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FDA approves Jazz’s Ziihera for HER2+ biliary tract cancer

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

Astellas’ loss is Apellis’ gain as FDA setback shakes up prospects

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

FDA accepts Dupixent sBLA resubmission for CSU

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

Syndax earns its 2nd drug approval in 2024 with Revuforj

The US Food and Drug Administration (FDA) has approved Syndax Pharmaceuticals’ Revuforj (revumenib). 18 November 2024

AlveoGene wins RPDD from FDA for AVG-002

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

FDA nod for AADC deficiency gene therapy from PTC

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

MeiraGTx wins 3 Rare Pediatric designations and updates on AAV-AIPL1

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

FDA slaps CRL on Ocaliva sNDA

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

FDA lifts clinical hold on Novavax' combo COVID-19 flu jab study

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

RAPT Thera ends zelnecirnon program after FDA worries

RAPT Therapeutics has announced the termination of its zelnecirnon program following regulatory concerns around safety. 12 November 2024

Autolus Therapeutics announces FDA approval of Aucatzyl

On November 8, the US Food and Drug Administration approved Aucatzyl (obecabtagene autoleucel; obe-cel) from US biopharma Autolus Therapeutics' 11 November 2024

FDA’s Orphan Products Grants Program awards significant funding for rare diseases

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

US FDA to consider Dizal’s first-in-class lung cancer drug

Shanghai-based Dizal has made a submission to the US Food and Drug Administration, seeking approval for sunvozertinib. 8 November 2024

J&J files with EMA and FDA for Darzalex in high-risk smouldering MM

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

FDA Rare Pediatric Disease status for AskBio’s AB-1003

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

Acadia Pharma sells priority review voucher for $150 million

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

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FDA approves REMS, advancing new drug disposal option for opioid analgesics

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

FDA accepts Ionis NDA for donidalorsen

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

Disc Medicine leaps as it gets positive FDA feedback

Disc Medicine today announced a successful End of Phase 2 with the US Food and Drug Administration (FDA) supporting the regulatory path forward, including potential for accelerated approval, for bitopertin in erythropoietic protoporphyria (EPP). 4 November 2024

FDA sets up Rare Diseases Innovation Hub

On Friday, the US Food and Drug Administration published the FDA Voices: “FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub,” by Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research (CDER) and Dr Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER). 4 November 2024

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Madrigal's efforts for resmetirom will set tone for future launches of NASH therapies, says analyst

Since the US Food and Drug Administration’s acceptance of Madrigal Pharmaceutical’s new drug application (NDA) and priority review for its THR-β agonist (resmetirom) in non-alcoholic steatohepatitis (NASH) in September 2023, the company has taken steps to fortify its leadership and financial position to gear up for the imminent launch of resmetirom. 7 December 2023

FDA delays review of Optinose's sNDA for Xhance

The US Food and Drug Administration (FDA) has extended by three months the review period of its supplemental new drug application (sNDA) requesting approval of Xhance (fluticasone propionate) as a treatment for chronic rhinosinusitis. 7 December 2023

As US FDA scrutiny intensifies on Indian drug facilities, a tangle of domestic factors deepens the challenge

In a major crackdown, the US Food and Drug Administration (FDA) has been conducting an increased number of inspections of pharmaceutical manufacturing facilities in India. 7 December 2023

Novartis receives FDA approval for Fabhalta in PNH

The US Food and Drug Administration (FDA) has approved Fabhalta (iptacopan) as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). 6 December 2023

FDA 'Breakthrough' status for Neurocrine's crinecerfont

Neurocrine Biosciences yesterday revealed it has received Breakthrough Therapy designation from the US Food and Drug Administration (FDA) for crinecerfont in congenital adrenal hyperplasia. 6 December 2023

J&J's TAR-200 wins breakthrough designation

Johnson & Johnson’s TAR-200 has been granted Breakthrough Therapy designation (BTD) to potentially treat patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC), who are ineligible for or elected not to undergo radical cystectomy. 5 December 2023

Safety setback for BTK blockers as FDA holds fenebrutinib trial

Roche subsidiary Genentech has provided an update on its multiple sclerosis program, revealing a partial trial stoppage for the oral BTK inhibitor fenebrutinib. 5 December 2023

US DoJ readout of Reproductive Rights Task Force meeting

In the latest move in the USA for rights to contraceptives and abortion drugs, on Monday, the Reproductive Rights Task Force (RRTF) provided a briefing to Attorney General Merrick Garland and Task Force Chair Associate Attorney General Vanita Gupta on the Department of Justice’s (DoJ) work to protect reproductive freedom under federal law. 5 December 2023

New FDA approval for Lilly's Jaypirca

The US Food and Drug Administration (FDA) on Friday approved another indication for Jaypirca (pirtobrutinib, 100mg and 50mg tablets) from US pharm major Eli Lilly . 4 December 2023

The week in pharma: action, reaction and insight – week to December 1, 2023

Among last week’s interesting research news, US biotech 89bio on Monday released encouraging new data on pegozafermin, its non-alcoholic steatohepatitis (NASH) candidate. Also, pharma major AbbVie reported long-awaited Phase II data for its lung cancer drug Teliso-V (telisotuzumab-vedotin). On the deal-making front, US RNA focused biotech Avidity Biosciences signed a collaboration with Bristol Myers Squibb, which could earn it as much as $2.3 billion. Of note, the US Food and Drug Administration (FDA) last week revealed it has received reports of T-cell malignancies associated with CAR-t cell therapy treatment and has instigated an investigation into the matter. AbbVie returned to the news limelight on Thursday, announcing it has reached agreement to acquire antibody-drug conjugates (ADCs) focused biotech ImmunoGen and its flagship cancer drug Elahere. 3 December 2023

FDA accepts Keytruda/Padcev sBLA for priority review

The US Food and Drug Administration (FDA) has accepted for priority review a new supplemental Biologics License Application (sBLA) seeking approval for Merck& Co’s blockbuster anti-PD-1 therapy Keytruda (pembrolizumab) in combination with Padcev (enfortumab vedotin-ejfv), an antibody-drug conjugate, for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (la/mUC). 1 December 2023

Karuna's schizophrenia drug NDA accepted by FDA

Boston, USA-based biopharma Karuna Therapeutics has announced the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for KarXT (xanomeline-trospium) to treat schizophrenia in adults. 30 November 2023

FDA investigating risk of malignancy linked with CAR-T cell therapies

The US Food and Drug Administration (FDA) yesterday said it has received reports of T-cell malignancies, including chimeric antigen receptor CAR-positive lymphoma, in patients who received treatment with BCMA- or CD19-directed autologous chimeric antigen receptor (CAR)-T cell immunotherapies. 29 November 2023

SpringWorks' Ogsiveo nabs first FDA approval for desmoid tumors

US biopharma SpringWorks Therapeutics has won a landmark approval from the US Food and Drug Administration (FDA). 28 November 2023

Abeona leaps, as FDA accepts its pz-cel BLA for priority review

The US Food and Drug Administration (FDA) has accepted and granted priority review for the Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), submitted by Abeona Therapeutics. 28 November 2023

The week in pharma: action, reaction and insight – week to November 24, 2023

After lengthy and tense negotiations, last week saw the UK government and trade group the Association of the British Pharmaceutical Industry (ABPI) and its members reach agreement on a new pricing framework for medicines. Across the Atlantic, the US Food and Drug Administration (FDA) said it would be holding an advisory committee meeting to review the supplemental Biological License Application (sBLA) for Bristol Myers Squibb and 2seventy bio’s multiple myeloma candidate Abecma, so delaying the final decision date. US Pharma giant Merck & Co agreed to acquire Caraway Therapeutics for up to $610 million, aiming to expand its neurodegenerative disease portfolio. Also of note, Germany’s Bayer saw its biggest share drop in a decade, after it said it is discontinuing development of blood thinning drug candidate asundexian, adding to other woes. 26 November 2023

FDA issues final guidance on developing drugs for COVID-19 treatment and prevention

The US Food and Drug Administration (FDA) yesterday issued the final guidance COVID-19: Developing Drugs and Biological Products for Treatment or Prevention. 23 November 2023

First-in-class blood cancer med could soon face regulatory test

It has been a challenging week for Germany's MorphoSys, with the firm’s share price falling as much as 40% in the last five days. 22 November 2023

Best practices for Orange Book listings

In 1984, Congress passed and the President signed into law the Drug Price Competition and Patent Term Restoration Act, better known as the Hatch-Waxman Act. 21 November 2023

FDA delays review of sBLA for Abecma for earlier use

Shares of US pharma major Bristol Myers Squibb fell more than 4% pre-market and partner 2seventy bio was down 17.6% at $1.76 on Monday, after the companies said the US Food and Drug Administration would miss its target date for potential approval of earlier use of a cancer therapy Abecma (idecabtagene vicleucel). 21 November 2023

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