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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
Bristol-Myers Squibb could now have to wait till May 2019 to hear if the US Food and Drug Administration (FDA) has approved its supplemental Biologics License Application (sBLA) for the use of Opdivo (nivolumab) plus low-dose Yervoy (ipilimumab) in metastatic first-line non-small cell lung cancer. 22 October 2018
Biotechnology
The US Food and Drug Administration has granted Rare Pediatric Disease (RPD) designation to lonafarnib in the treatment of both Hutchinson-Gilford Progeria Syndrome (HGPS or progeria) and progeroid laminopathies. 22 October 2018
Biotechnology
The US Food and Drug Administration has approved Dupixent (dupilumab) as an add-on maintenance therapy in patients with moderate-to-severe asthma aged 12 years and older with an eosinophilic phenotype or with oral corticosteroid-dependent asthma. 20 October 2018
Pharmaceutical
The US Food and Drug Administration’s Gastrointestinal Drugs Advisory Committee voted unanimously (10 to 0) that the risk-benefit profile of prucalopride (SHP555) supports the approval of this New Drug Application (NDA). 19 October 2018
Biotechnology
The US Food and Drug Administration approved Talzenna (talazoparib), a poly (ADP-ribose) polymerase (PARP) inhibitor, for patients with deleterious or suspected deleterious germline BRCA-mutated (gBRCAm), HER2 negative locally advanced or metastatic breast cancer. 17 October 2018
Pharmaceutical
The White House has signalled its intention to press ahead with proposals to force drugmakers to include drug pricing information in direct-to-consumer advertising, despite the apparent inability of Congress to sign up to the idea. 16 October 2018
Biotechnology
US Food and Drug Administration Commissioner Scott Gottlieb has issued a statement outlining the steps his agency is taking to modernize programs and “advance opportunities for developing more targeted therapies.” 16 October 2018
Biotechnology
Shares of RNAi therapeutics company Alnylam Pharmaceuticals fell 2.37% to $80.01 by close of trading on Monday, after the company revealed that it would not aim for accelerated approval of givosiran. 16 October 2018
Pharmaceutical
As of September 14, 2018, the US Food and Drug Administration had approved the launch of 36 novel drugs, not including vaccines, blood products and biologics.1 With less than three months left in 2018, is there still chance to surpass last year’s 21-year high of 46 approvals?2 This is certainly the hope for those invested in addressing the many unmet needs of patients who require novel treatments for symptom relief, says Dr Nicola Davies in her exclusive article for The Pharma Letter. 16 October 2018
Pharmaceutical
UK-based Acacia Pharma today provided an update following receipt of a Complete Response Letter (CRL) from the US Food and Drug Administration in respect of its New Drug Application (NDA) for Barhemsys (amisulpride injection) on October 5, 2018. 15 October 2018
Pharmaceutical
Regulatory news of interest last week included the US Food and Drug Administration’s approval of Akcea Therapeutics rare disease drug Tegsedi, but it came with a black box warning, and FDA acceptance of Karyopharm’s selinexor New Drug Application for multiple myeloma. On the deal making front, there was Roche's agreement to access Ionis Pharmaceuticals’ age-related macular degeneration research program, while in terms of drug trials, safety concerns caused Affimed to discontinue its AFM11 leukemia study. 14 October 2018
Biotechnology
The US Food and Drug Administration has lifted the clinical hold placed in May this year and accepted the Investigational New Drug application (IND) for CTX001 for the treatment of sickle cell disease (SCD). 11 October 2018
Biotechnology
France’s Valneva has won US FDA approval for an accelerated dosing regimen of its Japanese encephalitis (JE) vaccine, Ixiaro. 11 October 2018
Generics
US Food and Drug Administration (FDA) Commissioner Scott Gottlieb has championed the cause of generics since starting in the role nearly 18 months ago. 10 October 2018
Pharmaceutical
The market value of US biopharma Trevena is worth just a third of its worth at Monday’s close after a US Food and Drug Administration (FDA) report raised concerns with its opioid injection to treat acute pain. 10 October 2018
Biotechnology
Shares of US biotech firm Akcea Therapeutics gained 4.39% to $31.85 by close of trading and a further 2.04% to $32.50 in after-hours on Friday, after the company revealed that the US Food and Drug Administration had approved Tegsedi (inotersen) for the treatment of the polyneuropathy of hereditary transthyretin (hATTR) amyloidosis in adults. 8 October 2018
Pharmaceutical
The US Food and Drug Administration (FDA) has accepted for filing with priority review the New Drug Application (NDA) seeking accelerated approval for Karyopharm Therapeutics’ selinexor. 8 October 2018
Pharmaceutical
US Food and Drug Administration approvals dominated much of the news last week. Among these was the FDA’s approval of Regeneron’s Libtayo, marking the firm’s entry into the immuno-oncology sector. The FDA also cleared for marketing: Pfizer’s Vizimpro for a rare form of lung cancer; Paratek’s antibiotic Nuzyra and acne drug Seysara; and Roche’s hemophilia A drug Hemlibra in a much wider patient population, so boding well for the Swiss pharma giant’s moves to expand out of its core oncology franchise that is facing copycat competition. On the deal-making front, there was the unexpected news that Janssen is pulling out of its agreement with Aduro Biotech on cancer drug candidates. 7 October 2018
Biotechnology
There was good news for Roche with the coveted wider indication for its hemophilia drug in the USA, so increasing its focus on diseases beyond cancer to help replace revenue from older products that have, or will soon, lost patent exclusivity. 5 October 2018
Biosimilars
Kevin Noonan, a partner with McDonnell Boehnen Hulbert & Berghoff, provides commentary on the US Food and Drug Administration's Biosimilars Action Plan in an Expert View piece. 4 October 2018
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