The US Food and Drug Administration today alerted patients and health care professionals that a clinical trial (OCEAN, Study OP-103) evaluating Pepaxto (melphalan flufenamide) with dexamethasone to treat patients with multiple myeloma showed an increased risk of death. 28 July 2021
Chicago, USA-based antimicrobial resistance specialist Iterum Therapeutics announced a regulatory setback for its candidate, oral sulopenem, on Monday. 27 July 2021
US pharma giant Merck & Co’s blockbuster anti-PD-1 therapy Keytruda (pembrolizumab) has won US regulatory approval for yet another cancer indication. 27 July 2021
The latest voluntary withdrawal stemming from the US regulator’s review of accelerated approvals is Bristol Myers’ nod for Opdivo (nivolumab) in liver cancer. 26 July 2021
Last week saw the announcement of two large licensing agreements. First, Gilead Sciences’ subsidiary Kite entered into a deal with BioNTech, whereby the German biotech, mostly in the news due to its COVID-19 vaccine developed with Pfizer, has acquired an R&D platform and clinical manufacturing facility in Maryland. The second deal was US pharma giant Pfizer signing an agreement with Arvinas for rights to the latter’s breast cancer candidate, ARV-471, that could cost it more than $2 billion. On the regulatory front, US biotech bluebird bio gained regulatory approval from the European Commission for its gene therapy Skysona. Also, Albireo received approval from both the US Food and Drug Administration and the European Medicines for its rare liver disease drug Bylvay. 25 July 2021
The US Food and Drug Administration yesterday approved Bydureon and Bydureon BCie (exenatide extended-release) injection to be used in addition to diet and exercise to improve glycemic control (blood sugar levels) in pediatric patients 10 years or older with type 2 diabetes. 23 July 2021
The US Food and Drug Administration approved Keytruda (pembrolizumab) from Merck & Co in combination with Japanese drugmaker Eisai’s Lenvima (lenvatinib) for patients with advanced endometrial carcinoma' 22 July 2021
US clinical-stage biotech Magenta Therapeutics’ shares closed down more than 9% at $8.07 on Wednesday, after the company revealed it has received a clinical hold letter from the US Food and Drug Administration related to its Investigational New Drug Application (IND) filed in June 2021 to initiate a Phase I/II clinical trial of MGTA-117 in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). 22 July 2021
Venclexta (venetoclax), in combination with azacytidine, has been granted Breakthrough Therapy Designation (BTD) by the US Food and Drug Administration (FDA) for previously untreated intermediate, high- and very high-risk myelodysplastic syndromes (MDS) based on the revised International prognostic scoring system (IPSS-R). 21 July 2021
The US Food & Drug Administration approval of Bylvay (odevixibat), the first drug approved for the treatment of pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC), developed by Boston, USA-based Albireo Pharma. 21 July 2021
The US Food and Drug Administration has approved fexinidazole as the first all-oral treatment for both stages of the Trypanosoma brucei gambiense form of sleeping sickness (Human African trypanosomiasis) in patients six years of age and older and weighing at least 20 kg. 21 July 2021
Shares of California, USA-based cardiorenal specialist Ardelyx plunged more than 70% to $2.28 in after-hours trading on Monday, as the company revealed the US regulator is not ready to approve its tenapanor for dialysis patients. 20 July 2021
The US Food and Drug Administration (FDA) has granted Priority Review designation for the Biologics License Application (BLA) for the COVID-19 vaccine from Pfizer and BioNTech. 19 July 2021
The US Food and Drug Administration (FDA) approved Vaxneuvance (pneumococcal 15-valent conjugate vaccine) for active immunization for the prevention of invasive disease caused by Streptococcus pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, 22F, 23F and 33F in adults 18 years of age and older. 19 July 2021
Licensing agreements featured heavily in last week’s news, starting with US biotech Biogen entering into an agreement that could cost it $1 billion to get rights to a BTK inhibitor – orelabrutinib for multiple sclerosis – from China’s InnoCare Pharma. Denmark’s Novo Nordisk in-licensed rights to Prothena’s ATTR amyloidosis program, and PRX004, in a deal worth up to $1.2 billion. Also, Ionis Pharmaceuticals last Tuesday exercised an option on fellow USA-based Bicycle Therapeutics’ oligonucleotide targeting the transferrin receptor 1. On the M&A front, Eli Lilly revealed on Wednesday it was acquiring privately-held US company Protomer Technologies for what could be a $1 billion transaction, getting rights to its glucose-sensing insulin project. Additionally, the US Food and Drug Administration took issue with Amgen’s promotion of its cancer drug Neulasta, which the agency said could undermine confidence in biosimilars. 18 July 2021
Shares of USA-based Kadmon Holdings shot up more than 20% to $4.28 on Friday, after the company revealed its pharma unit had received its first regulatory approval, for Rezurock (belumosudil). 17 July 2021
The US Food and Drug Administration on Friday approved a new use for Prograf (tacrolimus) based on a non-interventional (observational) study providing real-world evidence (RWE) of effectiveness. 17 July 2021
USA-based FibroGen's shares have dropped by more than a third after a scientific panel of the US Food and Drug Administration voted against approval of roxadustat. 16 July 2021
Looking to overturn a regulatory rebuff, New Jersey, USA’s Merck & Co has announced positive event-free survival (EFS) data from the pivotal neoadjuvant/adjuvant Phase III study KEYNOTE-522. 16 July 2021
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