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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
Chinese biopharma Hutchmed today revealed that it completed the rolling submission of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for fruquintinib, its highly selective and potent oral inhibitor of VEGFR-1, -2 and -3, for the treatment of refractory metastatic colorectal cancer (CRC). 31 March 2023
Biotechnology
The US Food and Drug Administration (FDA) has lifted a partial clinical hold on the Phase I/II VELA trial of BLU-222, said the drugs developer, Blueprint Medicines, sending the US biotech’s shares up 4% to $42.57. 30 March 2023
Biotechnology
A short delay to bluebird bio’s US regulatory submission for lovo-cel (lovotibeglogene autotemcel) appears to have spooked investors, with shares in the company falling by a quarter. 30 March 2023
Biotechnology
New Jersey, USA-based cancer giant Merck has converted a conditional nod for its checkpoint blocker, Keytruda (pembrolizumab), into regular approval. 30 March 2023
Biotechnology
Shares of USA/Israel-based biotech BrainStorm Cell Therapeutics rose as much as 13% yesterday on more positive news from the US regulator, which in February issued a refusal-to-file (RTF) letter regarding the submission for its cell therapy NurOwn (autologous MSC-NTF cells). 28 March 2023
Pharmaceutical
Shares of Incyte fell 2.8% to $70.23 on Friday, after the US Food and Drug Administration (FDA) unexpectedly rejected an extended-release version of its blockbuster drug, Jakafi (ruxolitinib). 27 March 2023
Biotechnology
Clinical trial news last week included Japanese pharma giant Takeda presenting strong Phase IIb data at the American Academy of Dermatology meeting on its psoriasis asset TAK-279, which it licensed from Nimbus Therapeutics for a massive $4 billion upfront. Also, the USA’s 89bio was boosted last Wednesday with the release of encouraging results for its non-alcoholic steatohepatitis (NASH) candidate pegozafermin. And Sanofi last Thursday released stunning results for its blockbuster drug Dupixent in the treatment of chronic obstructive pulmonary disease (COPD). Elsewhere, in a move to broaden its pipeline, Germany’s BioNTech has licensed an oncology candidate, ONC-392, from USA-based OncoC4, paying an upfront $200 million. Also, last week saw US biotech Incyte gain Food and Drug Administration (FDA) approval of Zynyz for the treatment of locally advanced Merkel cell carcinoma. 27 March 2023
Biotechnology
The US Food and Drug Administration (FDA) has approved Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older, Dutch biotech firm Pharming announced late Friday. 25 March 2023
Biotechnology
US biotech Biogen is focusing on the positives after a US Food and Drug Administration (FDA) advisory committee (AdCom) meeting on tofersen, an investigational product for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). 23 March 2023
Pharmaceutical
Cidara Therapeutics saw its shares fall 10% to $1.71 in after-hours trading yesterday, despite receiving a regulatory approval for a new drug. 23 March 2023
Pharmaceutical
US pharma major AbbVie closed down almost 2% at $153.67 yesterday, after it revealed a regulatory setback for its investigational Parkinson’s treatment. 23 March 2023
Biotechnology
Delaware, USA-based firm Incyte has secured approval in the USA for its checkpoint blocker Zynyz (retifanlimab-dlwr). 23 March 2023
Biosimilars
Sandoz, the soon to be spun-out generics and biosimilars unit of Swiss pharma giant Novartis, today announced that the US Food and Drug Administration (FDA) approved a citrate-free high-concentration 21 March 2023
Generics
Zydus Lifesciences today revealed it has received final approval for Tofacitinib Tablets, 5mg and tentative approval for Tofacitinib Tablets, 10mg, generic versions of US pharma giant Pfizer’s Xeljanz from the US Food and Drug Administration (FDA). 21 March 2023
Biotechnology
Biopharma M&A news last week included the largest deal in around four years, with US pharma giant Pfizer last Monday confirming it is to acquire biotech firm Seagen, but with the price of $43 billion much higher than the $30 billion rumored prior to the formal announcement. Also, French pharma major Sanofi announced a $2.9 billion takeover bid for USA-based Provention Bio, along with its diabetes drug Tzield. Last week also saw the US Centers for Medicare and Medicaid Services (CMS) announce more details of its plan for savings on Medicare Part B drugs. Additionally, the US Food and Drug Administration made a surprise U-turn, calling for an advisory committee meeting ahead of its anticipated regulatory decision for Sarepta Therapeutics' Duchenne muscular dystrophy (DMD) candidate SRP-9001. 19 March 2023
Pharmaceutical
A US Food and Drug Administration (FDA) advisory committee has voted by 16 to one that data support the safety and effectiveness of Paxlovid (nirmatrelvir/ritonavir) for mild-to-moderate COVID-19 in adults at high risk for progression to severe illness. 17 March 2023
Pharmaceutical
A new approval for Swiss pharma giant Novartis will open up a broader use for Tafinlar (dabrafenib) plus Mekinist (trametinib) in oncology. 17 March 2023
Biotechnology
US precision genetic medicine for rare diseases developer Sarepta Therapeutics yesterday revealed that, at its late cycle meeting for the SRP-9001 (delandistrogene moxeparvovec) biologics license application (BLA), the US Food and Drug Administration’s Office of Therapeutics (OTP) has determined that an advisory committee meeting will be held for the drug candidate in advance of the May 29, 2023 regulatory action date. 17 March 2023
Biotechnology
USA-based tumor drug resistance specialist Deciphera Pharmaceuticals yesterday announced that its Qinlock (ripretinib) has been included in the latest National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology as a preferred regimen for second-line gastrointestinal stromal tumor (GIST) patients intolerant to sunitinib. 15 March 2023
Biotechnology
The US Food and Drug Administration (FDA) yesterday announced it has amended the emergency use authorization (EUA) for Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine, co-developed by US pharma giant Pfizer and Germany’s BioNTech. 15 March 2023
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