) for the treatment of hallucinations and delusions associated with dementia-related psychosis (DRP). 6 April 2021
Biotechnology
Tennessee, USA-based firm Rare Disease Therapeutics (RDT) has secured a new expanded indication for its antivenom product Anavip (crotalidae immune). 6 April 2021
Biotechnology
The US Food and Drug Administration has approved Qelbree (viloxazine extended-release capsules) for the treatment of attention-deficit hyperactivity disorder (ADHD) in pediatric patients six to 17 years of age. 6 April 2021
Biotechnology
The US Food and Drug Administration has approved French pharma major Sanofi’s Sarclisa (isatuximab-irfc), in combination with Amgen's Kyprolis (carfilzomib) and dexamethasone, for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received one to three prior lines of therapy. 2 April 2021
Biotechnology
The US Food and Drug Administration has approved Praluent (alirocumab) injection for adult patients with homozygous familial hypercholesterolemia (HoFH), a genetic condition that causes severely high cholesterol. Praluent is not intended to be used alone but instead added to other treatments for HoFH. 2 April 2021
Biotechnology
Japan’s Taiho Oncology and Taiho Pharmaceutical - both owned by Otsuka Pharmaceutical – have announced that the US Food and Drug Administration has granted Breakthrough Therapy designation (BTD) for futibatinib (TAS-120), a covalently-binding FGFR inhibitor. 2 April 2021
Biotechnology
The US Food and Drug Administration has accepted its New Drug Application (NDA) for atogepant, an investigational, orally-administered calcitonin gene-related peptide (CGRP) receptor antagonist (gepant), for the preventive treatment of migraine in adults who meet criteria for episodic migraine. 31 March 2021
Pharmaceutical
The NDA Group, a regulatory, drug development and medical device consultancy, has released findings from the eighth annual comparison of drug approvals in the European Union (EU) and the USA. 31 March 2021
Biotechnology
The US Food and Drug Administration has issued a Complete Response Letter (CRL) regarding Merck & Co’s supplemental Biologics License Application (sBLA) seeking approval for Keytruda, for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC). 30 March 2021
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