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Pharmaceutical
Sentynl Therapeutics, wholly owned by Zydus Lifesciences, has announced that the US Food and Drug Administration has accepted its New Drug Application for CUTX-101, a potential first treatment for Menkes disease. The application has been granted priority review, with a target action date set for June 30, 2025. 7 January 2025
Pharmaceutical
rug Administration (FDA) approved an update to the US Prescribing Information (USPI) for Pyrukynd (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency, Agios Pharmaceutical revealed in a Security and Exchange Commission (SEC) notice. 7 January 2025
Biotechnology
Among significant news in a holiday spattered week, Dutch biotech argenx gained another approval in Japan for its Vyvdura (efgartigimod alfa and hyaluronidase-qvf), this time for adult patients with demyelinating polyneuropathy (CIDP). Axsome Therapeutics released mixed results from the completed Phase III program for its Alzheimer’s agitation candidate AXS-05 (dextromethorphan-bupropion and aminoketone). Also, Sangamo plunged on the news that Pfizer had unexpectedly pulled out of their deal on the development of hemophilia A candidate giroctocogene fitelparvovec (giro-vec). 5 January 2025
Biotechnology
US biotech Capricor Therapeutics today announced the completion of the submission of its Biologics License Application (BLA) to the US Food and Drug Administration (FDA) seeking full approval for deramiocel, an investigational cell therapy, to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy 2 January 2025
Pharmaceutical
Kazia Therapeutics, an oncology-focused Australian drug developer, has been advised by the US Food and Drug Administration (FDA) that accelerated approval for its brain cancer drug, paxalisib, is unlikely. 2 January 2025
Pharmaceutical
The US Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) under the accelerated approval pathway for avutometinib, an oral RAF/MEK clamp, in combination with defactinib. 2 January 2025
Pharmaceutical
The US Food and Drug Administration (FDA) is reopening the comment period for the Federal Register notice (FRN), “Evaluating the Immunogenicity Risk of Host Cell Proteins in Follow-on Recombinant Peptide Products: Establishment of a Public Docket: Request for Information and Comments” due to high interest from industry. 31 December 2024
Biotechnology
The US Food and Drug Administration (FDA) on Friday approved nivolumab and hyaluronidase-nvhy, under the trade name Opdivo Qvantig, from US pharma major Bristol Myers Squibb, for subcutaneous injection. 28 December 2024
Biotechnology
The US Food and Drug Administration (FDA) has approved Tevimbra (tislelizumab-jsgr), in combination with platinum and fluoropyrimidine-based chemotherapy, for the first-line treatment of unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma (G/GEJ) in adults whose tumors express PD-L1 (≥1), Sino-China biotech BeiGene. 27 December 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has issued the Federal Register notice, Modifications to Labeling of Buprenorphine-Containing Transmucosal Products for the Treatment of Opioid Dependence, to encourage the submission of supplemental new drug applications to modify the labeling statements for buprenorphine-containing transmucosal products for the treatment of opioid dependence (BTODs). 27 December 2024
Generics
The US Food and Drug Administration (FDA) revealed on Monday that it has approved the first generic referencing Victoza (liraglutide injection) 18 milligram/3 milliliter. 24 December 2024
Pharmaceutical
US pharma major Eli Lilly today announced the US Food and Drug Administration (FDA) approved Zepbound its (tirzepatide) as the first and only prescription medicine for adults with moderate-to-severe obstructive sleep apnea (OSA) and obesity. 23 December 2024
Pharmaceutical
The US Food and Drug Administration (FDA) on Friday approved an expanded indication for Imcivree (setmelanotide), from Boston, USA-based Rhythm Pharmaceuticals, to include children as young as two years old. 23 December 2024
Pharmaceutical
US pharma giant Pfizer on Friday revealed that the US Food and Drug Administration (FDA) has approved its Braftovi (encorafenib) in combination with cetuximab and mFOLFOX6 (fluorouracil, leucovorin, and oxaliplatin) for the treatment of patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation, as detected by an FDA-approved test. 23 December 2024
Biotechnology
Denmark’s Novo Nordisk said late Friday that the US Food and Drug Administration (FDA) has approved Alhemo (concizumab-mtci) injection. 22 December 2024
Pharmaceutical
Denmark’s Zealand Pharma has been hit with a complete response letter (CRL) from the US regulator for glepaglutide, a long-acting GLP-2 analog. 20 December 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has approved Tryngolza (olezarsen) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a rare, genetic form of severe hypertriglyceridemia (sHTG) that can lead to potentially life-threatening acute pancreatitis (AP). 20 December 2024
Pharmaceutical
Privately-held Xcovery Holdings, an oncology-focused pharma company, has announced that the US Food and Drug Administration (FDA) has approved Ensacove (ensartinib) for the treatment of patients with anaplastic lymphoma kinase (ALK)-positive locally advanced or metastatic non-small cell lung cancer (NSCLC). 19 December 2024
Biotechnology
December 19, 2024
The US Food and Drug Administration (FDA) approved Ryoncil (remestemcel-L-rknd), from Australia’s Mesoblast, whose shares rocketed 54% to A$3.05 on the news. 19 December 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has approved Gilead Sciences Yeztugo (lenacapavir) - the company’s injectable HIV-1 capsid inhibitor - as pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV in adults and adolescents weighing at least 35kg. 19 June 2025
Biotechnology
The US Food and Drug Administration has approved US biotech Incyte’s Monjuvi (tafasitamab-cxix) with lenalidomide and rituximab for adults with relapsed or refractory follicular lymphoma (FL). 19 June 2025
Biotechnology
The biopharmaceutical industry is grappling with a complex mid-year outlook in 2025, as accelerating innovation collides with mounting policy and market uncertainty. From US drug pricing reforms to global tariff tensions, sentiment has taken a hit - reflected in a 16% drop in industry optimism. 19 June 2025
Biotechnology
EY today released its 35th annual Biotech Beyond Borders Report. This finds that the biopharma industry is currently at an inflection point – grappling with a unique environment of macroeconomic uncertainty and constrained access to capital, including high interest rates, inflation, shifting regulatory policies and tariff turmoil. 18 June 2025
Pharmaceutical
The US Food and Drug Administration has announced the Commissioner’s National Priority Voucher (CNPV) program aimed at enhancing the health interests of Americans. 18 June 2025
Biotechnology
US biotech Dyne Therapeutics has revised its regulatory approach for DYNE-101, a treatment in development for myotonic dystrophy type 1 (DM1), after discussions with the American medicines regulator. 18 June 2025
Biotechnology
Australian biotech CSL has announced that the US Food and Drug Administration (FDA) has approved Andembry (garadacimab-gxii), the only treatment targeting factor XIIa for prophylactic use to prevent attacks of hereditary angioedema (HAE) in patients aged 12 years and older. 17 June 2025
Biotechnology
Notable research news last week included US biopharma Avidity Biosciences announcing a positive update on its muscular dystrophy candidate delpacibart braxlosiran (del-brax), and Dutch firm NewAmsterdam released new Phase III on its investigational Alzheimer’s drug obicetrapib. US Health Secretary Robert F Kennedy Jr announced he has dismissed an independent panel of vaccine experts in order to restore trust in vaccines. Also of note, Merck & Co gained US food and Drug Administration (FDA) for its Enflonsia for the prevention of respiratory syncytial virus (RSV). 15 June 2025
Biotechnology
The Food and Drug Administration approved US pharma giant Merck & Co’s mega-blockbuster cancer drug Keytruda (pembrolizumab) for a new indication. 13 June 2025
Biotechnology
US mRNA specialist Moderna today announced that the US Food and Drug Administration (FDA) has approved mRESVIA (mRNA-1345), the company's respiratory syncytial virus (RSV) vaccine, for the prevention of lower respiratory tract disease (LRTD) caused by RSV in individuals 18-59 years of age who are at increased risk for disease. 13 June 2025
Pharmaceutical
The American medicines regulator has approved Zusduri (mitomycin), a hydrogel-based treatment developed by US biotech UroGen Pharma, for adults with recurrent low-grade, intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). 13 June 2025
Biotechnology
The US Food and Drug Administration (FDA) yesterday approved Nuvation Bio’s Ibtrozi (taletrectinib), a kinase inhibitor, for adults with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC). 12 June 2025
Pharmaceutical
Gilead Sciences has paused five clinical trials of its investigational HIV drugs GS-1720 and GS-4182 after the American medicines regulator raised safety concerns. The once-weekly oral regimen was being studied as an alternative to the company’s blockbuster daily pill, Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide). 11 June 2025
Biotechnology
Shares of Avidity Biosciences were up almost 5% at $38.00 pre-market today, as it announced the accelerated approval regulatory pathway in the USA is open for delpacibart braxlosiran (del-brax) in the treatment of facioscapulohumeral muscular dystrophy (FSHD). 9 June 2025
Pharmaceutical
George Medicines, a late-stage UK-based biopharmaceutical company focused on addressing unmet needs in cardiometabolic disease, revealed a regulatory approval that it says could reshape hypertension treatment in the USA. 9 June 2025
Biotechnology
Regulatory news last week included US biotech Moderna receiving Food and Drug Administration (FDA) approval for its new COVID-19 Vaccine mNEXSPIKE. On the M&A front, French pharma major Sanofi announced it is punting more than $9 billion to acquire Blueprint Medicines and its Ayvakit, BLU-808 and immunology pipeline. USA-based Cullinan Therapeutics has entered into a license deal with China's Genrix Bio for the latter’s velinotamig for multiple myeloma. Also of note, RegeNXBio released what lookedlike good results for its investigational Duchenne muscular dystrophy drug RGX-202. 8 June 2025
Biotechnology
German genetic medicine company ViGeneron has announced a rebrand to become VeonGen Therapeutics. 5 June 2025
Biotechnology
US rare disease meds developer Sarepta Therapeutics revealed that its rAAVrh74 viral vector used in the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy type 2E/R4, has been granted platform technology designation by the US Food and Drug Administration. 5 June 2025
Pharmaceutical
The American medicines regulator has committed to reviewing the safety of the abortion pill mifepristone, amid renewed political scrutiny and pressure from conservative lawmakers. 4 June 2025
Pharmaceutical
The American medicines regulator has approved Bayer’s Nubeqa (darolutamide) for broader use in patients with metastatic castration-sensitive prostate cancer (mCSPC), marking a step forward for the drugmakeFood and Drug Administraton,r’s prostate cancer franchise. 4 June 2025
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