HIV specialist ViiV Healthcare says that the US Food and Drug Administration has granted Breakthrough Therapy designation for its long-acting, injectable cabotegravir for HIV pre-exposure prophylaxis (PrEP). 18 November 2020
The US Food and Drug Administration has accepted for priority review the Biologics License Application (BLA) for avalglucosidase alfa for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency). 18 November 2020
On November 17, USA’s Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration announced additional steps to increase transparency regarding CDER and CBER’s review of the scientific information supporting the issuance of or revisions to an emergency use authorizations (EUAs) to promote public confidence in FDA’s scientific process and the appropriate use of authorized products. 18 November 2020
The high and rising cost of prescription drugs in the United States remains a concern for patients, physicians, payers, and policy makers. It is also a key priority for the US Food and Drug Administration, the Trump administration, and the Department of Health and Human Services (HHS). 16 November 2020
On Friday, the Food and Drug Administration granted accelerated approval for Keytruda (pembrolizumab), in combination with chemotherapy, for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS ≥10) as determined by an FDA approved test. 14 November 2020
Shares in East Coast, USA-based CNS disease specialist Supernus Pharmaceuticals ended the day down 15% on Tuesday, after a pair of regulatory setbacks from the US Food and Drug Administration. 11 November 2020
Stepping up the fight against the novel coronavirus, a new therapy to treat mild-to-moderate cases of COVID-19 has been approved by the US regulator, through an emergency use authorization (EUA). 10 November 2020
Hot on the heels of positive interim data for Pfizer and BioNTech’s candidate, another coronavirus vaccine program appears to be moving forward apace. 10 November 2020
US biotech Biogen gained 44% on Wednesday of last week when the US Food and Drug Administration (FDA) posted encouraging briefing documents on the company’s aducanumab filing for members of the Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee Meeting. 9 November 2020
The biggest M&A deal announced last week was Merck & Co’s $2.75 billion buy of VelosBio, while Sanofi’s acquisition of Kiadis was notable because the latter company’s future was in doubt just a year ago. Elsewhere, Bristol Myers Squibb had a major pipeline breakthrough and a US Food and Drug Administration briefing document on the Alzherimer’s candidate aducacumab sent shares in Biogen spiralling, even if a committee vote 6 November 2020
A third regulatory nod in just two days has been awarded to British drugmaker AstraZeneca, this time for Brilinta (ticagrelor), an oral P2Y12 receptor antagonist. 6 November 2020
US biotech bluebird bio looked set to open Thursday’s trading almost 14% lower after announcing an update on its US filing of LentiGlobin for sickle cell disease (bb1111). 5 November 2020
Regular pharma market observers distracted by the US elections had cause to suddenly look back towards the industry on Wednesday morning. 4 November 2020
An analysis published in the journal Alzheimer's & Dementia, authored by David Knopman, David Jones and Michael Greicius, has stated that a further Phase III trial of the investigational Alzheimer’s drug aducanumab has failed to demonstrate efficacy and should undergo a further Phase III study. 3 November 2020
The Food and Drug Administration has accepted for priority review the supplemental Biologics License Application (sBLA) for PD-1 inhibitor Libtayo (cemiplimab-rwlc) to treat patients with first-line locally advanced or metastatic non-small cell lung cancer (NSCLC) with ≥50% PD-L1 expression. 3 November 2020
The US Food and Drug Administration has approved the new drug application for Bronchitol (mannitol) inhalation powder submitted by the US subsidiary of Italian privately-held drugmaker Chiesi Farmaceutici. 2 November 2020
The US Food and Drug Administration yesterday, published the guidance for industry entitled “Referencing Approved Drug Products in Abbreviated New Drug Application (ANDA) Submissions.” 28 October 2020
A further boost for antibody-drug conjugate (ADC) Enhertu (trastuzumab deruxtecan) has come in the form of Priority Review designation from the US regulator. 28 October 2020
Today, the US Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Neimann-Pick disease, type C (NPC). 20 September 2024
Vanda Pharmaceuticals’ shares were down more than 10% at $4.45 pre-market today, as the) company provided an unfavorable update on its tradipitant development program. 19 September 2024
Applied Therapeutics saw its stock price climb by over two-thirds after an encouraging update on the US Food and Drug Administration review of govorestat. 19 September 2024
Pharma giant Merck & Co has announced that the US Food and Drug Administration (FDA) has approved Keytruda (pembrolizumab), alongside pemetrexed and platinum chemotherapy, for the first-line treatment of adults with unresectable advanced or metastatic malignant pleural mesothelioma (MPM). 18 September 2024
AstraZeneca’s Fasenra (benralizumab) has been approved in the USA for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA, the UK pharma major announced today. 18 September 2024
Yesterday, the US Food and Drug Administration (FDA) issued a draft guidance, “Integrating Randomized Controlled Trials for Drug and Biological Products into Routine Clinical Practice” 18 September 2024
The US Food and Drug Administration (FDA) has approved Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) for the treatment of relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS), from Swiss pharma giant Roche. 16 September 2024
The Gastrointestinal Drugs Advisory Committee (GIDAC) of the US Food and Drug Administration (FDA) met on Friday to discuss Intercept Pharmaceuticals’ supplemental New Drug Application (sNDA) for Ocaliva (obeticholic acid, OCA). 16 September 2024
Oligonucleotide therapeutics are rapidly emerging as a promising class of drugs capable of addressing a variety of genetic and complex diseases by targeting RNA sequences.1 16 September 2024
On Thursday, the US Food and Drug Administration released a Drug Safety Communication warning that Veozah (fezolinetant), which is used to treat hot flashes due to menopause, can cause rare but serious liver injury. 13 September 2024
The US Food and Drug Administration (FDA) has approved Tecentriq Hybreza (atezolizumab and hyaluronidase-tqjs), the first and only PD-(L)1 inhibitor for subcutaneous (SC) injection for patients in the USA, Swiss pharma giant Roche subsidiary Genentech announced yesterday. 13 September 2024
The US Food and Drug Administration (FDA) has approved Tremfya (guselkumab) for the treatment of adults with moderately to severely active ulcerative colitis (UC). 12 September 2024
Intercept Pharmaceuticals’ Ocaliva (obeticholic acid) will come under the microscope when the US Food and Drug Administration’s (FDA) Gastrointestinal Drugs Advisory Committee meets on Friday. 12 September 2024
The US Food and Drug Administration (FDA) yesterday announced revised final guidance for industry entitled, “ANDA Submissions – Amendments to Abbreviated New Drug Applications under GDUFA.” 12 September 2024
Cartesian Therapeutics today announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to Descartes-08 for the treatment of juvenile dermatomyositis (JDM). 9 September 2024
ProKidney announced strategic updates to its Phase III program for rilparencel, an investigational treatment to potentially preserve kidney function in patients with type 2 diabetes and advanced chronic kidney disease (CKD). 9 September 2024
Last week’s research news included French pharma major Sanofi releasing mixed Phase III results for it tolebrutinib in progressive multiple sclerosis. Also of note, US vaccine developer Vaxcyte announced strong early-stage trial results for its VAX-31 in pneumococcal disease. On the regulatory front, US drugmaker Travere Therapeutics gained full approval from the US Food and Drug Administration (FDA) for its kidney disease drug Filspari. US cancer drug developer IN8bio announced a prioritization of its R&D pipeline to focus on INB-100 in acute myeloid leukemia, along with a workforce cutback. 8 September 2024
San Diego, USA-based Travere Therapeutics saw its shares close up more than 8% at $10.75 yesterday on positive news form the US regulator. 6 September 2024